These RA/QA news rolls are available in full only to paid subscribers. Upgrade here. If you haven’t already taken our reader survey, it should only take 1 minute here.

This is our biweekly recap of news and industry happenings curated for our paid subscribers. Our goal is to bring you the headline news and the stories, research, and insights that might be harder to find.

If you haven’t upgraded to a paid subscription yet, you can do so here. Also, make sure to follow us on LinkedIn and catch the latest episodes of our podcast, The Life Science Rundown.

Here are some key items from the past couple of weeks.

💊 = Pharma, biologic, and biotech.
💉 = Medical device, medtech, diagnostics, and digital health.

FDA Leadership and Administration

• Simple Reform restructuring takes effect: The FDA’s Simple Reform restructuring formally took effect on October 1, the first day of fiscal 2027. Most of the changes fall in shared services, such as the Office of Operations and a new enterprise technology office, while the product centers saw more targeted changes. Those include a new Office of Innovation and Clinical Trial Modernization at CDER with one division for real-world evidence and another for artificial intelligence. Marie Bradley, a longtime CDER real-world evidence policy leader, will lead the RWE division, according to her LinkedIn announcement. Leaders for the new office itself and its AI division have not yet been announced.

• Two new FDA offices still lack permanent leaders: The FDA is still recruiting directors for two offices created under Simple Reform, the Office of Health Advancement and the Office of National Health Security. The Office of Health Advancement takes over many functions of the former Office of the Chief Medical Officer, including the offices of Clinical Policy, Orphan Products Development, Combination Products, Women’s Health, Minority Health, and Pediatric Therapeutics. The Office of National Health Security, which sits in the Office of the Commissioner, will oversee public health preparedness and supply chain resiliency staff. An FDA spokesperson said Acting Chief Information Officer Sanjay Sahoo is also serving as acting director of the new Office of Mission Information Technology Services, and that “organizational charts will be published as soon as possible.” The national health security vacancy comes as U.S. officials monitor reports of a pneumonic plague case in Russia, though there is no indication of wider spread.

• New acting director for the Office of Generic Drugs: 💊 Darby Kozak is now acting director of the FDA’s Office of Generic Drugs, the FDA confirmed. Kozak was previously the office’s deputy director. Former Director Iilun Murphy stepped down to take an associate director position overseeing regulatory science, a move first reported by the Pink Sheet.

• HHS acting general counsel departs: Robert Foster, HHS’s acting general counsel, has left the department, Endpoints News reported. Foster joined HHS in January 2025 and briefly served as acting chief counsel at the FDA. His LinkedIn page now lists him as the department’s former acting general counsel. HHS announced a reorganization of its Office of General Counsel in September that appears to eliminate the chief counsel for food, research, and drugs position Foster held from November 2025 to May 2026.

• FDA workforce still down for the year: Updated Office of Personnel Management data show the FDA has lost 744 net employees in 2026, with figures current through August. That is a large improvement from 2025, when the agency lost 3,880 employees to layoffs. Even so, ongoing retirements and departures appear to have offset the FDA’s renewed hiring.

• Klomp sets a five-year development goal: 💊 HHS Chief Counselor Chris Klomp said at The Wall Street Journal’s Future of Health Summit on October 7 that the U.S. should aim to cut the average time from concept to commercialization for new molecules from 15 years to five. He said that goal should be met “without a single ounce of compromise on safety and efficacy.” Klomp argued that when trials move overseas, “our best scientific minds sometimes follow,” which weakens the academic medical centers that would otherwise run those studies. He pointed to HHS’s Operation TrialBlazer, which is designed to keep trials in the U.S., and to a new ARPA-H initiative.

• ARPA-H launches a clinical trial infrastructure push: 💊 The Advanced Research Projects Agency for Health, working with HHS, launched an initiative on September 30 to build technical infrastructure for continuous, adaptive clinical trials. The program, called SURPASS, will fund teams over five years that propose a phase-less platform trial in a single disease area, with goals that include simulation-based design environments and AI-enabled operational systems, according to the solicitation notice. ARPA-H is also launching projects on AI for trial site activation, national data-sharing infrastructure, and patient-contributed real-world data, all tied to HHS’s Operation TrialBlazer. The FDA launched a related AI-Enabled Optimization of Early-Phase Clinical Trials pilot in April, which appears not to have advanced since comments closed in late June.

FDA General

• FDA lays out a Transparency Roadmap: 💊 The FDA published a blog post on October 2 by Lowell Zeta, deputy commissioner for strategic initiatives and special counsel, describing a new Transparency Roadmap that could change long-standing practices for disclosing applications and clinical data. Zeta wrote that the FDA will encourage sponsors “to make public the existence of an Investigational New Drug Application (IND) upon filing,” though the agency estimates 96% of novel drug or biologic filings are already publicly known. The FDA also plans more post-approval data disclosures, with redactions for confidential commercial and trade secret information, including publication of Clinical Study Reports for approved applications, alignment with EMA data transparency practices where feasible, and a pilot of expanded clinical dataset transparency. The EMA has published clinical data submitted to support marketing applications since 2016, though only through a specific portal and to qualified individuals, and it publishes specific data related to approvals, including clinical overviews, summaries, study reports, and protocols. The post does not mention Investigational Device Exemption applications.

• Rare Disease Framework Initiative released: 💊 The FDA and HHS released the FDA’s Rare Disease Framework Initiative on October 14, calling for a “clear, consistent regulatory approach tailored to the specific challenges of rare disease drug development.” The stated goal is to make “fit-for-purpose evidentiary expectations” the norm for all rare disease applications. The document mainly lays out the case for change and the framework’s guiding principles, which include more predictable decision-making, embedding the patient voice in development and review, drawing on external expertise, and risk-based approaches to manufacturing and inspections. The FDA said it is considering revisions to existing regulations, possibly new ones, and a review of its guidance documents. OIRA cleared its release only days after receiving it. A Federal Register notice opens the white paper for comment, and according to the FDA and HHS announcement, the Reagan-Udall Foundation will host a public meeting on the initiative on October 28. The FDA and Reagan-Udall also scheduled an October 16 meeting on the patient voice in rare disease medical product development featuring several FDA rare disease leaders.

• Peptide compounding policy reaches the White House: 💊 A document titled “Interim Access Policy for Compounding Using Certain Peptides” is now under review at the White House Office of Information and Regulatory Affairs. The document has not been released, but its title suggests a temporary enforcement discretion policy for compounding certain unapproved peptides, likely including some of the peptides the Pharmacy Compounding Advisory Committee reviewed at a July meeting. That committee recommended six of seven peptides for the list that permits compounding, overriding FDA staff concerns. The Washington Post reported on October 7 that HHS and the White House were planning to allow the temporary sale of some previously restricted peptides while officials write new regulations, and The Atlantic previously reported that Heidi Overton has worked on peptide policy in her role on the Domestic Policy Council. It is not yet clear whether the policy would cover outsourcing facilities.

• FDA shares early thinking on ibogaine trials: 💊 The FDA published a request for information on October 6 sharing its “preliminary thinking” that early-stage ibogaine trials should test a single dose in an “intensively monitored” inpatient setting. The RFI suggests adults with opioid use disorder or post-traumatic stress disorder as possible study populations and asks 18 questions on trial design, safety, and oversight, given ibogaine’s known risks such as QTc prolongation. In a Washington Examiner op-ed the same day, Acting Commissioner Kyle Diamantas and CDER Director Mike Davis wrote that “simply opening the floodgates without proper guidelines would be dangerous and irresponsible.” The input could support new guidance building on the psychedelics guidance the FDA finalized in July. In April, President Trump directed HHS to support research on psychedelic treatments, including ibogaine. Comments are due November 20.

• Clinical trial enrollment action plan guidance under review: 💊💉 A new FDA guidance titled “Clinical Trial Enrollment Action Plans to Improve Representation of the U.S. Intended Use Population in Clinical Studies” arrived at the White House for review on October 6. The title closely resembles the FDA’s existing guidance, “Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies.” In a recent FDA Voices post, FDA center leaders Mike Davis, Karim Mikhail, and Michelle Tarver wrote that “trials that fail to enroll American patients can be less easily generalized to the population the products are intended to treat.”

• OTC monograph forecast looks like last year’s: 💊 The FDA released its annual forecast of over-the-counter drug monograph changes on September 30, listing updates planned for four monographs in fiscal 2027. For the cold and cough monograph, the FDA plans to address codeine’s status as a cough medicine ingredient, propose a warning for propylhexedrine products about the risk of abuse and misuse, and reassess the “risks and benefits of cough-cold drug products in children under age 6 years.” The internal analgesic monograph would add updated pregnancy labeling for NSAIDs and weight- and age-based acetaminophen dosing for children under 12. The other two updates would address the status of benzocaine and phenol oral health products and of ipecac for poison treatment. An archived version of the page shows the forecast is nearly identical to last year’s.

• Petition asks the FDA to restore its old homeopathy policy: 💊 A citizen petition from the Alliance for Natural Health-US and Meditrend, posted September 30, asks the FDA to revoke its current enforcement framework for homeopathic drugs and return to a 1988 compliance policy guide. The FDA’s December 2022 guidance treats homeopathic products as unapproved drugs and focuses enforcement on higher-risk products, such as those for serious conditions or vulnerable populations, or those with “significant quality issues.” The older CPG 400.400 essentially allowed any homeopathic product to be marketed if it was listed in the Homeopathic Pharmacopoeia of the United States and met basic requirements on labeling and the conditions it could treat. The petitioners argue the FDA violated the Administrative Procedure Act when it withdrew the old framework.

• VRBPAC backs Southern Hemisphere flu strains: 💊 The FDA’s Vaccines and Related Biological Products Advisory Committee voted 9-0 on October 1 in favor of the proposed composition of egg-based trivalent flu vaccines for the 2027 Southern Hemisphere season. The recommendation covers two influenza A strains (H1N1 and H3N2) and one influenza B/Victoria lineage strain. The lineages match the FDA’s recommendations for the 2026-2027 U.S. season. Watch the discussion here.

• Final guidance on biosimilar manufacturing changes: 💊 The FDA finalized its guidance on postapproval manufacturing changes for biosimilar products, revising a draft issued in July 2024. According to an FDA email bulletin, the final version aligns with the International Council for Harmonisation’s Q5E guidance, clarifies how applicants can use data from their original BLA to support a manufacturing change, and revises what information is needed to support a dosage form or strength not previously licensed. The FDA also reissued its biosimilar development Q&A document, withdrawing one question now covered by the new guidance. The guidance fulfills a commitment from the 2022 BsUFA reauthorization.

• ANDA prioritization program turns one: 💊 October 3 marked one year since the FDA launched its program to prioritize review of abbreviated new drug applications that meet certain domestic testing and manufacturing criteria. “By prioritizing and expediting these ANDA reviews, this program rewards investment in U.S. drug manufacturing, research, and development and strengthens the domestic pharmaceutical supply chain,” the FDA wrote in a LinkedIn post. The program is one of several FDA efforts to encourage domestic drug manufacturing.

• CBER updates its PDUFA payment procedures: 💊 CBER updated its standard operating procedures and policies document for processing Prescription Drug User Fee Act application payments. According to the change log, the update reflects user fees now being handled as a shared service. Payments now route through the Office of User Fees under the Division of User Fee Revenue Management, in line with the Simple Reform changes, instead of the Office of Regulatory Operations. CBER made similar changes earlier in October to an SOPP describing how it processes BLAs and NDAs.

Read more

Welcome to Insider issue #53.

Also, hello to the 310 life science professionals who joined us last month. If you haven’t already upgraded to a paid subscription to unlock our in-depth analysis issues like this one, you can do so here. Read our past deep dives here.

This month, Leon Chagal opens with FDA's August 2026 draft guidance on container closure systems, which would trade the existing predefined testing exercise for a product-specific risk assessment that runs from development through every postapproval change. He covers the five risk factors, the 1.5 mcg/day safety concern threshold, and why a container that worked for one drug doesn't automatically qualify for the next.

Kittu Rao reads out a warning letter from the dialysis chair. Lots went out with endotoxin results above specification, the paperwork said they passed, and the only endotoxin instrument had never been calibrated. Kittu takes care of dialysis patients, and his recommendations come straight from the clinic.

Jonathan Wacks closes with a private-label eye drop maker that, according to the warning letter, kept releasing sterile product after three failed media fills and drew a second warning letter for problems the FDA first cited in 2023. The firm suspended production and recalled more than 3 million bottles. Jonathan explains what a failed media fill should set in motion, and why every ophthalmic manufacturer (and other firms, too) should read this letter.

Download our slide decks to use any way you’d like:

Read more

These RA/QA news rolls are available in full only to paid subscribers. Upgrade here. If you haven’t already taken our reader survey, it should only take 1 minute here.

This is our biweekly recap of news and industry happenings curated for our paid subscribers. Our goal is to bring you the headline news and the stories, research, and insights that might be harder to find.

If you haven’t upgraded to a paid subscription yet, you can do so here. Also, make sure to follow us on LinkedIn and catch the latest episodes of our podcast, The Life Science Rundown.

Here are some key items from the past couple of weeks.

💊 = Pharma, biologic, and biotech.
💉 = Medical device, medtech, diagnostics, and digital health.

Before we jump in, make sure to catch our recent conversation with former two-time FDA Commissioner Dr. Robert Califf, who shares his perspectives on what it will take to see population-level benefit from GLP-1s:

FDA Leadership and Administration

• Overton faces the HELP Committee: Heidi Overton, the president’s nominee for FDA commissioner, appeared before the Senate Health, Education, Labor and Pensions Committee on September 24 for her confirmation hearing. Senators questioned her mainly about vaccine safety, mifepristone availability, FDA decisions on vapes, and food safety oversight, and some of the toughest questions came from Republican members. In her opening remarks, Overton said that if confirmed, she would “work with the dedicated career professionals to make sure every aspect of clinical trials and drug development is optimized.” She also said she would take the recommendations of career staff on product determinations, and she called advisory committee meetings a “critical part” of the FDA’s processes. “I think they are extremely valuable from everything that I know about the FDA,” she said.

• FDA plans a new associate commissioner role: The FDA announced its intent to hire an associate commissioner for regulatory policy coordination, a position that appears to be new to the agency. The person would report to Grace Graham, the FDA’s deputy commissioner for policy, legislation, and international affairs. The role would be heavily involved in drafting new regulations and policies and moving them through the agency’s internal approval process.

• HR shortages slow FDA hiring: STAT News reported that the FDA’s efforts to hire new staff are being slowed by a shortage of HR personnel to process applications. As a result, would-be hires are stuck in what the report called “pre-onboarding limbo.” The FDA’s HR staff now works under a “shared services” model, which has created further problems affecting all product centers. The FDA’s workforce has shrunk, and the agency is now trying to staff up to meet urgent needs.

• Acting commissioner invited to the next MAHA Summit: Acting FDA Commissioner Kyle Diamantas has been invited to discuss FDA modernization with White House policy advisor Calley Means at an upcoming Make America Healthy Again Summit, which was announced in mid-September. The summit will also include a session on psychedelic medicine with HHS Deputy General Counsel Matt Zorn and the CEOs of three biotech companies. One of those companies, Compass Pathways, received a Commissioner’s National Priority Voucher in April.

• Kennedy says he remains committed to changing vaccine policy: 💊 HHS Secretary Robert F. Kennedy Jr. told Children’s Health Defense, the advocacy group he founded and previously led, on September 17 that he remains committed to changing how the federal government reviews and recommends vaccines. “I intend to stay in this fight as long as I am able,” Kennedy said in his keynote address. He also asked for patience as he works through administrative procedures and legal requirements, saying, “In government, a bunch of things have to happen before something else happens or you get sued.” Kennedy referenced recent executive orders on vaccines and called President Trump an ally of the organization.

• Trump calls for splitting childhood vaccine doses: 💊 President Trump said at the White House on September 18 that he will be “demanding” vaccine makers sequence their products into more shots with smaller doses, a change that could require developers to submit new formulations to the FDA for approval. Trump said the administration would recommend five doses at 20% strength, given at six-month intervals, and would split up some vaccines. He also said the administration would remove “certain vaccines that people are a little worried about” from recommended schedules. Trump said he believes these changes will result in a “massive reduction in autism.”

• HHS seeks information on electromagnetic field exposure: 💉 HHS issued a request for information on September 18 on the human health effects of electromagnetic field exposure, including radiofrequency radiation and radiation from wireless sources. HHS said the responses will help it assess the current scientific evidence, compare domestic and international safety standards, identify research gaps, and inform recommendations for policymakers, schools, and families. The RFI appears to focus mostly on consumer goods. Two citizen petitions filed with the FDA in June and August, however, raised safety issues for magnetic resonance imaging products.

• HHS will forecast its PPE purchases: 💉 HHS announced a new system to forecast its demand for personal protective equipment. The project, led by the Office of the Assistant Secretary for Financial Resources, will be “the first official, recurring process to identify, consolidate, and publish anticipated HHS purchases of personal protective equipment.” HHS said the goal is to give American PPE manufacturers “a clearer and more predictable signal of future HHS demand.” The first forecast is due October 15, with quarterly updates after that.

• Executive order creates America.gov: President Trump signed an executive order on September 29 directing the creation of America.gov, a single website where the public can access government services and information. The order describes the site as a secure point of entry where people can sign in, ask questions in plain language, get answers, and, where possible, complete government transactions without visiting multiple agency websites. The FDA runs many consumer-facing forms, including adverse event reporting, and it is not yet clear how the agency will apply the order to them. The Office of Management and Budget is expected to issue implementation guidance to agencies within 90 days.

FDA General

• IND pilot could lead to an Australia-style trial system: 💊 The FDA has asked Congress to create a “clinical trial notification” system modeled on Australia’s risk-based framework, and agency leaders said on September 15 that the new expedited IND pilot could serve as a bridge to it. Speaking at an Endpoints News event, Deputy Commissioner Grace Graham said the pilot, which will start with eight to 10 pairs of drug sponsors and research institutions, would give the FDA “a better sense of what to look for” in third parties if Congress grants that authority. CBER Director Karim Mikhail said Australia’s system depends on its human research ethics committees, which oversee some low-risk trials directly. If qualified research institutions can speed research with fewer clinical holds, Mikhail said, “then we know we have a case that hopefully we can evolve to a CTN one day.”

• Graham outlines PDUFA VIII priorities: 💊 The 54-page draft commitment letter for the next Prescription Drug User Fee Act was the focus of a September 16 public meeting, where FDA Deputy Commissioner Grace Graham said the recommendations center on America First, manufacturing, and rare disease. Graham pointed to a proposed cut in application fees for companies “anchoring” Phase 1 trials in the U.S. She said the negotiations gave the agency “a great window” into how it can improve its processes so the chemistry, manufacturing, and controls portion of applications is not “a lagging indicator.” She also highlighted plans to fold several pilot programs, including the Rare Disease Endpoint Advancement program, into the regular meeting framework, and said the FDA will deliver the final recommendations to Congress by the January 2027 deadline.

• FDA pushes back on claims of rising first-cycle rejections: 💊 CDER issued a report on September 16 with a long-term analysis of first-cycle complete response rates for new drug applications and biologics license applications. The FDA said outside analyses suggesting an increase in complete responses rely on public data that may not capture all applications, so it used its internal Document Archiving Reporting and Regulatory Tracking System instead. The report found that first-cycle complete response rates fell from 47% for the fiscal 2008 receipt cohort to about 27% for fiscal 2024, averaging 33.7% across the period. It described a rise from fiscal 2018 to 2020 as a “transient uptick” and closed with a list of agency activities, such as guidance and webinars, that “may reduce the likelihood of complete responses.”

• Draft strategy on interchangeable biosimilars: 💊 The FDA announced on September 17 that it published a draft strategy document based on a September 2025 public hearing on developing interchangeable biosimilar products. The document outlines “specific actions FDA will take to facilitate interchangeable biosimilar product development.” It is meant to support a final strategy document that will guide FDA actions under the next Biosimilar User Fee Act.

• BsUFA commitment letter released: 💊 The FDA released the draft commitment letter for the next Biosimilar User Fee Act on September 24 and scheduled an October 26 public meeting to discuss it. The next BsUFA program would take effect October 1, 2027, if Congress reauthorizes it. Proposed changes include new supplement categories with four- and six-month review timelines, a provisional approval process similar to the one for generic drugs, and a review extension that would let the FDA address “resolvable issues” late in review that might otherwise lead to a complete response. The letter would also fold the Biosimilar Initial Advisory Meeting into Type 2b meetings, add a “risk-based lifecycle approach” to manufacturing facility deficiencies, create new meetings to resolve facility-related approvability issues, and sunset the BsUFA Regulatory Science Pilot Program.

• Gene therapy potency guidance under White House review: 💊 The White House Office of Information and Regulatory Affairs is reviewing an FDA guidance on potency assurance for cell and gene therapy products, according to an administrative record. The document appears to be a final version of draft guidance the FDA issued in December 2023, which drew significant feedback and some concerns from industry. CBER had listed it on its guidance agenda for this year. Its release would follow a separate draft guidance on potency assessments for active immunotherapy products.

• Final guidance on buffy coat blood collection systems: 💊💉 The FDA finalized a guidance document on September 16 for developers of blood collection, processing, and storage systems used to manufacture blood components for transfusion. The guidance applies to systems that use the “buffy coat method,” which separates collected blood by spinning it. It details the information the FDA will ask sponsors for to support a regulatory filing. It also encourages companies moving away from DEHP-based collection systems, a transition the FDA supports, to contact the agency for help.

• FDA replaces animal testing references in its regulations: 💊 The FDA issued a direct-to-final rule that replaces references to animal testing with the term “nonclinical tests” in nearly 40 places in its regulations. The rule carries out changes Congress made in late 2022, which defined nonclinical tests to include in vitro, in silico, in chemico, and nonhuman in vivo methods. The changes take effect February 4, and the FDA also published a companion proposed rule, with comments due December 7, in case it receives a significant adverse comment. Alongside the rule, the FDA launched a database of 25 case examples of review documents involving new approach methodologies, and Acting Commissioner Kyle Diamantas said the goal “is not to replace one rigid approach with another.” HHS described the rule as part of a wider set of actions that includes new NIH funding opportunities and smaller animal labs at NIH and CDC.

Read more

Ask a cardiologist what success looks like, and you’ll most likely hear about the patients in front of them. Ask a public health official, and the answer will cover a population that includes people who never make it to a clinic.

Dr. Robert Califf has spent his career in both roles, and he thinks GLP-1 therapies are one of the clearest examples of how those two definitions of success can diverge.

We’re genuinely honored to share this conversation. Dr. Califf has served as Commissioner of the FDA twice, has led some of the most important clinical trials in cardiovascular medicine, and founded the Duke Clinical Research Institute. Having him on The Life Science Rundown is a highlight for us, and we’re grateful he gave us his time and such a thoughtful conversation.

The question we put to him was this: how can society turn the scientific promise of GLP-1 therapies into a true public health success, with affordability, equitable access, trustworthy information, and responsible regulation?

Dr. Califf is a cardiologist by training who grew up in South Carolina and has been associated with Duke University for more than half of its 100-year history. His interest in learning which treatments actually work drew him into clinical trials early, which led to the Duke Clinical Research Institute and, over time, to working closely with FDA. He was appointed Commissioner in 2016 and returned to the role in 2022. Between those terms he worked with Alphabet’s health businesses, Verily and Google Health.

Apple Podcasts | Spotify | YouTube | Web + Others

Dr. Califf's key insights and practical takeaways

If you’re short on time, here are the most important lessons from the discussion.

Individual care and public health answer to different people. In a doctor-patient relationship, Dr. Califf’s responsibility was to the individual patient. Public health aims to improve longevity and healthy longevity across the entire population, including all its segments. GLP-1s, in his view, are a good example of where those responsibilities can come into tension and need to be resolved.

Obesity was treated as a moral failing, and that never worked. He described a long period in which people gaining weight felt admonished for it, as if it were a personal shortcoming. The pharmacologic side didn’t go much better. Before GLP-1s, most drugs tried for obesity had serious toxicities, some where the harm clearly outweighed the benefit, which is why he spent part of his academic career pushing for outcome studies. Meanwhile, a great deal of money was made selling diet books and products whose net effect, as he put it, was almost nothing.

The biology may teach us far more than weight loss. When he refers to GLP-1s, he means a class of drugs acting on multiple pathways between the gut and the brain that researchers are only beginning to understand. What excites him most isn’t only that they work. It’s what the science is revealing about biology and behavior. Preliminary data and secondary analyses suggest effects on cravings for alcohol, tobacco, gambling, and ultra-processed food, and definitive trials are underway. He was careful to note that neuroscientists still debate whether all of this fits the classical definition of addiction.

The opportunity is to reverse a chronic disease trend. He acknowledged the irony plainly: a food environment contributed to widespread weight gain, and now an expensive class of drugs is helping reverse it. But given where things stand, he sees a real chance. If these drugs can be distributed equitably, and the class keeps improving, it’s possible to reverse much of the current chronic disease burden. He pointed to Global Burden of Disease research identifying nutrition and weight as the largest drivers of the flattening of improvements in life expectancy, particularly in the United States.

Early access followed resources rather than need. Dr. Califf borrowed a line from science writer Ed Yong about technological fixes drifting toward society’s penthouses while disease seeps into its cracks. In the early phase, GLP-1s went largely to people with money or the education to obtain them, while those who needed them most had little access. He considers that an ongoing problem.

The people most at risk are the ones with the least room to maneuver. Public awareness has grown that weight gain reflects the food environment rather than personal failure, and he expects that alone to slow the trend. GLP-1s could get there faster. The risk of falling short lands mostly on people without good insurance, without spare cash to buy direct to consumer, and without the influence to get a foot in the door, while high costs lead some insurers and employers to leave these drugs out of their plans.

Innovation needs rewarding, and broad impact needs a different path. Dr. Califf was explicit that he isn’t in the camp that thinks drugs cost nothing to make. Trials are expensive; nine out of ten drugs that enter Phase 1 never reach the market, and companies need incentives to take those risks. His concern is that the system now rewards charging a high price to a narrow slice of the population, partly because patent windows are short and clinical adoption takes time. For the occasional drug with outsized public health impact, he named Gleevec in cancer and GLP-1s as examples, he believes society would be better served if far more people used it at a lower price. He suggested an early way of identifying those drugs, and believes companies could still be profitable under such a model. He also noted that U.S. generic drug prices are actually lower than in the rest of the world.

Access is more than the prescription. Adhering to a GLP-1 can mean working through nausea and other side effects that often pass if you stay with it. The more support someone has, whether money, friends, or stable circumstances, the easier that is. Equitable access includes that support.

Compounding was legal during the shortage. It isn’t now. When demand outstripped what the two manufacturers could produce, the law allowed compounding, and it initially happened through relatively controlled channels. Now that manufacturing capacity has caught up, compounding look-alikes of approved GLP-1 drugs on the market is no longer permitted. People buying compounded versions today are getting products without the testing and quality control of approved medications, a risk he described as unquantified but definitely not zero. Much of the starting material also comes from different sources than those used by the approved manufacturers.

His larger worry is untested peptides. He sees a spillover from that period into something he considers genuinely dangerous: novel peptides sold online, including new GLP-1-type compounds that are essentially new drugs and haven’t been through even basic evaluation, such as whether they cause cancer in animals. People are buying them at high cost based on hearsay and online influencers. He also said he understands why people look elsewhere when their employer's plan won’t cover a drug they need.

Trust follows trustworthy behavior. Asked about misinformation, Dr. Califf reframed the question. Before the internet, health care largely served the people who showed up. Now we can see everyone, all 340 million people in the U.S. and the full global population, along with every disparity, which makes clear these problems are solvable. Surveys show people distrust large institutions while trusting their own doctors, nurses, and pharmacists. His view is that every sector is full of good people working inside a system that isn’t configured well, and that no messaging fixes a mismatch between need and access. He cited a line he likes: if you want to be trusted, act trustworthy.

Technology can help put care where the need is. He described an ARPA-H program he’s helping with called ADVOCATE, which is developing AI agents to support cardiovascular care. In parts of rural America, reaching a heart failure specialist can mean a three-hour drive, and the gap between rural and urban health outcomes is widening. Alongside technology, he’d like to see a system that distributes care to where the need is.

What success over the next decade would require. His first priority is reform of the U.S. insurance system, and he noted that even high-income countries with universal coverage aren’t distributing GLP-1s widely because of cost. Beyond that, he’d like to see the pharmaceutical model evolve, with better definition of who actually benefits from a drug or device, wider use among those people, and less use among those who don’t. Electronic health records and modern computational tools make that more achievable than ever. In his words, we don’t need to worship AI, but it can help with large problems like distribution, supply chains, and deciding who would benefit.

And underneath all of it, a sense of wonder. Dr. Califf asked that his concerns not be read as negativity. He called the GLP-1 story something like a miracle: a line of research tracing back to a lizard and years of basic science at NIH, first pursued for diabetes, then revealing far broader effects. We’re only scratching the surface of what it will teach us about behavior and biology. He sees the gaps as an opportunity for more people to live healthy lives.

Robert M. Califf, MD, MACC, served twice as Commissioner of Food and Drugs: as the agency’s 22nd Commissioner from February 2016 to January 2017, and as its 25th Commissioner from February 2022 to January 2025. Before his first term, he served as FDA’s Deputy Commissioner for Medical Products and Tobacco. Between his terms, he was head of medical strategy and senior advisor at Alphabet, contributing to strategy and policy for Verily Life Sciences and Google Health. A cardiologist, Dr. Califf spent decades at Duke University, where he was a professor of medicine, vice chancellor for clinical and translational research, director of the Duke Translational Medicine Institute, and founding director of the Duke Clinical Research Institute. While at Duke he led major initiatives to improve clinical research methods and infrastructure, including the Clinical Trials Transformation Initiative, a public-private partnership co-founded by FDA and Duke. He earned his medical degree from Duke University School of Medicine, completed his internal medicine residency at the University of California, San Francisco, and completed his cardiology fellowship at Duke.

Who is The FDA Group?

The FDA Group helps life science organizations rapidly access the industry's best consultants, contractors, and candidates. Our resources assist in every stage of the product lifecycle, from clinical development to commercialization, with a focus on staff augmentation, auditing, remediation, QMS, and other specialized project work in Quality Assurance, Regulatory Affairs, and Clinical Operations.

With thousands of resources worldwide, hundreds of whom are former FDA, we meet your precise resourcing needs through a fast, convenient talent selection process supported by a Total Quality Guarantee.

Here’s why 17 of the top 20 life science firms access their consulting and contractor talent through us:

• Resources in 75 countries and 48 states.

• 26 hours average time to present a consultant or candidate.

• Exclusive life science focus and expertise.

• Dedicated account management team.

• Right resource, first time (95% success).

• 97% client satisfaction rating.

Talk to us when you're ready for a better talent resourcing experience and the peace of mind that comes with a partner whose commitment to quality and integrity reflects your own.

Subscribe to The Life Science Rundown:

Apple | Spotify | YouTube | Web + Others

Check out our newly launched AI-powered QMS audit tool, AICA (the Audit Intelligence Compliance Assistant).

Dr. Califf joins Nick Capman to talk about how GLP-1 therapies could become a true public health success, and what it will take to get there: affordability, equitable access, trustworthy information, and responsible regulation. He brings the perspective of a cardiologist, clinical trialist, and two-time FDA leader, and he closes on a note of genuine optimism about what this class of drugs may teach us.

Some of what he covers:

• The difference between the goal of individual medical care and the goal of public health

• Why obesity was long treated as a moral failing, and why earlier drugs fell short

• What GLP-1 research may reveal about cravings and behavior more broadly

• Why early access followed resources rather than need

• How compounding was permitted during the shortage, and why it no longer is for approved drugs

• His concern about untested peptides sold online

• Why trust follows trustworthy behavior

• What success could look like over the next decade

Our sincere thanks to Dr. Califf for his time and insight.

About Dr. Robert Califf
Robert M. Califf, MD, MACC, served as Commissioner of Food and Drugs from 2016 to 2017 and again from 2022 to 2025, and previously as FDA's Deputy Commissioner for Medical Products and Tobacco. Between his terms, he was head of medical strategy and senior advisor at Alphabet for Verily and Google Health. A cardiologist, he spent decades at Duke University, where he was founding director of the Duke Clinical Research Institute, and he is one of the most frequently cited authors in biomedical science.

About The FDA Group
The FDA Group helps life science organizations rapidly access the industry's best consultants, contractors, and candidates. Our resources assist in every stage of the product lifecycle, from clinical development to commercialization, with a focus on staff augmentation, auditing, remediation, QMS, and other specialized project work in Quality Assurance, Regulatory Affairs, and Clinical Operations. Learn more: https://www.thefdagroup.com/

At RAPS Convergence 2026 in Charlotte, Jennifer Albert, a regulatory project manager and regulatory scientific reviewer at FDA, shared her advice for sponsors preparing BLAs for CBER’s Office of Therapeutic Products (OTP).

RAPS covered the session, and the advice is worth reading in full.

None of it should strike anyone as particularly surprising or novel. We’d characterize it more as the kind of guidance that’s easy to agree with and easy to underexecute. So for each tip below, we’ve summarized what Albert said as RAPS reported it, then added some additional practical advice from our own BLA support work.

1. Run everything through the RPM

Jennifer noted that the regulatory project manager is the applicant’s single point of contact for everything related to the BLA, including clinical and regulatory questions, and coordinates the submission across all six OTP offices.

She added that applicants should always use secure FDA email, put the BLA number in every amendment subject line, and clearly mark urgent communications. Individual reviewers should not be contacted during a review. And as a courtesy, applicants should give the RPM a heads-up before submitting an amendment.

We’d take this advice a step further by setting up secure email well before you need it. CBER sends regulatory communications to sponsors by secure email, and its policy requests to establish it go to SecureEmail@fda.hhs.gov. That setup might take time, and you need a non-ISP email domain to do it. Don’t let your first urgent information request get delayed because secure email hasn’t been established yet!

2. Take the end-of-phase 2 and pre-BLA meetings, and build against the official record

Jennifer encouraged sponsors to request end-of-phase 2 and pre-BLA meetings even when they aren't required, saying there's no disadvantage to having them.

End-of-phase 2 is the chance to confirm that the FDA agrees with your pivotal trial design before committing to phase 3. OTP grants one pre-BLA meeting per product or indication, and that meeting covers content and format, topline data, outstanding issues, and labeling concepts. Issues raised there are expected to be addressed in the BLA. She described it as a strategic checkpoint instead of a formality.

We’d add that timing matters a lot here. Hold the pre-BLA meeting when you have enough data and a settled enough application structure for FDA’s feedback to be concrete, and early enough that you have time to act on what you hear. OTP currently recommends that the pre-BLA meeting occur at least two months before the planned BLA submission, which generally means submitting the meeting request at least four months in advance.

When the FDA’s meeting summary arrives, treat it as the official record and turn it into a tracker you can use. List each agreement, recommendation, action item, or unresolved issue, who owns the response, and where in the BLA it’s addressed .That tracker becomes your internal pre-submission check, and it can also feed your reviewer's guide.

3. If you qualify for rolling review, wait for the grant letter

Jennifer noted that products with RMAT, breakthrough therapy, or fast track designation can qualify for rolling review, which lets applicants submit completed BLA modules on a rolling basis.

Rolling review comes up during the pre-BLA meeting, and the formal request can go through the associated IND. She cautioned though not to submit BLA modules until you’ve received a grant letter.

Rolling review doesn't mean sending partially finished modules. The modules you submit early should be complete and stable, even though other parts of the BLA may still be in development. We encourage teams to build a realistic module-submission schedule, discuss it at the pre-BLA meeting, submit the formal request through the IND, and not start the rolling BLA until FDA grants the request.

4. Know exactly what belongs in Module 3

Jennifer noted that the FDA doesn’t allow a BLA to reference CMC information from a Type II drug master file. It does permit some information from Type III and Type IV master files, such as packaging materials, container closures, excipients, and raw materials used in preparing the drug substance, drug substance intermediate, or drug product.

Read more

When we pull lessons from our audit work, they usually come from the observations and recommendations that make it into the report. An audit we ran earlier this year gave us almost none of those, but it’s still worth passing along since these clean results provide lessons just the same.

It was a periodic requalification audit of a contract testing lab, conducted on behalf of a sponsor organization.

The lab is one site within a larger multi-site organization. Our audit ran in two parts: two days reviewing the organization’s global quality systems remotely, followed by one day on-site at the facility. We assessed compliance with 21 CFR Parts 11, 58, and 820, as well as internal policies and procedures.

The result was zero critical, major, or minor observations and a single recommendation, and the lab was confirmed as an approved vendor for all services it provides to the client. That’s a good outcome! The report is also a useful picture of what a well-run vendor program looks like, and of where even a mature one has room to tighten.

Learn more and get in touch if you need audit support now or in the future. We’re getting into 2027 audit planning season right now. Get our free audit planning Excel workbook here.

1. Let a centralized review carry the shared material

The vendor’s corporate quality assurance function operates as we see some of the best firms organize themselves: a centralized audit program covering procedures common across all its sites.

That central program covered SOP harmonization, data integrity, CSV and IT security, training, vendor management, archiving and data retention, QA unit operations, and protocol and reporting standards. The program is built to complement individual site audits, not replace them. It front-loads the shared topics so site visits can concentrate on compliance verification and site-specific issues.

Our auditor found no observations in that review and described it as a very efficient way to cover the vendor’s quality systems in an organized manner.

2. Know which SOPs are global and which are local

The vendor uses a smart three-tier SOP structure:

1. Fully harmonized SOPs that permit no site variation.

2. Aligned SOPs that allow limited site-specific adjustments.

3. Site-specific SOPs for unique functions.

Our report’s summary of the centralized review listed justified site-specific variations and legacy system transitions at some locations among its areas for ongoing attention. It also suggested requesting validation documentation for site-specific systems as needed.

3. “Within our procedure” isn’t the whole answer

Our audit’s one formal recommendation concerned SOP revision. Some procedures hadn’t been revised in more than five years. That was within the allowable interval under the vendor’s own procedures, but we still recommended establishing a stricter revision schedule unless the current practice could be fully justified without any lingering risks.

Under the sponsor’s audit program, that finding was classified as a recommendation rather than an observation.

Read more

Most Quality teams are staffed for their “steady state.” The trouble comes from everything else that inevitably happens: an inspection date on the calendar, a backlog that quietly grew faster than anyone could close it, a remediation window after an FDA visit, a QMS in need of help without a specialist, a key role sitting empty while new candidates are sent through the modern hiring gauntlet.

Those situations need someone on contract to work within the team, often full-time, for a defined period. Project-based consulting doesn’t quite fit because the work isn’t a discrete deliverable. It’s daily or otherwise regular execution alongside the people who are already there.

That’s the model we call staff augmentation. A full-time consultant, or FTC. The consultant works as part of the client’s Quality organization, within their systems, reporting to their leaders, for as long as needed, under a flexible contract that can be extended if needed.

We’re seeing more Quality needs naturally lend themselves to this model, and it can be used to address a variety of needs. Here are four recent engagements that show what that looks like in practice in case it’s a strategic solution for you — now or later.

1. Clearing an investigation backlog while building the bench

We’ve been working with a pharma manufacturing site that had built up a significant backlog of open and overdue deviation investigations. At the same time, it was starting to implement an investigator qualification program to strengthen its own people.

We dropped in two full-time consultants who have embedded themselves in many similar organizations before for very similar work, primarily on-site, for roughly three to six months. The job was to write, manage, and drive manufacturing investigations through to closure, covering equipment failures, human error, and procedural noncompliance, with root cause analysis that would hold up.

For the second half of the scope, our experts were asked to coach and train site personnel on root cause investigation as the qualification program came online. And the client specified prior cGMP inspection experience, so the people closing investigations would recognize the weaknesses an investigator looks for. This “get the work done, then coach on doing that work” model is a motif that extends to all kinds of Quality work through a staff augmentation model.

It fits here so well because a backlog is volume work that has to happen inside the site’s own systems and with its own people. Pairing execution with coaching was designed to pay down the backlog while building the site’s capability to keep up with investigations on its own.

It’s an excellent use case for FTCs.

2. Leading a medical device site through FDA inspection preparation

A medical device manufacturer we’ve worked with on several projects had an FDA inspection scheduled at a site outside the United States, with a team that was new to FDA inspections. A mock audit was already on the calendar.

They needed an experienced QA leader to work 30 to 40 hours a week for at least three months, traveling to the site regularly. The role would attend the mock audit, then lead the roughly four weeks of remediation, documentation, and storyboarding that followed. During the inspection itself, the consultant would serve as the primary contact, coordinating responses and representing the company.

An interesting part of this engagement was that the company preferred industry-side inspection experience over prior FDA investigator experience for this role to avoid conflicting perspectives. The job mixed hands-on work with coaching, and company leadership wanted a steady physical presence during the critical weeks to build confidence with site leadership.

The FTC model works well here, too, because this kind of inspection preparation has a fixed end date and many judgment calls along the way. The team needed one accountable person fully allocated to handle planning and actual execution, not a series of advisory calls.

3. Putting validation engineers on the floor after FDA observations

We commonly augment internal teams in response to regulatory events, too. In one recent example, a high-volume manufacturing site running continuous production had received FDA inspectional observations via a 483. They hadn’t affected production, and the team wanted to keep it that way by getting ahead of the process fixes.

We deployed a small team of on-site validation quality engineers with specific process validation experience in high-speed, high-volume manufacturing, starting right after a planned plant shutdown, on first shift, for at least six months.

Company leaders also raised a related need for on-site quality systems support covering CAPA and management controls.

Bringing in contracted specialists worked here since validation work at a running plant is extremely hands-on and has to happen where the equipment is. The company needed experienced engineers on the floor for a sustained stretch, timed to the plant’s own schedule.

4. Bridging to a permanent hire during a busy inspection season

FTCs are useful if you’re a clinical-stage company, too. We recently worked with a clinical-stage sponsor whose Clinical QA Director was heading into a period with regulatory inspections on the calendar in more than one region, alongside pharmacovigilance oversight, CRO oversight, and ongoing internal audit follow-up. The company planned to hire a permanent QA manager but needed coverage right away. (FTCs are great when you don’t have time to wait for an FTE to get hired and onboarded — or if the work for that role won’t be permanent and ongoing.)

We embedded a hands-on QA manager-level professional to act as the director's right hand:

• Fielding daily questions from clinical teams.

• Leading responses to internal audit findings and CAPAs in the company's quality management system

• Representing QA in meetings

• Signing off on documents so the director could concentrate on inspection preparation.

The consultant would work closely with the director until the permanent hire was onboarded.

This is the clearest version of a bridge. The permanent role was coming, but the inspection calendar wasn't going to wait for recruiting to finish. A full-time consultant covered the gap while recruiting ran its course.

If this sounds familiar…

If your Quality team is looking at an inspection date, a backlog, a remediation effort, or an open role it can’t fill fast enough, this is the kind of support we provide. Tell us what the next one to six months look like, and we’ll tell you whether a full-time consultant is the right answer. Learn more and get in touch.

Watch our great conversation with Mark Shapiro on clinical outsourcing vs. insourcing for more on this topic:

Who is The FDA Group?

The FDA Group helps life science organizations rapidly access the industry's best consultants, contractors, and candidates. Our resources assist in every stage of the product lifecycle, from clinical development to commercialization, with a focus on Quality Assurance, Regulatory Affairs, and Clinical Operations.

With thousands of resources worldwide, hundreds of whom are former FDA, we meet your precise resourcing needs through a fast, convenient talent selection process supported by a Total Quality Guarantee. Learn more and schedule a call with us to see if we’re a fit to help you access specialized professionals and execute your projects on time and on budget.

Share The FDA Group's Insider Newsletter

This guidance breakdown is available in full to paid subscribers. Only paid subscribers get regular full access to our guidance breakdowns and other analyses. If you’re not already a paid subscriber, you can upgrade here.

We have a breakdown for the device professionals among us. On September 18, 2026, CDRH and CBER issued a draft guidance titled Electronic Submission Template for Premarket Approval Applications (PMA).

If and when it’s finalized and implemented, it would require specified PMA submission types to be submitted electronically using FDA’s electronic submission template. eSTAR, the structured dynamic PDF, is currently the only template available for these submissions.

eSTAR has been mandatory for 510(k) submissions (unless exempted) since October 1, 2023, and for De Novo submissions, unless exempted, since October 1, 2025. PMAs can use it today on a voluntary basis. This draft sets up the requirement.

Comments are due within 60 days of the Federal Register notice announcing the draft. The docket number will appear in that notice.

Again, this is just a draft guidance right now.

Who and what this covers

The draft applies to five “Designated Application Types”: Original PMAs, Panel-Track Supplements, 180-day Supplements, Real-Time Supplements, and 30-Day Notices/135-Day Supplements. It does not cover PMA Modules or Modular Shells.

It also does not address the information needed for filing review, and FDA says it’s not meant to specify eSTAR’s user interface or detailed content. Its job is narrower: to establish the PMA electronic format and standards under section 745A(b)(3) of the FD&C Act.

Part of this will be binding

Most FDA guidance is nonbinding, and most of this one is, too.

But the draft is explicit that where it sets out standards, a timetable, or criteria for waivers and exemptions under section 745A(b), those provisions will have binding effect once final. The FDA signals which parts those are through mandatory words like must and required.

Section 745A(b)(3) gives FDA statutory authority to set these requirements through guidance, which is why the usual rule that guidance cannot create enforceable obligations does not apply to those provisions.

The practical consequence sits in section VI so read it carefully! Once the requirement takes effect, a Designated Application Type not submitted as an electronic submission “will not be considered received” unless an exemption applies.

The FDA anticipates technical screening instead of RTA review

This is the operational change we see as most likely to matter to a PMA team (and the consultants among us).

Because a properly prepared eSTAR should represent an administratively complete submission, the FDA says Original PMAs and Panel-Track Supplements (the two types currently subject to refuse-to-accept review) are not anticipated to go through RTA.

In its place, the FDA says it intends to run virus scanning and a technical screening. For Original and Panel-Track PMAs, the draft guidance notes that technical screening confirms that the content of the PMA Acceptance Checklist has been submitted.

Technical screening checks two things: that your eSTAR responses accurately describe the device (the guidance’s example is confirming there are in fact no tissue-contacting components if you indicated none), and that there is at least one relevant attachment for each applicable attachment-type question.

What the template asks for

Table 1 of the draft lays out the current PMA eSTAR structure, from submission type and device description through benefit-risk, labeling, the technical testing sections, and amendment responses.

A few details worth flagging:

The FDA also notes that eSTAR is structured to closely follow SMART, the Submission Memo And Review Template reviewers use internally for certain PMA submission types.

Read more

These RA/QA news rolls are available in full only to paid subscribers. Upgrade here. If you haven’t already taken our reader survey, it should only take 1 minute here.

This is our biweekly recap of news and industry happenings curated for our paid subscribers. Our goal is to bring you the headline news and the stories, research, and insights that might be harder to find.

If you haven’t upgraded to a paid subscription yet, you can do so here. Also, make sure to follow us on LinkedIn and catch the latest episodes of our podcast, The Life Science Rundown.

Here are some key items from the past couple of weeks.

💊 = Pharma, biologic, and biotech.
💉 = Medical device, medtech, diagnostics, and digital health.

FDA Leadership and Administration

• FDA names permanent center directors and a first-ever AI deputy: The FDA promoted three existing officials, making Mike Davis the permanent director of the Center for Drug Evaluation and Research, Karim Mikhail the permanent director of the Center for Biologics Evaluation and Research, and Jared Seehafer the agency’s first deputy commissioner for technology and artificial intelligence. Davis and Mikhail had both been serving in an acting capacity. Seehafer, most recently a senior advisor, will be the senior leader for technology, software, and AI and will set agency-wide strategy in those areas, though the full remit is expected to be detailed after a new commissioner is confirmed. All three have maintained lower public profiles than their predecessors, and all three have operational rather than purely academic backgrounds: Seehafer and Mikhail were company founders and CEOs before joining the agency, and Davis has extensive experience leading within CDER. Mikhail and Seehafer hold scientific master’s degrees rather than the doctorates most FDA medical leaders have traditionally held.

• Overton’s nomination formally transmitted to the Senate: The White House sent Heidi Overton’s nomination for FDA commissioner to the Senate on September 14, clearing the way for confirmation hearings to be scheduled. The hearings are likely to involve the Health, Education, Labor and Pensions Committee and possibly the Senate Finance Committee. Overton currently serves on the White House Domestic Policy Council, which makes it likely she was aware of and signed off on the recent center director promotions, since those officials would be among her closest colleagues if she leads the agency.

• CBER fills out its leadership roster: 💊 CBER updated its leadership page to show acting office directors and an acting deputy director, including John Scott as acting director of the Office of Biostatistics and Pharmacovigilance and Vincent Amatrudo as acting director of the Office of Compliance and Biologics Quality. Sudhakar Agnihothram is acting deputy director of the Office of Vaccines Research and Review, Megha Kaushal is acting deputy director of the Office of Therapeutic Products, Steven Oh is acting director of OTP’s Office of Cellular Therapy and Human Tissue, and Steven Fleischer is acting director of OTP’s Office of Pharmacology/Toxicology. A seat is still unfilled: Mikhail is still listed as acting director of OTP, the cell and gene therapy super office, following Vijay Kumar’s departure earlier this year. Several of the changes appear to predate the webpage update, with two of the named officials listing earlier start dates on their own profiles.

• New CBER director outlines how he weighs access against evidence: 💊 In his first public post since becoming permanent CBER director, Karim Mikhail signaled that he will weigh the cost of patients going without a therapy more heavily when deciding whether to approve it. Writing on LinkedIn, Mikhail said the scientific record tells you what a therapy does but cannot tell you what waiting for it costs, and described learning that from families whose disease has no approved treatment, for whom a review timeline is not a metric but time they may not have. He added that he wants frameworks fit for the science in front of the agency rather than the science behind it, with sponsor and regulator functioning as two disciplines accountable to the same person rather than two parties negotiating across a table.

• Davis signals a return to regular order at CDER: 💊 In an interview with BioCentury, new CDER Director Mike Davis described his intent to listen more to center review staff and convene more advisory committee meetings. The publication characterized the theme as a return to regular order, a notable contrast with the prior leadership’s approach to both staff input and advisory committees.

• FDA and SEC sign an information-sharing agreement: The FDA and the Securities and Exchange Commission signed a memorandum of understanding intended to provide a framework for enhanced cooperation in their regulatory and enforcement responsibilities. The MOU covers information-sharing protocols, including non-public information, with much of the document describing how each agency will safeguard what it receives. Publicly traded companies are typically required to disclose material information about their FDA interactions, including complete response letters, approvals, application filings and acceptances, and compliance actions. The agreement arrives as betting platforms drive increased interest in clinical trial readouts.

• HHS opens the EUA pathway for military operations: 💊 HHS issued an emergency use authorization declaration covering drugs identified and supported by the Department of Defense as addressing an unmet military operations-related medical need. EUA declarations typically respond to a defined public health emergency such as COVID-19, mpox, Zika, or Ebola, with specific indications attached. This one names no specific public health concern, instead opening the pathway to drugs for managing moderate to severe acute pain in casualties caused by biological, chemical, radiological, or nuclear agents, or by agents of military combat including firearms, projectiles, and explosive devices, where there is an imminently life-threatening and specific risk to U.S. forces. No product list or therapeutic areas have been identified. An existing MOU between the FDA and DOD already provides for enhanced collaboration while preserving the FDA’s authority over medical product authorizations.

FDA General

• Expedited IND pilot opens for applications: 💊 The FDA began accepting applications on September 15 for its pilot program to expedite Investigational New Drug applications, announcing the program’s final design at the same time. The agency expects to select between eight and 10 applications, submitted jointly by sponsors and research institutions planning to work together. Applications close October 30, with participants chosen by December 18. The FDA wants to test whether collaboration between sponsors and research institutions improves IND submission quality, and it plans to review submissions on a rolling basis through the program. Mikhail said on a press call that what the agency learns from the first cohort can inform the future of early-stage drug development in the U.S., while Davis noted that future approaches could include a formal accreditation model recognizing qualified research institutions. For the pilot itself, the FDA retains full regulatory authority and oversight.
  

• New draft guidance on hepatic impairment pharmacokinetics: 💊 The FDA issued a new draft guidance on how drug developers should assess pharmacokinetics in patients with impaired liver function. The question matters because many drugs are metabolized through the liver, and reduced hepatic function can cut efficacy or raise safety concerns. The document covers how sponsors should decide whether to run dedicated PK studies and, if so, how to design them and analyze the results. It appears to replace a 2023 guidance that the agency withdrew the same day.

• Prior knowledge guidance draws calls to broaden its scope: 💊 The comment period closed on the FDA’s June draft guidance describing how sponsors can leverage prior knowledge to develop human genome-editing products. Many stakeholders urged the agency to extend the recommendations beyond genome editing products, and several pushed for clarity on how the guidance connects to related concepts including the plausible mechanism framework and platform technology designation. The document could help sponsors repurpose existing manufacturing, nonclinical, and clinical data, reducing what they need to collect anew when testing novel products.

• Temporary compounding flexibilities for neonatal parenteral nutrition: 💊 The FDA published guidance effective immediately that allows compounders certain flexibilities to make starter parenteral nutrition drug products for neonates, citing potential serious supply disruptions. The flexibilities run 180 days, through March 8. The agency has issued similar stopgaps in recent years, though this one arrives amid heightened attention to this patient population.

• FDA lays out its thinking on psychedelic drug development: 💊 New CDER Director Mike Davis and several other agency officials wrote that the FDA is open to reviewing and approving psychedelic therapy for mental health disorders, though how such treatments fit existing regulatory frameworks is still evolving. The article, coauthored with Division of Psychiatry Director Tiffany Farchione and Division of Neuroscience Director Teresa Buracchio, expands on guidance the agency finalized in July addressing the functional unblinding problem that complicates research in this field. The authors wrote that the FDA is open to comparators other than inert placebos, including active comparators or low doses of the compound under study, but may still expect a placebo-controlled trial. They acknowledged that psychedelics can be dosed intermittently and described the agency as receptive to approval based on efficacy over a relatively short period, followed by longer-term assessment of the need for and safety of additional doses. They also noted the agency may require a Risk Evaluation and Mitigation Strategy.

• Sunscreen monograph finally loses two ingredients: 💊 The FDA amended its sunscreen ingredient monograph to remove aminobenzoic acid and trolamine salicylate, which it had previously determined were no longer considered safe or effective.

• Priority review voucher redemption fee set for fiscal 2027: 💊 The FDA set the fee for redeeming a priority review voucher in fiscal 2027 at $1,798,596. Unlike the Commissioner’s National Priority Voucher program, these vouchers are not free to redeem. Companies must pay both the standard filing fee and an additional fee to cover the extra staff work required to expedite a review. The amount is set by a formula estimating the cost of a priority review relative to a standard one.

• Botanical drug development draws a request for input: 💊 The FDA said it wants to accelerate development of botanical drug products, including algae, beyond the scope of its 2016 guidance, and posted a Federal Register notice on September 3 seeking stakeholder input. Botanical drugs, made by growing plants or fungi rather than through chemical synthesis, raise distinct regulatory questions around dose standardization, plant purity, and the effects of growth methods on safety, including pesticide use and heavy metal uptake from soil. The notice asks about development challenges and how the agency can help through guidance or other support. A workshop with the European Medicines Agency is in the works.

• Survey planned on pregnancy exposure registry enrollment: 💊 The FDA announced a need for data on pregnancy exposure registries and proposed a new survey of health care providers to identify perceived barriers to patient enrollment. The notice indicates the agency believes many registries fail to enroll adequately, and that it is weighing ways to encourage enrollment through providers based on what the survey finds.

• Oncology outcomes workshop scheduled for October: 💊 The Oncology Center of Excellence will hold its annual workshop on clinical outcome assessments in cancer trials on October 15. Two panels will examine whether researchers are measuring the outcomes that matter most to patients, including how therapies such as CAR-T create new assessment challenges and whether technologies such as AI-assisted monitoring can be designed to gather more meaningful data. An agenda and speaker list are still to come.

• Rare disease statistics workshop opens its docket: 💊💉 A Federal Register notice published September 15 opened a docket for the next event in the FDA’s Rare disease Innovation, Science, and Exploration workshop series, titled “RISE to the Challenge: Statistical Considerations for Rare Disease Clinical Investigations.” The hybrid event takes place September 29, with draft sessions covering randomization and control arms, navigating trade-offs, and improving efficiency, plus a case study on statistical considerations for brain-computer interface devices in amyotrophic lateral sclerosis. The agency developed two pre-read documents, one for drugs and biologics and one for devices, which had not yet been posted at the time of the notice. Comments run through November 13.

• Patient voice meeting planned for rare disease development: 💊💉 The FDA will host a meeting October 16 with the Reagan-Udall Foundation to explore the patient voice in rare disease medical product development across biologics, drugs, and devices. The session will also look at additional opportunities for patients to engage in the product development process.

• Compounding quality conference opens its call for proposals: 💊 The FDA opened a call for proposals for its Compounding Quality Center of Excellence Conference, which it will cohost with the International Society for Pharmaceutical Engineering. The conference arrives after a year in which the agency significantly stepped up oversight of certain compounding activities, particularly those involving GLP-1 and other weight loss drugs.

• Pharmacogenetic associations table gets its first update in years: 💊 The FDA updated its table of known pharmacogenetic associations, genetic markers that affect drug safety or efficacy, for the first time in nearly four years. The update adds more than 20 drug products, including gentamicin, bupropion, and dextromethorphan.

• Two final dental device guidances published: 💉 The FDA published final guidance documents covering premarket notification submissions for dental composite resin devices and dental curing lights. Both finalize 2024 drafts and replace final guidances from 2005 and 2006. The composite resin guidance updates the specification of radiant power output at the tip and removes resin depth-of-cure measurements.

• Eight device types moved to Class II: 💉 The FDA announced it is classifying eight device types as Class II after finding them moderate-risk despite being innovative, which lets them serve as predicates for future premarket notifications. The list covers whole-room microbial reduction devices, vaginal hydrogel packing systems, implanted tibial electrical urinary continence devices, cooperative powered surgical assist devices for ENT surgery, anti-tumor necrosis factor alpha monoclonal antibody test systems for inflammatory bowel disease, extravascular support for arteriovenous fistulas, and two esophageal protection devices for cardiac ablation procedures, one temperature-regulating and one mechanical.

• Cardiovascular machine learning software classified as Class II: 💉 The FDA classified cardiovascular machine learning-based notification software as Class II, allowing future submissions to use the 510(k) pathway. The first such device cleared by the agency was authorized in August 2023.

• Biocompatibility town hall covers the new ISO standard: 💉 The FDA’s first biocompatibility town hall on September 9 focused on updates to the international biocompatibility standard ISO 10993-1:2025 and the continued relevance of the agency’s guidance on using it. CDRH’s Shuliang Li said the major change in the sixth edition is realignment with ISO 14971, the risk management standard the FDA recognizes in full. The revisions refine device categorization to account for exposure duration including intermittent contact, and remove the externally communicating device category in favor of categorizing by tissue type and whether skin or mucosa is intact, breached, or compromised. The standard carries a three-year transition, so the agency will accept declarations to the 2018 version until July 1, 2029. Li added that the FDA is not raising its expectations for biological evaluations.

• Device lists for AI, VR, and sensors refreshed: 💉 The FDA released updated webpages listing approved or cleared devices that use artificial intelligence, augmented and virtual reality, and digital health technology sensors. The lists let developers identify predicate devices to reference and get a clearer picture of the agency’s evidentiary expectations.

• Emerging pathogen diagnostics guidance nears finalization: 💉 A guidance document addressing how sponsors can validate in vitro diagnostics intended to detect emerging pathogens during a declared public health emergency is advancing, according to a new White House notice. The guidance was released in draft form in January 2025. The agency’s aim appears to be better preparation for future emergencies by clarifying in advance what it expects from developers of tests for emerging pathogens.

• CDRH publishes a regulatory reliance resource: 💉 CDRH posted a new Regulatory Reliance Portal, an educational webpage describing how the center oversees and regulates medical devices, intended as a resource for foreign regulatory authorities. Reliance differs from harmonization: rather than converging on a single common framework, one regulator gives significant weight to another’s decisions. The page offers information on establishing the FDA as a comparable regulator whose assessments other authorities can consider.

• New regulatory science tool for respirator materials: 💉 CDRH published a new regulatory science tool for developing personal protective equipment materials, a Respirator Filtration Efficiency Calculator. The calculator helps developers assess respirator and mask textile materials by estimating particle filtration efficiencies against a reference, a surgical N95 respirator. Regulatory science tools do not carry the weight of qualified Medical Device Development Tools, but they give developers a way to demonstrate performance where standards and MDDTs do not yet exist.

Read more

Most preclinical companies, Baseimmune’s CEO Kevin Walton argues, are building their evidence backward.

Cheap assays first, a run of supportive results, the expensive question deferred until the package looks respectable enough to survive it. Eighteen months later, the money is gone and the central hypothesis still hasn’t actually been tested.

Kevin says that’s the wrong order. He spent years in investment banking watching companies do exactly this, then took over a platform-stage biotech and made the opposite call.

Nick Capman sat down with him to work through the obstacles facing a lot of small biotechs right now:

• Turning science into something investable.

• Building culture with a lean team.

• Steering through the pivots that early-stage companies rarely avoid.

Kevin is the CEO and board director at Baseimmune, a London-based company developing active immunotherapies for fibrosis, with idiopathic pulmonary fibrosis as its lead indication. He joined as Chief Business Officer and stepped into the CEO role, where his first move was a strategic pivot. Before Baseimmune he was Senior Director of Corporate Development at Moderna, VP of Corporate Development at StrideBio, Senior Director of Business Development at Curis, and held corporate development, strategy, and chief of staff roles at G1 Therapeutics. He started in investment banking and consulting and holds a law degree from Duke.

The team has 23 people and Kevin lives in Boston. None of that is particularly convenient, and a good bit of the conversation is about what he’s built to make it work anyway.

Apple Podcasts | Spotify | YouTube | Web + Others

Kevin's key insights and practical takeaways

If you’re short on time, here are the most important lessons from the discussion.

A platform’s problem is that everything looks possible. Baseimmune started as a platform company, which sounds like an advantage until you have to choose! Kevin’s framing is that potential and possibility are the same thing as ambiguity when you’re deciding where to spend a limited budget. His team ran a large analysis before landing on IPF, and he describes picking the beachhead as the genuinely hard part. Not the science — the choosing.

Go after the data that could end the program. This is the core of the argument. Run the experiment that tells you whether this works, and do so early, before the construct is optimized and the supporting package is assembled. Either you find out you have something and raise on it, or you find out you don’t and you pivot while there’s still runway to pivot with.

The reason people avoid this isn’t laziness. Kevin’s read is subtler than that. Science is genuinely difficult, and there’s a persistent belief that continued tinkering will produce better proof and more confidence. It usually produces neither.

“It gives you a false sense of confidence.” His words on incremental de-risking, and the most useful sentence in the episode. You feel like you’re reducing risk. What you’re actually doing is spending time and money to postpone the question. Nick’s version: is the juice worth the squeeze?

Fast, cheap, good, and the bill you pay twice. Everyone knows the triangle. Kevin says he never wanted to believe it and has come around, with one hard-won addition. Choose fast and cheap and you get neither, because you’ll redo the work with someone else. His ordering now is quality first, speed second, and accept that cost surfaces somewhere regardless. Pick quality and cheap, and the slowness extends every downstream timeline, which is a cost with a different name.

Vet the people, not the logo. On CRO selection, Kevin and Nick converged. Kevin’s point is that the relationship matters more than the transaction, and that finding a partner who can actually hold your timeline is worth real effort. Nick added the practical version: reputation counts for something, but interview the individuals who will work your project, understand their approach, and confirm they’ve done this before. A recognized name doesn’t tell you who gets assigned.

Culture starts from where you are. Kevin has been at companies that began the culture exercise by asking what they wanted to be, and he thinks that’s the wrong door. If the aspiration is disconnected from the present, people can’t simply switch into it. He starts instead with an honest read: if someone walked in today and watched, what would they say our values are? Then you build stepwise from that.

Nick offered a method for this that’s worth stealing. Each member of his leadership team independently named five of their best employees and wrote descriptors for them. Then they put all the descriptors up together and looked for overlap. What emerged sat between aspiration and reality, because these were real people already living it, which made the resulting values credible rather than pie in the sky.

Data over ego. Baseimmune’s core tenet, and Kevin means it operationally rather than as wall decoration. Show me the data I probably don’t want to see. The behavior he’s protecting against is the instinct to figure out how to soften bad news before delivering it, which costs the team the discussion it should be having.

Transparency is a defense against invented stories. Nick’s framing, and Kevin agreed immediately. When you’re on the line about whether to share something, share it, because people will construct an explanation for the gap and their version is usually worse than reality. Nick added the part that makes it workable: the truth sets you free with hope. There’s a meaningful difference between telling your team things are going badly and telling them what you’re facing alongside how you intend to get through it.

Kevin’s own version of the job is turning uncertainty into clarity and action. Start with the direct account of where things stand, then say what happens next.

Write the decision down. Kevin’s team is in England and he’s in Boston, five hours behind, remote most of the year. What he’s lost is the hallway conversation that resolves the ambiguity a meeting created. So decisions get written, along with who owns what and what comes next. His line on why it matters more at distance: ambiguity compounds a lot faster when he’s out of the office for two or three straight weeks.

Nick runs something in parallel: a short-term to-do list reviewed at the top of every meeting before anything else is discussed. Last week you said you’d do this by today. Did it happen?

One name per item. Both were blunt here. If more than one person is responsible, nobody is. Nick’s supporting evidence was his daughter rewatching The Office and hitting the episode where Jim becomes co-manager.

Servant leadership has a cost, and that’s the point. Kevin gets up most days at 4:00 or 4:30 to work London hours, then works Boston hours after. He could make the team meet at his convenience. He doesn’t, and he’s clear that the commitment is visible and that visibility is what builds the reciprocal thing, where people know he has their back. Harder to establish at 70% remote than it would be sitting next to someone.

Nick connected it to a story about Arthur C. Brooks, who worried before joining Harvard about whether he’d succeed and was told he’d just need to be twice as productive and four times as nice.

Hire a scientific advisory board that argues with you. Among the signals Kevin watches for a needed pivot, SAB feedback ranks alongside the data itself and the capital environment. His standard is explicit: you hire a good SAB not to tell you you’re right but to challenge you and make the work better. He credits Baseimmune’s with real influence on the pivot decision.

Nick’s parallel is Vistage, the CEO group he’s been part of for 11 years, where the valuable sessions aren’t the ones that confirm what he already does. Those are the comfortable ones. The useful ones sting.

Change management takes twice as long as you plan. Every time. Kevin says he’s been wrong by roughly a factor of two, consistently, because the work isn’t with data sets, it’s with people. His three C’s are clear, consistent communication, repeated past the point where it feels redundant. And the shape of it surprised him: a long slow buildup where nothing appears to move, then alignment arriving almost at once. Looking back afterward, you see how long the invisible part took.

The story has to work for three audiences. Not just employees. Partners and investors need to understand why you’re pivoting, why it makes sense, and why you specifically are suited to do it.

Board management during a pivot is a deliberate workstream. Kevin paired the pivot with a corporate strategic plan, ran frequent board communication, and held multiple special board meetings to walk people through it. The plan leaned on competitive analysis, the fundraising environment, what large pharma is looking for, why the platform fit the direction, and SAB input for credibility. He describes investor management as a real part of the job rather than an interruption to it, and he put the time into making sure the board understood where the company was going and got genuinely interested in it.

Map your runway against your data honestly, even when it’s ugly. His number one instruction for founders raising early. Lay out when the data arrives, what it costs to get there, and where that lands against your runway. If the data you need for the next raise arrives after the money runs out, you’re in serious trouble, and that’s a fact worth discovering on a spreadsheet rather than in month fourteen.

Being always in fundraising mode means something specific. Not constant pitching. Telling investors what you’re going to do, when the data will land, and then following up when it does. The compounding effect is that they watch you predict and deliver. If the data is good, they now believe two things, because investors back teams as much as science.

Science gets you the meeting. Then it stops. Asked to weight science, team, execution, and strategy, Kevin gave science the most weight and then immediately qualified it. From banking he saw excellent science paired with weak teams and very little came of it. You need experience and credibility, and you need egos that won’t drive bad decisions. Strategy points the science somewhere, and pointing nowhere is worse than pointing imperfectly. Execution is what carries any of it toward patients.

Keep headcount flat and rent the rest. Kevin’s headcount hasn’t grown since he joined, by design. Work arrives in waves, three CRO studies running or a lull while the science resets, and a large fixed organization can’t titrate to that. He leans on CROs, CDMOs, and fractional people in the lab or in technical areas when the need spikes. Lean during the quiet, able to ramp when it isn’t.

Disagree and commit, with no ledger. His closing thought, tied back to data over ego. Have the hard debates, challenge each other with respect, then decide and move as a group regardless of whose idea won. The reason it works at Baseimmune, in his telling, is that he doesn’t track who said what. He tracks the best way forward.

Kevin Walton is Chief Executive Officer and a board director at Baseimmune, a London-based biotechnology company developing active immunotherapies for fibrosis, with idiopathic pulmonary fibrosis as its lead indication. He joined the company as Chief Business Officer before moving into the CEO role and leading a strategic pivot. Previously, he served as Senior Director of Corporate Development at Moderna, Vice President of Corporate Development at StrideBio, Senior Director of Business Development at Curis, and held roles in corporate development, strategy, and as chief of staff at G1 Therapeutics. He began his career in investment banking and consulting and holds a law degree from Duke University School of Law.

Connect with Kevin on LinkedIn here.

Who is The FDA Group?

The FDA Group helps life science organizations rapidly access the industry's best consultants, contractors, and candidates. Our resources assist in every stage of the product lifecycle, from clinical development to commercialization, with a focus on staff augmentation, auditing, remediation, QMS, and other specialized project work in Quality Assurance, Regulatory Affairs, and Clinical Operations.

With thousands of resources worldwide, hundreds of whom are former FDA, we meet your precise resourcing needs through a fast, convenient talent selection process supported by a Total Quality Guarantee.

Here’s why 17 of the top 20 life science firms access their consulting and contractor talent through us:

• Resources in 75 countries and 48 states.

• 26 hours average time to present a consultant or candidate.

• Exclusive life science focus and expertise.

• Dedicated account management team.

• Right resource, first time (95% success).

• 97% client satisfaction rating.

Talk to us when you're ready for a better talent resourcing experience and the peace of mind that comes with a partner whose commitment to quality and integrity reflects your own.

Subscribe to The Life Science Rundown:

Apple | Spotify | YouTube | Web + Others

Check out our newly launched AI-powered QMS audit tool, AICA (the Audit Intelligence Compliance Assistant).

He and Nick Capman get into beachhead decisions, why most preclinical teams run their experiments in the wrong order, how to keep a distributed team aligned when the CEO is an ocean away, and what investors actually weight when science, team, execution, and strategy are all on the table.

Some of what comes up:

• Run the experiment that could kill the program first, before the supporting package exists

• Accumulating incremental data feels like de-risking and mostly isn't

• Pick fast and cheap on a CRO and you'll pay for it twice

• Build culture from where you actually are, not from where you'd like to be

• Write decisions down, name one owner, and accept that ambiguity compounds faster at distance

• Hire a scientific advisory board that will argue with you

• Science gets you the meeting. Team, strategy, and execution decide what happens after

About Kevin Walton
Kevin Walton is CEO and a board director at Baseimmune, a London biotech developing active immunotherapies for fibrosis with IPF as its lead indication. Before Baseimmune he held corporate development and business development leadership roles at Moderna, StrideBio, Curis, and G1 Therapeutics, following a start in investment banking and consulting. He holds a law degree from Duke University School of Law.

About The FDA Group
The FDA Group helps life science organizations rapidly access the industry's best consultants, contractors, and candidates. Our resources assist in every stage of the product lifecycle, from clinical development to commercialization, with a focus on staff augmentation, auditing, remediation, QMS, and other specialized project work in Quality Assurance, Regulatory Affairs, and Clinical Operations. Learn more: https://www.thefdagroup.com/

The FDA just announced the final design of its Expedited Investigational New Drug Pilot and began accepting applications. Applications can be submitted until October 30, after which the FDA says it plans to review and choose participants by December 18. It expects to select eight to ten sponsor-QRI pairs for the initial cohort.

The pilot is part of HHS’s Operation TrialBlazer, and its stated goal is to compress the time between identifying a drug candidate and dosing the first human.

The FDA’s framing in the announcement strikes a competitive tone. The agency says first-in-human trials may take up to two years to complete in the United States, and that comparable trials finish considerably faster in China and Australia.

What the expedited IND pilot does

In short, sponsors pair with a qualified research institution, or QRI, that brings scientific expertise to IND preparation. The pair applies together, with the sponsor submitting the application.

The “mechanism” is a rolling review. Rather than waiting for a complete IND package before starting work, FDA will review and accept individual components during the pre-IND phase. The theory is that problems surface and get resolved while there is still time to fix them, which lowers the odds of a clinical hold once the 30-day IND review begins.

Note what that does and does not change: The 30-day IND review still happens. FDA states plainly that it retains full regulatory authority throughout, including whether an investigation may proceed and whether to impose a clinical hold. The pilot is designed to reduce the risk of a hold by front-loading the conversation, not to remove the possibility of one.

The pilot also encourages running IRB review and clinical trial site activation alongside IND development rather than in sequence.

What the FDA said about the feedback it got

Endpoints News reported from a press call on launch day, and the details there are maybe the most useful part of the announcement for anyone deciding whether to apply.

On who qualifies as a QRI, the FDA is taking what Endpoints characterized as a “non-prescriptive approach.” CBER Director Karim Mikhail reportedly described eligible institutions as potentially “private or public, academic or not,” so long as they can provide partnership and consultative support to the sponsor. Per the Endpoints report, the breadth is intentional, and Mikhail said the FDA wants to diversify the types of institutions it accepts to see whether different profiles produce different results. So this does not appear restricted to academic medical centers.

Regarding conflicts of interest, which came up in public comments on the proposal, Mikhail said the FDA is not delegating authority to anyone and that review teams will continue to conduct standard IND reviews during the pilot. It’s worth noting that the pilot webpage has specific conflict-of-interest requirements, so the practical constraints are addressed there rather than in the press release.

On the sharper question of whether adding a QRI could add bureaucracy rather than remove it, Endpoints reported that CDER Director Michael Davis said the expectation is that the sponsor and institution will have already worked through internal discussion and refinement of the IND before it reaches FDA. Davis described the pilot as built to reduce “unnecessary delays at every stage of early development.”

The IRB angle is one we think is worth watching here. Endpoints reported FDA’s view that IRB review has not been running in tandem with agency IND review, which delays trial starts, and that FDA hopes CROs and other trial organizers will propose faster ways to stand up IRBs while preserving protocol evaluation, leading to quicker site activation.

One more piece of context is that Mikhail and Davis are newly named permanent directors of CBER and CDER. Both were serving in an acting capacity as recently as early September.

Again, the FDA plans to review applications and choose participants by December 18, which would put selected pairs at the starting line around year-end.

Who is The FDA Group?

The FDA Group helps life science organizations rapidly access the industry's best consultants, contractors, and candidates. Our resources assist in every stage of the product lifecycle, from clinical development to commercialization, with a focus on Quality Assurance, Regulatory Affairs, and Clinical Operations.

With thousands of resources worldwide, hundreds of whom are former FDA, we meet your precise resourcing needs through a fast, convenient talent selection process supported by a Total Quality Guarantee. Learn more and schedule a call with us to see if we’re a fit to help you access specialized professionals and execute your projects on time and on budget.

Share The FDA Group's Insider Newsletter

We recently published our comprehensive guide to annual audit planning and companion checklist for Q3.

To step back and tackle GxP audit planning more generally, we put a question to our account managers who build client audit schedules and our auditors who travel to the sites: what do teams get wrong about scheduling, and what would you tell them to do differently next year?

What came back was heavy on sequence, confirmation, and leaving room. Here are seven of them, roughly in the order we’d act on them.

Download our free Excel audit planning workbook if you haven’t already:

Download the Excel workbook

1. Place the hard audits first in the schedule

Most schedules get built easiest-first. These are usually the domestic sites, the suppliers you talk to every month, the ones where you already know who to call. The “harder” audits end up in whatever slots are left over simply because the extra work and stress of scheduling get put off until after the easy ones are done.

We suggest trying to flip it if this sounds familiar. Place the constrained audits first, while the calendar is still empty and the people who can run them are still available. That means the site is in a region you’ve never audited, the one that needs an auditor who speaks a different language, the one where the auditor has to hold two or three qualifications, and anywhere the site itself only has a narrow window.

Easy audits stay easy as we get closer to the end-of-year crunch time. Hard ones don’t!

2. Know what your interval means before you pick dates

“Annual” means different things in different quality systems. Maybe that’s the calendar year. Maybe it’s 12 months from the last audit date. Maybe it’s 12 months with a documented grace period. Some procedures measure from the report date rather than the audit date.

Check which one yours says before you assign quarters because the difference is real. A site audited in March 2026 under an anniversary-based interval can’t sit in November 2027 without a deviation. Under a calendar-year interval, maybe it can!

Teams find this out after the schedule is locked more often than you’d expect.

3. Give one person the “pen” on every audit

One of the more common scheduling hiccups we see has nothing to do with auditor availability. It’s both sides assuming the other one is contacting the site.

Decide per audit, in writing, who books it. If you’re keeping the site relationships, your resourcing partner works through you on availability and you make the calls. If they’re coordinating, they need the contact name and the authority to reach out directly.

Either model works fine. A schedule where that field is blank invites problems that can always be avoided.

4. Confirm people along with dates

A confirmed date isn’t a confirmed audit. The SME who owns the system being audited must be on site that week, and so must someone with the authority to attend the closing meeting and respond to findings.

Auditors tell us the audits that could have gone more smoothly from a logistics standpoint are usually the ones where the right person turned out to be on vacation or otherwise not available, and nobody had checked.

5. Leave report time between audits

Report writing is part of the audit, not something that happens afterward in spare time. Booking an auditor Monday and Tuesday at one site and Wednesday and Thursday at another looks efficient on a calendar, but in practice, the first report gets written on a plane or in a hotel room or three weeks later, and findings are always sharpest when they’re written while the evidence is fresh.

Whenever possible, leave a few days between audits for the same auditor. If you’re combining two sites into one trip to save on travel, try to build in a buffer day, because a closing meeting that runs long at the first site will eat the opening at the second.

6. Decide on-site or remote before you book

Remote and hybrid audits work well a lot of the time, but they depend on things that take time to arrange:

Pick the mode when you build the schedule. Switching from on-site to remote three weeks out usually means the remote audit happens without the setup it needed, and everyone has a worse time.

7. Plan for the 10 to 15% that will move

Some share of your audits will move no matter how well you plan. Sites get inspected, contacts leave, and other things happen. A supplier has a recall and goes dark for a month.

If your calendar is packed to capacity, the first cancellation has nowhere to land, and it starts a chain reaction that ends with two audits falling out of Q4. Leave slack in Q2 and Q3 on purpose. That’s where a displaced audit can go without pushing anything else out. A schedule with room in it absorbs surprises. A full one is “fragile” and can easily break from things outside your control.

Putting it into a planning sheet

None of this is particularly complicated, but it's much easier to hold in a spreadsheet than in your head. Our free audit planning workbook gives you a row per audit with fields for most of what's above: mode, duration, fixed dates, site contact, who's coordinating, and which auditor you want back.

And if you’re looking at a 2027 schedule you still need to resource, now is the right time to talk. We cover GMP, GCP, GLP, GCLP, CSV, pharmacovigilance, and quality system audits worldwide. One proposal can cover your whole program, and you can add to it or drop from it as the year moves.

Get in touch with us

We’ve had quite a few conversations about everything auditing. Check them out if you haven’t already:

Thermo Fisher Scientific’s Director of Corporate Quality, David Festa, gives some advice on approaching supplier audits:

NubGenix’s Founder and CEO, Divya Gowdar, discusses the pitfalls and lessons learned on the front lines of audit and inspection readiness throughout the FDA-regulated industries:

And it’s an oldie, but a goodie, our conversation about the ins and outs of FDA PAIs with former FDA investigator, Chris Smith:

Who is The FDA Group?

The FDA Group helps life science organizations rapidly access the industry's best consultants, contractors, and candidates. Our resources assist in every stage of the product lifecycle, from clinical development to commercialization, with a focus on Quality Assurance, Regulatory Affairs, and Clinical Operations.

With thousands of resources worldwide, hundreds of whom are former FDA, we meet your precise resourcing needs through a fast, convenient talent selection process supported by a Total Quality Guarantee. Learn more and schedule a call with us to see if we’re a fit to help you access specialized professionals and execute your projects on time and on budget.

Share The FDA Group's Insider Newsletter

Regulatory operations professionals tend to get described by their outputs.

Hyperlinks, bookmarks, publishing, the mechanics of getting a dossier out the door correctly. Carla Griffin’s argument is that the description has always been too small and that a shift now underway at the FDA will make that obvious.

Nick Capman sat down with Carla to discuss what happens to reg ops professionals as the FDA reimagines how clinical data reaches the agency, and how they can lead that shift rather than be disrupted by it.

Carla is CEO and Principal Operations Consultant at The Sugar Water Operations Team, known as SWOT, which provides right-sized regulatory operations, submissions management, project management, and quality support to emerging biotech and pharmaceutical companies.

She has close to two decades of experience in regulatory and business operations, including eight years at Esperion, where she led development operations, regulatory operations, submissions management, and medical writing, and later headed strategic business operations. Before that, she held regulatory roles at Sarah Cannon Research Institute, LIQUENT (a PAREXEL company) specializing in eCTD dossiers and submissions, and Human Genome Sciences. She holds a master’s in regulatory science from Johns Hopkins and a master’s in professional, technical, business, and scientific writing from Towson University.

Apple Podcasts | Spotify | YouTube | Web + Others

Carla's key insights and practical takeaways

If you’re short on time, here are the most important lessons from the discussion.

What’s actually changing is the gap between data existing and FDA seeing it. Carla has been in the industry for twenty years, starting when submissions still involved paper. Plenty has advanced since, but one thing has stayed constant: the time it takes to receive data and get it to the agency. Real-time clinical data collapses that window. That’s the change, and everything else follows from it.

The current model waits on Module 5. While data is being produced and cleaned, teams prepare Modules 1 through 4: administrative, summary, CMC, and nonclinical. Then Module 5, the clinical content, arrives wrapped in a clinical study report, and the whole package goes at once unless you’re doing a rolling review. If the agency has been watching signals as they emerge, the questions that follow are open ones. What does that mean for the ESG gateway? Does information get resubmitted after the fact? Do clinical study reports serve the same function? Carla’s answer is that the industry just doesn’t know yet.

The data cleaning question cuts both ways. Between data entry and data management, a lot happens before FDA normally sees anything, and it’s generally viewed as a good thing that the agency doesn’t receive raw data. How data gets cleaned before reaching the agency under a real-time model, and whether the agency scrutinizes it differently knowing the sponsor hasn’t had the usual time, is something Carla says remains to be seen. She wouldn’t call it a positive or a negative yet.

The clear positive is patient access. Shortening the interval between knowing a drug is effective and getting it to patients is the point, and these programs are aimed at serious unmet medical need. Carla is unambiguous that this part is a win.

On timelines, she speculates and says so. Asked how much time might come out of review and approval, Carla offered fifty percent, while noting that many pilot details had not been made public. Her reasoning is that if clinical data arrives continuously, the other modules have likely already been reviewed in some form, leaving clinical review, labeling, and launch. Worth reading alongside FDA’s own projection, which is a 20 to 40 percent reduction in trial duration, a related but different measure.

The hardest adjustment is professional, not technical. Reg Ops is a black-and-white crew, in Carla’s words, and that systematic instinct is a genuine strength. It’s also the thing most likely to get in the way here. ESG and the processes built around it are fifteen to twenty years old in places. Adapting may mean clearing the slate rather than mapping new requirements onto old workflows, and asking “how will this work?” too early can become a reason not to move.

Expect less runway than usual. FDA has historically given industry time to absorb change. Carla’s expectation is that this window will be shorter, and reasonably so, because the whole point is getting products to patients faster.

She raises the possibility that ESG itself changes. Carla floats this and immediately hedges it, so read it as a question rather than a forecast. Her observation is that FDA now has tools it didn’t have when ESG was built, including the NextGen Portal, which has made things like INTERACT meeting requests and BLA number requests considerably simpler than routing everything through the ECTD and ESG structure. Her point is less that ESG disappears and more that industry shouldn’t expect the FDA to remain anchored to a standard just because the industry invested heavily in it.

Reg ops people are problem solvers who’ve been siloed. This is the heart of her argument. Reg ops specialists are often the first to see a solution, and they’ve narrowed it down to links, bookmarks, and document construction. Their capability is broader than the role usually allows. A shift this significant creates the reason to bring them, and IT, into decisions much earlier.

Getting to the table earlier is the whole opportunity. If the goal is getting data to the FDA sooner, you have to examine whether your current infrastructure supports it, and that examination requires reg ops and systems people in the room. Carla’s warning is direct: reg ops has historically not been included as early as it should be, and if that continues, the function does itself a disservice and falls behind.

Speed to market is where the cost of waiting shows up. For biotech in particular, exclusivity and getting there before the market saturates are what keep investors satisfied. Carla’s picture of the company that doesn’t adapt is one that ends up on the sideline, potentially readjusting an indication because someone else arrived first.

AI tools are what reg ops already used, on steroids. Reg ops professionals like technology and are constantly looking to improve what they have, so the enthusiasm is natural. Her caution is the industry’s standing one: just because you can do something doesn’t mean you should. But she pairs it with the reverse: that something looking risky isn’t a reason to run from it.

Look for options, not solutions. This is the sharpest reframe in the conversation. When evaluating technology, reg ops shouldn’t be hunting for a solution. It should generate options, then apply what it knows to eliminate risk and turn an option into something viable. That’s a problem-solving posture rather than a purchasing one.

Everything compresses. Carla expects what would have taken five years to take two, and what would have taken two to take six months, simply because of the pace of technology. Her conclusion is that sitting on the sidelines waiting for others to lead on something like this is a luxury the industry no longer has.

But don’t chase everything, either. Asked how much to get ahead versus wait, she called for balance and meant it. Chasing every solution produces exhaustion, and there’s still day-to-day work to do. Carve out time to listen to podcasts, attend webinars, and talk to peers about what they’re doing. There’s also something to be said for letting others vet solutions and joining when the timing suits you.

Three things to do in the next twelve months.

1. Stay aware of what’s out there without dismissing it as short-lived. Free podcasts and webinars let you monitor developments without spending real resources, which matters when you’re a team of one or two.

2. Understand the quality implications, because what makes a strong reg ops specialist isn’t knowing how to use a tool but understanding the consequences of using it.

3. And learn to vet vendors properly!

Put on your FDA hat! Carla spent significant time on FDA inspections, and she uses that lens deliberately.

• Where are the data integrity questions going to come from?

• Why did we do what we did, and how?

• How do we troubleshoot?

• How do we demonstrate that a human was in the loop alongside an AI solution? Her recommendation is to sit with your quality professionals, read your guidances and regulations, and pressure-test what you’re planning before you do it.

Vendor vetting is the most concrete advice in the episode. Plenty of vendors will tell you they can build it, and Carla assumes they can. The question is whether they can build for this industry. Are they aware of the regulations you’re held to, and how do they help you address them? If a vendor can’t answer that, and the task is to get a viable solution running quickly, she doesn’t spend time there. Those vendors fall away, and the ones who understood your challenges from the start are the ones worth pursuing.

Old technology isn’t the enemy. She’s excited about what’s next, but not for its own sake, and she values the things in this industry that have worked for a long time. ESG took enormous effort and remains a strong system. The task now is finding the balance: keeping what’s tried and true, enhancing it with capabilities that didn’t exist before. Old technology isn’t the enemy. It’s just at a disadvantage now.

Carla Griffin, M.A., M.S., is CEO and Principal Operations Consultant at The Sugar Water Operations Team (SWOT), which provides right-sized operational support to emerging biotech and pharmaceutical companies across regulatory operations, publishing and systems, submissions and project management, clinical operations, standard operating procedures, and medical writing. She has close to two decades of regulatory and business operations experience, including eight years at Esperion, where she led development operations, regulatory operations, submissions management, and medical writing before heading strategic business operations. Earlier roles include Senior Manager of Regulatory Affairs at Sarah Cannon Research Institute, Senior Regulatory Affairs Consultant for eCTD dossiers and submissions at LIQUENT (a PAREXEL company), and Manager of Regulatory Operations at Human Genome Sciences. She holds a Master of Science in regulatory science from Johns Hopkins University and a Master of Arts in professional, technical, business, and scientific writing from Towson University.

Connect with Carla on LinkedIn here.

Who is The FDA Group?

The FDA Group helps life science organizations rapidly access the industry's best consultants, contractors, and candidates. Our resources assist in every stage of the product lifecycle, from clinical development to commercialization, with a focus on staff augmentation, auditing, remediation, QMS, and other specialized project work in Quality Assurance, Regulatory Affairs, and Clinical Operations.

With thousands of resources worldwide, hundreds of whom are former FDA, we meet your precise resourcing needs through a fast, convenient talent selection process supported by a Total Quality Guarantee.

Here’s why 17 of the top 20 life science firms access their consulting and contractor talent through us:

• Resources in 75 countries and 48 states.

• 26 hours average time to present a consultant or candidate.

• Exclusive life science focus and expertise.

• Dedicated account management team.

• Right resource, first time (95% success).

• 97% client satisfaction rating.

Talk to us when you're ready for a better talent resourcing experience and the peace of mind that comes with a partner whose commitment to quality and integrity reflects your own.

Subscribe to The Life Science Rundown:

Apple | Spotify | YouTube | Web + Others

Check out our newly launched AI-powered QMS audit tool, AICA (the Audit Intelligence Compliance Assistant).

Carla and Nick Capman get into why the hardest adjustment is professional rather than technical, how to vet technology vendors who don't understand your regulations, and the three things reg ops professionals should do in the next twelve months.

A few of Carla's key takeaways:

• Reg ops specialists are problem solvers who've been siloed into links, bookmarks, and document construction

• The instinct that makes reg ops strong, wanting everything systematic, is what will make this shift hardest

• Getting reg ops and IT into decisions earlier isn't optional if the goal is getting data to FDA faster

• When evaluating technology, look for options rather than solutions, then eliminate risk to make an option viable

• Ask vendors whether they understand the regulations you're held to. If they can't answer, move on

• Put on your FDA hat: where do the data integrity questions come from, and can you show a human was in the loop?

• Old technology isn't the enemy. It's just at a disadvantage now

Note that this was recorded in September 2026. The FDA's real-time clinical trials work has been developing quickly, and Carla's forward-looking comments are her own assessment rather than announced agency policy. New developments may supersede the information provided here.

About Carla Griffin
Carla Griffin, M.A., M.S., is CEO and Principal Operations Consultant at The Sugar Water Operations Team (SWOT), supporting emerging biotech and pharmaceutical companies across regulatory operations, submissions management, and quality systems. She has close to two decades of experience, including eight years at Esperion leading development and regulatory operations, with earlier roles at Sarah Cannon Research Institute, LIQUENT (a PAREXEL company), and Human Genome Sciences. She holds a master's in regulatory science from Johns Hopkins.

About The FDA Group
The FDA Group helps life science organizations rapidly access the industry's best consultants, contractors, and candidates. Our resources assist in every stage of the product lifecycle, from clinical development to commercialization, with a focus on staff augmentation, auditing, remediation, QMS, and other specialized project work in Quality Assurance, Regulatory Affairs, and Clinical Operations. Learn more: https://www.thefdagroup.com/

Welcome to Insider issue #52.

Also, hello to the 214 life science professionals who joined us last month. If you haven’t already upgraded to a paid subscription to unlock our in-depth analysis issues like this one, you can do so here. Read our past deep dives here.

This month, Diamond Dent-Mitchell walks through the finalized guidance on formal meetings between the FDA and sponsors of PDUFA products. It replaces the 2017 and 2018 versions and rewrites the rules for every formal meeting a drug or biologic sponsor requests during development. We unpack the new 10-question ceiling, how briefing packages are due with the request for certain meeting types, a three-day window to confirm you still need a meeting, and the agency’s new authority to turn your meeting into a written response whether you agree or not!

Regina Atim covers the revised human factors guidance, updated August 3, 2026, and how it harmonizes usability engineering with your QMSR obligations. The short version: human factors stop being a standalone box to check and get wired into your actual design controls. Don’t miss this one if you’re on the device side.

Jesse Hart closes with the Linemaster Switch warning letter. It’s the first warning letter the FDA posted under the QMSR and the citations are written against the ISO 13485 clause. Jesse just finished a 483 remediation for a small manufacturer and recognized nearly every finding, so he walks both sides: what the agency cited, and what the fix actually looks like on paper.

Download our slide decks to use any way you’d like:

Read more

These RA/QA news rolls are available in full only to paid subscribers. Upgrade here. If you haven’t already taken our reader survey, it should only take 1 minute here.

This is our biweekly recap of news and industry happenings curated for our paid subscribers. Our goal is to bring you the headline news and the stories, research, and insights that might be harder to find.

If you haven’t upgraded to a paid subscription yet, you can do so here. Also, make sure to follow us on LinkedIn and catch the latest episodes of our podcast, The Life Science Rundown.

Here are some key items from the past couple of weeks.

💊 = Pharma, biologic, and biotech.
💉 = Medical device, medtech, diagnostics, and digital health.

Special Announcement!

We're excited to announce a very special guest coming on the Life Science Rundown podcast in September: Robert M. Califf, MD, MACC!

Dr. Califf currently serves as an Instructor in the Department of Medicine and a Member of the Duke Clinical Research Institute at Duke University, and is an Adjunct Professor of Medicine in the Division of Cardiology. He previously served as FDA Commissioner and Deputy Commissioner for Medical Products and Tobacco. Before his FDA roles, Dr. Califf was a professor of medicine and vice chancellor for clinical and translational research at Duke, director of the Duke Translational Medicine Institute, and founding director of the Duke Clinical Research Institute.

He is also a Member of the National Academy of Medicine, has served on multiple FDA advisory panels and NIH advisory committees, and has led major initiatives to improve methods and infrastructure for clinical research, including the Clinical Trials Transformation Initiative (CTTI).

More details to come. If you aren't already, make sure you're subscribed wherever you listen to podcasts so you don't miss our conversation! We’ll of course post it here on the Insider Newsletter, too.

Alright, back to our regular newsroll items:

FDA Leadership and Administration

• Commissioner search ends with a pick: The White House’s search for a permanent FDA commissioner turned several times this month, with reporting that Rep. Buddy Carter (R-Ga.) had entered the running before later reporting indicated President Trump had picked White House policy aide Heidi Overton for the job. Carter is a licensed pharmacist who previously ran his own pharmacy and is leaving Congress at the end of this term after losing his state’s Republican primary. Reporting through the summer had described a three-way contest between Overton, senior Pentagon health official Stephen Ferrara, and New York Cancer & Blood Specialists Chief Executive Jeff Vacirca. Overton’s confirmation runs through the Senate HELP Committee, where several members are expected to treat mifepristone regulation as a litmus test. Acting Commissioner Kyle Diamantas can continue serving until early December under the Vacancies Act, a term that extends upon the White House's formal submission of a nominee. We wrote about Heidi Overton here:

• HHS plans two new deputy commissioner roles: CNBC reported that HHS is planning to appoint deputy commissioners for drugs and for technology at the FDA, two positions that would report directly to the commissioner and oversee medical product portfolios. The FDA previously had a deputy commissioner for medical products and tobacco, a role once held by former Commissioner Robert Califf before it was eliminated under former Commissioner Scott Gottlieb. According to the report, the technology and artificial intelligence position could go to FDA senior advisor Jared Seehafer, who joined the agency last year and previously led the quality management software company Enzyme. No candidate has surfaced for the drugs role. Taken together with the pending commissioner nomination, the vacant principal deputy commissioner position, and the unfilled permanent director jobs at CDER and CBER, the agency’s leadership structure is likely to look substantially different by the start of 2027, with center directors holding less influence than they do now.

• Tarver meets with a sterilization firm and a standards body: 💉 The FDA’s leadership public calendar shows two entries for CDRH Director Michelle Tarver, one with representatives from Steris and the other with the British Standards Institution. The topic of the Steris meeting was not listed, though the firm plays a significant role in the device supply chain, an administrative priority. The BSI meeting is notable because the organization does not currently appear in the agency’s database of recognized consensus standards.

FDA General

• FDA opens the conversation on regulating generative AI devices: 💉 The FDA issued a discussion paper on the regulation of generative artificial intelligence-enabled medical devices, open for comment through October 19. Digital Health Center of Excellence Director Rick Abramson described the paper as a jumping-off point rather than a formal policy statement. The proposals include a new type of master file for the foundation models that underpin AI functions and a competency-based approach to performance assessment, drawn from clinician credentialing. Some concepts fall outside current agency practice, including the involvement of users of generative AI tools in postmarket surveillance planning. Abramson told STAT in a subsequent interview that the agency wants input from the broader ecosystem before translating the concepts into policy, and that the eventual guidance will be stronger because it has gone through the process.

• Citizen petition asks where vision language models fall: 💉 A citizen petition from Mosaic Clinical Technologies asks the FDA to clarify where AI-enabled vision language models sit within its regulatory paradigms. As the petition describes it, some VLM functions fall outside FDA oversight under the statutory definition of software as a medical device, while others would be regulated as devices. The petition specifically asks whether a VLM marketed with the expectation of later fine-tuning on institution-specific data is itself a device requiring premarket clearance or approval, whether such a model is a component or a finished device, and how these tools fit the agency’s carve-out for the practice of medicine.

• New draft guidance on choosing between ANDA and 505(b)(2): 💊 The FDA issued draft guidance on August 17 that would revise and replace 2019 recommendations describing whether a sponsor should submit an Abbreviated New Drug Application or a 505(b)(2) application. The ANDA pathway covers generic drugs, while 505(b)(2) applies to products closely related to but not identical to already approved drugs. The new draft adds information on duplicates and ANDA approval eligibility, clarifies language throughout, and adds footnotes. Updating this guidance has sat on the agency’s agenda since at least 2023.

• Therapeutic equivalence guidance finalized: 💊 The FDA released a final guidance describing how it determines whether a brand-name product and its would-be generic are therapeutically equivalent and how it assigns the resulting Orange Book codes. Therapeutic equivalence codes carry real weight in clinical practice and patient access, since certain coding permits automatic substitution by pharmacies. The final document closely mirrors the July 2022 draft, with substantive changes limited to the evaluation request process and information on revisions.

• Fifty-five new product-specific guidances published: 💊 The FDA published 55 new and revised draft product-specific guidances on August 21 to aid generic drug development by describing how to demonstrate therapeutic equivalence. More than 30 cover drugs with no currently approved generic, including 15 for complex products. Two, for ferumoxytol injection and a benzoyl peroxide/tretinoin topical cream, address products supported by GDUFA-funded research. The agency also added 11 new PSGs to its list of documents under development.

• Workshop scheduled on testosterone in menopausal women: 💊 The FDA announced on August 17 that it will host a public workshop on September 17 focused on testosterone use in menopausal women. The notice says the event will examine current scientific evidence and knowledge gaps to inform future research and potential drug development of testosterone products for this population. The meeting continues a line of agency attention that began under former Commissioner Martin Makary, who convened an expert panel on hormone replacement therapy for menopause and subsequently moved to remove long-standing safety warnings. A December panel on testosterone replacement therapy in men was followed by an April call for sponsors of approved products to contact the agency about a potential indication for low libido in men with idiopathic hypogonadism, and by class-wide safety labeling changes in July.

• Bayesian guidance webinar set for October: 💊 The FDA will host a webinar October 14 to review its January draft guidance on Bayesian statistics, according to a newly posted calendar listing. Agency statisticians John Scott, James Travis, and Van Tran will present, with Division of Biometrics II Director Mark Rothmann and Division of Pediatric and Maternal Health Deputy Director John Alexander serving as panelists. The three presenters also recorded a video overview of the guidance that the agency posted recently as part of CDER’s Small Business and Industry Assistance work.

• ASCO warns on ifosfamide supply: 💊 The American Society of Clinical Oncology warned that some oncology practices are experiencing critical supply disruptions of the chemotherapy drug ifosfamide, leaving them with less than a few weeks of inventory. According to the society, the FDA plans to temporarily allow importation from overseas manufacturers that do not typically supply the U.S. market. The agency’s database shows the drug unavailable from Baxter Healthcare, Fresenius Kabi, and Hikma Pharmaceuticals. Fresenius and Hikma both cited increased demand and expect more supply in August and September respectively, while Baxter attributed its shortage to requirements related to complying with good manufacturing practices and expects to recover in September.

• Explainer posted on active immunotherapy potency guidance: 💊 The FDA posted a short webinar explaining its recent draft guidance on potency assessments for active immunotherapy products. Ramjay Vatsan of CBER’s Office of Therapeutic Products provides the overview. The agency is accepting feedback on the draft recommendations through November 18.

• Stakeholders question the expedited IND pilot design: 💊 More than 200 comments were submitted on the FDA’s request for information about its proposed pilot for expediting Investigational New Drug applications, including roughly 100 the agency added to the docket on August 26. Stakeholders generally supported the objective and agreed that Phase 1 trials could be accelerated in the U.S., with many describing the pilot as a worthwhile experiment. Others questioned whether the envisioned network of qualified research institutions would duplicate work or introduce conflicts of interest. BIO and PhRMA both expressed skepticism that adding QRIs to the process would meaningfully speed timelines, suggesting instead that the proposed rolling IND submission process could be expanded.

• Fourth TEMPO participant is a mental health app: 💉 The FDA announced a fourth participant in its Technology-Enabled Meaningful Patient Outcomes pilot for digital health products. Under TEMPO, developers can offer products without premarket authorization within CMS’s companion ACCESS pilot. The newest addition is the SonderMind Adjunctive Care Application, described on the TEMPO landing page as a smartphone-delivered mental health app intended to reduce levels of depression and anxiety in adults 22 and older who are already in treatment with psychotherapy or pharmacotherapy. It is the second digital health product added this month, following the Limbic Unpacked AI-voice agent therapy chatbot. Both functions are contraindicated for serious mental health issues, and both companies participate in the CMS side of the joint pilot.

• Latest MDUFA performance report posted: 💉 CDRH published its Q3 2026 MDUFA report, covering the agency’s progress against the medical device user fee performance goals set out in the MDUFA V commitment letter. The reports give turnaround times for specific application types across the device divisions and show that CDRH generally meets its performance goals. They also provide a snapshot of overall submission volume to the center.

• Accessory classification criteria remain muddled: 💉 The FDA published its regularly scheduled proposal on August 17 to give distinct classification to certain proposed accessories as Class I devices, with comments open through mid-October. Accessories are products that support, supplement, or augment the performance of one or more parent devices. On August 26 the agency posted a new webpage explaining how it considers the criteria for distinct Class I classification, and the two documents lay the issue out differently. The Federal Register notice lists three criteria, the webpage lists four, and the 2017 final accessories classification guidance asks two questions. The divergence turns on how the need for design controls is handled: the third criterion asks whether general controls suffice, while the sometimes-listed fourth asks whether design controls specifically are necessary and, if so, whether the accessory is automated with computer software, in which case the design control requirement would continue to apply even in Class I.

• FDA seeks a transcranial ultrasound modeling tool: 💉 A contract notice posted earlier in August says the FDA’s Office of Science and Engineering Laboratories is seeking a research contractor to develop a computational modeling package for simulating transcranial ultrasound. The package would be disseminated to device developers, researchers, and regulators as a regulatory science tool. OSEL’s catalog of RSTs is a peer-reviewed resource intended for use where standards and qualified Medical Device Development Tools do not yet exist. RSTs can offer directional guidance for developers working on novel technology types, but they do not carry the regulatory weight of MDDTs.

• Centers align on digitally derived measures: 💊💉 A new white paper jointly published by the FDA’s medical product centers seeks to align expectations across the agency for leveraging digitally derived measures. The paper stops short of offering new policy recommendations, instead pulling together relevant material from existing guidance that could apply to DDM development and use, including the final guidance on digital health technologies in drug development, the four-part Patient-Focused Drug Development series, and human factors considerations. It arrives as the agency prepares a guidance document on engaging with the FDA about the use of DHTs in drug development.

• Updated COVID-19 vaccines approved for the fall: 💊 The FDA approved updated COVID-19 vaccines from Pfizer and BioNTech, Moderna, and Sanofi on August 27. The new formulations target the JN.1-lineage XFG variant recommended by the agency’s Vaccine and Related Biological Products Advisory Committee at its May meeting. All three are approved for adults 65 and older and for younger individuals with at least one underlying condition placing them at high risk of severe outcomes. Pfizer’s vaccine carries the high-risk indication down to age 5, while Moderna’s and Sanofi’s extend to age 12. Pfizer and Moderna said their vaccines would be available within days. Sanofi, which acquired rights to its vaccine from Novavax, had not issued a statement as of publication, though Novavax said the product was approved for the 2026-2027 season.

Compliance and Enforcement

• FDA “wins" against compounders on shortage determinations: 💊 The 5th U.S. Circuit Court of Appeals affirmed a district court judgment in favor of the FDA on August 27 in two closely related cases over whether declaring a drug shortage resolved amounts to a federal rulemaking exercise subject to the Administrative Procedure Act. The appellate court found that any error by the agency in not proceeding through notice-and-comment procedures was harmless and that its ultimate action was not arbitrary or capricious. The court noted the agency had been regularly communicating status updates in a way that in practice extended well past the APA’s notice-and-comment period, which let it sidestep arguments about whether the APA applied at all or whether, as the FDA argued, the entire set of actions constituted an adjudication rather than a rulemaking.

• Report finds QMSR inspections reaching deeper into quality systems: 💉 A new report from MasterControl identifies differences between inspection outcomes under the Quality Management System Regulation, which took effect this year, and those under the previous Quality System Regulation. The report found that QMSR inspections focus more on continuous readiness, with investigators shifting toward a comprehensive risk-management view rather than periodic status checks on compliance with individual requirements. It also covers changes under the March draft guidance on responding to Form 483s, which so far applies only to drug manufacturers, and describes the increasing convergence of global quality standards.

Read more

The FDA just published a post on its “Voices” blog telegraphing to the industry that, as the title suggests, “Good Clinical Practices Are Not Optional.”

Four officials signed it: the acting directors of CDER and CBER, the director of CDRH, and the director of the Oncology Center of Excellence. Drugs, biologics, and devices, in one message, so everyone involved in clinical work should pay attention to this one.

Before we dig into it, this is a blog post, not a guidance document. The FDA says so directly, calling this the logical application of existing law rather than a new policy. It’s best framed as the FDA telling the industry where enforcement attention is moving and what a program should therefore look like from that vantage point.

If this sparks a need for GCP support in any dimension, get in touch with us to get paired with an SME from our bench. We support firms across basically any axis of clinical work, especially clinical quality operations staff augmentation and auditing.

And be sure to watch our conversation on GCP compliance and inspection readiness if you haven’t already:

The two core mechanisms behind the FDA’s warning

The FDA goes through two different mechanisms to reject data in its post, and the distinction matters for how you assess risk.

For devices, the post frames the same principle through evidence standards: all evidence in a device submission must be credible, including clinical evidence, and the FDA relies on valid scientific evidence to determine whether there is reasonable assurance that a device is safe and effective.

The access problem the agency is encountering

Maybe the most concrete passage in the post is the FDA describing what it has run into around access. For some foreign inspections of clinical trial sites, the agency has said it's been denied access outright, or told that inspection would require signing agreements that restrict the scope or conduct of the inspection, or that attest to geopolitical principles unrelated to the inspection.

The FDA is pretty clear here: it will not sign those agreements! Which means the exposure at such a site has nothing to do with the quality of the science being done there. Good data at an inaccessible site is still data that the FDA may decline to accept.

We’ve heard auditors working in this space often describe the sponsor-side version of the same wall: being refused access to electronic medical records and source documents at some foreign sites unless they agreed to conditions no auditor could reasonably accept. That experience points to something useful, though. If your own monitors or auditors have hit access restrictions at a site, you already have evidence about what the FDA might (or likely will in some cases) encounter. That friction is a leading indicator sitting in your own files. If you’ve encountered it, address it.

The FDA’s framing of the obligation is actually worth quoting in your internal materials to make sure it accommodates:

• Before the FDA opens the door to let a product reach the American market, sponsors and investigators must open their doors to the FDA.

• That means access to sites and records, including source data and consent documentation.

• And sponsors should treat an inability to inspect or access all relevant data as a material factor in regulatory strategy rather than an administrative footnote.

What changes operationally

To our reading, there are three actions in the post that could be cast as resourcing and process changes rather than statements of principle.

1. More foreign BIMO resources

The FDA says it’s adding resources for foreign Bioresearch Monitoring inspections, including inspecting more Phase 1 and early-stage trials, and expanding the scope of some inspection assignments to cover more trials occurring at a given facility. It’s also planning updates to the risk-based criteria it uses to select sites to better reflect compliance risks in specific countries or regions.

There are two very important implications there:

Read more

We’ve been wanting to have a conversation about the benefits of bringing Quality in earlier for a long time.

While we see some teams make progress here, it’s still the case that by the time QA is consulted, many technical decisions have already been made. The capital is committed, the headcount is set, and investor expectations are locked. So when a problem surfaces, the question isn’t whether to fix it — it’s how expensive the fix has to be.

That’s the argument Dr. Andrea Bell makes, and her framing is sharper than the usual case for involving Quality sooner. She argues (and we agree) that late QA involvement doesn’t just add cost. It removes the option to remediate cheaply, which is the whole premise behind right-the-first-time. We see teams find this out the hard way more often than we'd like.

Nick Capman sat down with Andrea to talk about moving QA from late-stage gatekeeper into an early, risk-appropriate technical partner.

Andrea is a biotech executive with more than 25 years of experience leading Quality and CMC organizations across biologics, RNA therapeutics, vaccines, and small molecules. She currently serves as a Vice President of Global Quality.

Throughout her career, she’s been recognized for building high-performing teams, developing fit-for-purpose organizations and quality systems, and driving complex programs from early development through commercialization, helping bring innovative therapies to patients worldwide.

She holds a doctorate in Global Health Sciences, a master’s degree in Quality Assurance and Regulatory Affairs, an MBA in International Business, and is passionate about advancing global access to transformative medicines.

She was also candid about why the topic matters to her. She’s worked on both sides of the divide, in organizations where Quality wasn’t consulted early or empowered to act, and in companies where Quality was built in as a partner from the start. So rather than the typical theory, she speaks to what she’s actually seen work (and fail).

The views expressed in this conversation are Andrea's own and do not represent the position or views of her employer.

Apple Podcasts | Spotify | YouTube | Web + Others

Andrea’s key insights and practical takeaways

If you’re short on time, here are the most important lessons from the discussion.

Late QA involvement removes your cheap options. This is the core of it! When QA sits organizationally downstream and isn’t consulted until after technical decisions are made, the costs land after the company has already committed capital, headcount, and investor expectations. At that point, you can still fix the problem. You just can’t fix it inexpensively.

Most of the cost of poor quality sits below the waterline. Andrea referenced the FDA’s iceberg model. Above the line are the direct costs everyone counts: recalls, lost revenue, legal and regulatory expenses. Below it lies the larger, less visible aggregate, including scrap rates, cycle-time delays, market share loss, costly investigations, and reputational damage that can take years to repair or may never fully recover. Those eventually show up in stock performance!

A single 483 carries a real remediation cost before you count the opportunity cost. Andrea cited a range of roughly $250,000 to $5 million in direct remediation for one 483 and noted that the figure excludes delayed clinical timelines and stalled BLAs. Both of those affect patient access.

Early means early in the conversation, not just early in the lifecycle. This distinction is easy to miss. Andrea’s point is that “early” also applies to the decision forums that occur at every phase: design-of-experiments reviews, tech transfer meetings, and the discussions where the technical calls are actually made. Being invited to those is different from being involved early in a general sense.

ICH Q8 grounds the argument. Quality target product profiles and critical quality attributes should be defined before process development begins. Having QA at the table when those are defined, rather than reviewing them afterward, is where the value shows up.

What this looks like across the lifecycle. Andrea walked through it phase by phase. In R&D and discovery, through first GMP, QA joins cell line, cell bank, and asset qualification discussions as they happen, with systems staying fit for purpose and a focus on traceability and data integrity.

• In Phases 1 and 2, QA reviews the design of experiments alongside process scientists so critical process parameters get identified upfront rather than reverse-engineered after a deviation.

• In late Phase 3 and pivotal work, QA shifts toward process lockdown support, and the CPPs, CQAs, and control strategy get finalized there rather than under filing pressure or in response to agency questions.

• At the NDA and BLA stages, validation is already complete, so PPQ confirms a known relationship rather than discovering one, and QA moves from inspection to inspection readiness by stress-testing systems before the PAI.

• Post-launch, QA stays in the continuous process verification loop with change control and real-world manufacturing knowledge feeding back into the program.

Risk-based doesn’t mean less rigorous. It means rigor pointed where the risk is. ICH Q9 frames this around two principles: risk evaluation grounded in scientific knowledge and tied to patient protection, and effort, formality, and documentation commensurate with risk rather than maximal by default. Andrea’s read is that applying commercial-level scrutiny at Phase 1 doesn’t make you safer. It misallocates resources away from where the actual risk sits.

The rigor doesn’t loosen; it strengthens! You still identify hazards, analyze and evaluate risk, and put controls in place, using tools like FMEA systematically. What you gain is a defensible reason behind every decision, which is what agencies are looking for. A uniform checklist applied across the board is a weaker position, and agencies will challenge it.

Three questions focus the risk analysis. Is there an impact on the patient, the product, or the process? Andrea uses that frame during Phase 1 and 2 technical evaluations to keep attention where it belongs.

The barriers are cultural, not procedural. This is the part most organizations get wrong. The obstacles to embedding QA earlier are usually a lack of a quality culture and a reporting structure, along with a genuine misunderstanding of QA’s responsibilities. A QA function only involved in late development can often measure nothing but deviation closure rates, which pushes it into a reactive mode no matter how capable the people are.

Three concrete moves leaders can make. Give quality standing membership in the technical forums. Run a formal maturity assessment to establish a baseline worth tracking against, using ISPE’s Advancing Pharmaceutical Quality program or PDA’s quality culture assessment tool. And commit at the leadership level, which ICH Q10 identifies as foundational, meaning resources are committed and quality is genuinely empowered.

Culture follows what leadership measures and rewards. Andrea’s observation from culture survey data. If leadership doesn’t empower Quality and doesn’t reward behaviors that support patient safety, the organization won’t produce it, regardless of what the procedures say.

The Q8, Q9, Q10 trifecta is the operating framework. Q8 defines quality through QTPPs and CQAs. Q9 supplies risk prioritization methods and the tools for determining critical process parameters and controls. Q10 provides the pharmaceutical quality system lifecycle that carries those decisions forward. Newer capabilities, such as real-time release testing and process analytical technology, build on that foundation by generating data during the process rather than only at batch record review.

Data makes QA proactive only if people are empowered to act on it. Both halves are required. Andrea framed the quality management review as where this gets tested, and a functioning one does three things: it surfaces metrics that can be acted on rather than burying them in a report nobody reads until the next audit, it escalates when a threshold is breached instead of waiting for the next scheduled review, and it carries real authority.

Recurring unresolved issues in the QMR cycle are visible to agencies. Andrea was pointed here. Where health authorities have issued 483s or provided feedback, a pattern of issues recurring quarter after quarter without resolution signals either that they aren’t receiving attention or that leadership isn’t empowered. Raising the issue isn’t enough. Showing the data isn’t enough! State of control means acting in real time rather than batching problems downstream, where they compound and sometimes become unsolvable.

QA earns credibility by changing what it brings, not just when it shows up. The gatekeeper posture is arriving to say no. The partner posture is to sit with the technical team, ask what could go wrong and how bad it would be, then work on the problem together. Andrea’s framing is that QA becomes a co-author of the argument you’ll eventually make to the regulator, not the signature at the end.

Regulations are open to interpretation, and agencies want to see your logic. This is the nuance underneath the credibility question. Any regulation means something slightly different in the context of a specific company and program. Regulators want to see a consistent rationale and sound scientific logic behind a decision, rather than evidence that a box was checked. QA’s combination of regulatory knowledge and technical understanding is exactly what produces that.

The three-batch example shows where credibility actually comes from. Three consecutive validation batches became standard industry practice, and Andrea’s question is where that number came from. FDA guidance has moved away from prescribing it. Now you justify your number based on risk and process understanding. Put three down, and you should expect to be asked why three. Three may be too few, or a statistically justified alternative may be more defensible for your situation. Helping build that logic early is where QA earns its seat, not by enforcing a rule.

What to develop if you want to do this work. Andrea’s list starts with genuine curiosity, an open mind, and the soft skills that show up as “how” rather than “no.” Then scientific fluency, real operational knowledge of what you’re overseeing, statistical and risk analysis literacy, working knowledge of the ICH guidelines and tools like FMEA, and data and digital fluency, including how AI-driven tools can be leveraged, how they need to be validated, and what proper quality oversight of them looks like. Together, those build the trust that gets QA invited to the table.

Design governance so early involvement is the path of least resistance. ICH Q10 already lays out the pharmaceutical quality system lifecycle. Instead of a single late-stage gate, build phase-appropriate risk and analytical checkpoints into the process as programs mature. Practically, that means standing membership in technical discussions rather than only change review boards, explicit decision rights, and scoping the risk level up front. The goal is not an approval step bolted on top. It’s making the earliest possible involvement the easiest thing to do.

Three steps to start tomorrow.

1. Run a baseline quality culture maturity assessment using ISPE’s APQ or PDA’s tool, so you understand where quality actually sits in your development timeline based on evidence rather than anecdote.

2. Pilot the technical partnership model on a single active program, putting QA in the room for QTPP and CQA definition and DOE planning, and running FMEA jointly with the technical team.

3. Then establish QMR metrics with real leadership commitment, using FDA’s voluntary Quality Management Maturity program, and supplement deviation closure counts with leading indicators like right first time, cost of poor quality, cycle time, and root cause closure.

Andrea E. Bell, DHS, MS, MBA, is a Vice President and Global Head of Quality Assurance with more than 25 years in biotechnology, focusing on harmonization, phase-appropriate quality systems, and quality risk management. She’s built quality and CMC organizations across RNA, biologics, and small-molecule programs and has led complex programs from early development through commercialization. She holds a doctorate in global public health with a focus on vaccines from Massachusetts College of Pharmacy and Health Sciences, a master's in quality assurance and regulatory affairs from Temple University, an MBA in international business, and a bachelor's in biology.

Connect with Andrea on LinkedIn here.

Who is The FDA Group?

The FDA Group helps life science organizations rapidly access the industry's best consultants, contractors, and candidates. Our resources assist in every stage of the product lifecycle, from clinical development to commercialization, with a focus on staff augmentation, auditing, remediation, QMS, and other specialized project work in Quality Assurance, Regulatory Affairs, and Clinical Operations.

With thousands of resources worldwide, hundreds of whom are former FDA, we meet your precise resourcing needs through a fast, convenient talent selection process supported by a Total Quality Guarantee.

Here’s why 17 of the top 20 life science firms access their consulting and contractor talent through us:

• Resources in 75 countries and 48 states.

• 26 hours average time to present a consultant or candidate.

• Exclusive life science focus and expertise.

• Dedicated account management team.

• Right resource, first time (95% success).

• 97% client satisfaction rating.

Talk to us when you're ready for a better talent resourcing experience and the peace of mind that comes with a partner whose commitment to quality and integrity reflects your own.

Subscribe to The Life Science Rundown:

Apple | Spotify | YouTube | Web + Others

Check out our newly launched AI-powered QMS audit tool, AICA (the Audit Intelligence Compliance Assistant).