RA/QA News Roll: Mid December 2024 ❄️
Here's what's happening at FDA and across the broader life science RA/QA space.
In the spirit of giving this holiday season, this RA/QA News Roll is free for all subscribers. From all of us at The FDA Group, happy holidays to you and yours.
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 and the Administration Transition
The New York Times has reported that Aaron Siri, a lawyer known for frequently submitting citizen petitions to the FDA, is being considered for the position of HHS general counsel under Robert F. Kennedy Jr. According to The Times, one of Siri's petitions requested that the FDA revoke its approval of the polio vaccine.
POLITICO reports that Rep. Buddy Carter (R-Ga.), a candidate for chair of the Energy and Commerce Committee’s Health Subcommittee, announced on Thursday his decision to join the Congressional DOGE Caucus. The Health Subcommittee has oversight of the FDA, and Carter’s involvement with the Department of Government Efficiency (DOGE) could increase focus on FDA-related issues within the department.
In an interview with TIME Magazine, President-elect Donald Trump discussed potentially significant changes to childhood vaccination programs, including the possibility of ending some. Trump indicated that he plans to have a "big discussion" with Robert F. Kennedy Jr. about the safety and benefits of vaccines. He expressed concerns about the connection between vaccines and autism. Although open to changing vaccination policies if deemed necessary, Trump predicted that these changes would not be very controversial ultimately. He also mentioned listening to Kennedy on vaccination issues. Regarding access to abortion drugs like mifepristone, Trump noted it was "unlikely" that his administration would restrict access, though he stopped short of confirming that there would be no limitations.
The Senate Committee on Homeland Security and Governmental Affairs has published the latest edition of the Plum Book, detailing the thousands of leadership positions within the federal government that are subject to appointment by the incoming Trump Administration. The publication includes several key positions at the FDA, such as FDA Commissioner, Associate Commissioner for External Affairs, Associate Commissioner for Legislative Affairs, Deputy Commissioner for Policy, Legislation and Internal Affairs, Associate Commissioner for Congressional Appropriations, and Chief Counsel, among other vacancies. The Plum Book provides comprehensive accounting to aid in understanding which roles may have new appointees. Additionally, a website has been set up to facilitate easier searching through this data.
The New York Times reports on remarks from Eli Lilly CEO David Ricks, in which he stated that the company would actively defend the FDA against potential changes proposed by incoming regulators. Ricks emphasized the importance of maintaining the agency’s role in ensuring the safety and efficacy of medicines.
In a new op-ed published in The Wall Street Journal, FDA Commissioner Robert Califf responds to an earlier op-ed by DOGE co-leads Elon Musk and Vivek Ramaswamy. Califf urges DOGE to “work with the bureaucracy, not against it,” emphasizing that government regulators often possess the best insights for reforming regulatory processes. He argues that collaboration, rather than opposition, is key to achieving meaningful improvements.
FDA Guidances, Regulations, and Related Documents
The FDA has issued a new guidance document titled “Standardized Format for Electronic Submission of Marketing Application Content for the Planning of Bioresearch Monitoring [BIMO] Inspections for Center for Drug Evaluation and Research (CDER) Submissions.” The guidance outlines how companies should submit standardized data to the FDA to assist in planning and conducting BIMO inspections. 💊
The ICH, a global regulatory harmonization group that includes the FDA, has published a new Q&A document providing updated guidance on implementing pharmaceutical quality systems and quality-by-design (QbD) principles. This revised document clarifies key aspects of quality system implementation to help manufacturers ensure consistent product quality throughout the drug development and manufacturing process. 💊
The FDA has released an updated Manual of Policies and Procedures (MAPP) regarding assessing the “Appropriate Net Container Content for Injectable Drug and Biological Products” by the OPQ. The update reflects changes made following a major reorganization of OPQ and outlines the agency's approach to ensuring that injectable drug and biological products meet appropriate container content requirements. 💊
The FDA has released its long-awaited revised draft guidance on the accelerated approval program. The guidance primarily focuses on the agency's new accelerated withdrawal authorities. Still, it provides limited discussion on its authority to require confirmatory trials to be “underway” before accelerated approval or within a specified time frame afterward. The FDA stated that these concerns will be addressed in a separate, forthcoming guidance document. 💊
The FDA has released a new report examining the risks and benefits of non-device software functions. The report, required annually by Congress, follows the 21st Century Cures Act of 2016, which removed the FDA's authority to regulate certain types of software as medical devices. While no longer regulating these software functions, Congress tasked the FDA with monitoring developments in the space and reporting on potential impacts. 💉
The FDA has released its 2025 list of generic drug science and research priorities, which remain largely consistent with previous years. Notably, this year’s list includes a new focus on artificial intelligence. The FDA’s Office of Generic Drugs aims to utilize AI to “improve the efficiency and consistency of scientific assessments and advice” as part of its efforts to enhance the generic drug review process. 💊
The FDA has released its long-anticipated guidance on predetermined change control plans (PCCPs) for AI-enabled device software functions. PCCPs serve as roadmaps outlining how a device may evolve after approval, allowing the FDA to prospectively approve changes within a defined set of criteria. This is particularly valuable for AI-enabled devices, which may change as new data is used to train the AI. Alongside the release, the FDA has announced an upcoming webinar to provide further details and clarify the guidance. 💉
FDA General
The FDA’s CDER has announced the creation of the CDER Center for Real-World Evidence Innovation (CCRI). The new center is tasked with coordinating, advancing, and promoting the use of real-world data (RWD) and real-world evidence (RWE) in regulatory decision-making within CDER. As the center begins its operations, it is in the process of recruiting a new leader following the upcoming retirement of John Concato, CDER’s longtime lead on RWE policy. Patrizia Cavazzoni, the Director of CDER, described CCRI as a “collaborative core for innovation” aimed at ensuring consistency and transparency in CDER’s handling of RWD and RWE topics. A webpage for the center has been set up. 💊
The Drug Information Association (DIA) has announced that its Advanced Therapies Community and Regulatory Policy and Intelligence Community will host Nicole Verdun, Director of the Office of Therapeutic Products (OTP), at the CBER for an online fireside chat on January 7, 2025. OTP is responsible for regulating cell and gene therapies. 💊
The CDER recently published a blog post/podcast discussing the agency's focus on improving data quality through centralized statistical monitoring. Dr. Paul Schuette and Tina Wang discuss the crucial role of centralized statistical monitoring in improving data quality and integrity within clinical trials, as detailed in FDA's guidance. They advocate for using advanced technologies like the SMART software and electronic records that facilitate efficient remote monitoring. They also highlight the challenges and limitations of current monitoring tools, including data inconsistencies and the impact of external factors like the pandemic, while suggesting future directions involving artificial intelligence and decentralized clinical trials to enhance monitoring efficacy tailored to specific trial needs. 💊
In an interview, FDA Commissioner Robert Califf emphasized the challenges of AI quality oversight, particularly in the postmarket phase. He noted the necessity for continuous validation of AI models in their operational environments. Califf expressed concern that no U.S. health system currently has the capability to perform such validations, suggesting the likely existence of faulty algorithms. He highlighted the urgent need to develop a new approach to information technology that supports effective validation processes. 💉
The FDA, in collaboration with the Journal of the American College of Cardiology (JACC), has launched an initiative to identify "urgent evidence gaps" in clinical research priorities, particularly in the field of cardiovascular health. According to the initiative's webpage, the FDA is seeking input from clinicians on "pressing questions" they have concerning the use of FDA-regulated products and their effects on cardiovascular health. The initiative aims to collect responses by February 14. Notably, FDA Commissioner Robert Califf, who is one of the leads on this project, will have left the agency by the deadline for submissions. 💊
The FDA has updated a webpage that tracks its generic drug approvals for fiscal year 2025. 💊
In an interview with POLITICO, Peter Marks, the FDA’s top biologics official, reflected on lessons learned from the Covid-19 pandemic and how they can inform the U.S. response to an emerging avian influenza outbreak. One key takeaway Marks highlighted is the ineffectiveness of mandates. “We know that mandates, they just don’t work,” he said. “What we’ve learned is, we have to convince people based on the evidence in language that they can understand.”
Governing
The Senate has approved the FDA Modernization Act 3.0, which mandates the FDA to issue an interim final rule for implementing the FDA Modernization Act 2.0. The earlier legislation, FDAMA 2.0, stipulated that preclinical animal testing is not required in all cases before human studies can commence. The newly passed FDAMA 3.0 specifies that certain sections within Chapter 21 of the Code of Federal Regulations, which contains FDA regulations, must be updated at a minimum.
Sen. Ron Johnson (R-Wisc.) has introduced new legislation to clarify that agencies within HHS, potentially including the FDA, do not have the authority to regulate the practice of medicine. While the full text of the legislation has not yet been released, the bill's title suggests it may specifically target the FDA's regulatory powers.
The Eliminate Useless Reports Act of 2024, having passed both the House and Senate, directs federal agencies to identify "outdated or duplicative reporting requirements" in their annual budget justifications. The legislation also requires agencies to recommend whether to terminate, modify, consolidate, or reduce the frequency of such reports or plans. This act is particularly relevant to the FDA, which is mandated to produce numerous annual reports, the usefulness and insights of which vary significantly.
Sens. Tom Cotton (R-Ark.) and Tim Kaine (D-Va.) introduced the Protecting Our Essential Medicines Act. The legislation would require HHS to maintain a list detailing the country of origin for certain critical drugs marketed in the United States. It also calls for creating an interagency task force, including the FDA, CDC, and defense and intelligence agencies, to manage these lists. The bill outlines two types of lists: one publicly available list and a confidential list specifically identifying “drugs that are produced exclusively in China.”
Compliance and Enforcement
ProPublica has reported that the FDA has not inspected Glenmark Pharmaceuticals, an Indian drug manufacturer. The company has issued seven recalls for the same issue: pills that did not dissolve properly. This flaw could potentially cause dangerous spikes in potassium levels. Glenmark has acknowledged that at least three deaths and three hospitalizations have been linked to this problem. 💊
The FDA has issued a new warning regarding Intercept Pharmaceuticals’ drug, Ocaliva, citing its association with cases of serious liver injury. The agency recommends that patients using Ocaliva undergo liver monitoring tests to detect any worsening liver function. 💊
The DOJ has issued a press release announcing the conviction of two biotech CEOs for misleading investors about the regulatory status of their investigational products during the Covid-19 pandemic. The CEOs were found guilty of providing false information to investors regarding the progress and approval status of their products, resulting in significant financial losses. 💊
Reuters reports on a 2023 FDA inspection of Elon Musk's Neuralink, which identified “objectionable” conditions in its animal laboratories. While the article presents the information as new, the Form 483 detailing the findings has been publicly available for some time. The inspection issues were specifically related to water sample collection for quality testing. Reuters notes that the FDA classified the findings as requiring voluntary action rather than the more serious designation of “Official Action.” 💉
The FDA has issued a warning letter to Applied Therapeutics following a bioresearch monitoring inspection of the company’s clinical trial site, citing “objectionable conditions.” Key concerns include deleting electronic data and associated audit trails by a third-party data vendor contracted by the company. This occurred after the FDA had requested access to specific data, limiting the agency’s ability to verify records for 47 trial enrollees. Additionally, the trial administered an incorrect dose of the investigational product, leading to the under-dosing of patients.
The SEC has announced a settlement of charges against Houston-based biotherapeutics company Kiromic BioPharma and two former executives for failing to disclose critical information about its interactions with the FDA. Specifically, the FDA had placed Kiromic's drug candidates on clinical hold, an order delaying the proposed clinical investigations. The SEC found that Kiromic did not disclose these clinical holds in its SEC filings, investor roadshow presentations, or due diligence calls ahead of its offering, despite acknowledging the hypothetical risk of a clinical hold and its potential negative impact on the company’s business.
Other
The Association for Accessible Medicines (AAM) has published a blog post urging Congress to pass the Increasing Transparency in Generic Drug Applications Act. This legislation is designed to end practices by brand-name drug manufacturers that delay generic competition, thereby enabling them to maintain higher prices for longer periods. The proposed act would grant generic drug companies access to additional information necessary to demonstrate the "sameness" of a generic drug to its reference-listed brand-name counterpart. Although the legislation has previously advanced in the House of Representatives, it has not yet been passed by the full House. 💊
Friends of Cancer Research has released a press release highlighting new regulatory research on artificial intelligence models used to assess HER2 in breast cancer whole-slide images. The findings will be discussed in detail at an upcoming public meeting hosted by the organization. 💉
A recent press release announces a study finding that a laser pulse oximeter can accurately estimate blood oxygenation levels in individuals with darker skin pigmentation. This is significant because the FDA has noted that conventional pulse oximeters often perform less effectively in people with darker skin. In response to these discrepancies, the FDA is actively developing a guidance document that will outline enhanced non-clinical and clinical performance testing for such devices. 💉
The industry trade group BIO has requested that the FDA extend the comment period on the accelerated approval draft guidance document, released last week, by an additional 30 days. Such extensions are typically granted by the FDA for modest requests like this. 💊
Friends of Cancer Research has announced a meeting on “advancing the future of diagnostics and regulatory innovations,” scheduled for February 4, 2025. The event will feature Michelle Tarver, the new director of the FDA’s CDRH. 💉
AdvaMed has released a new white paper addressing third-party modifications of interventional X-ray equipment, arguing that such modifications can “pose unforeseen risks for patients and providers.” The paper aligns with AdvaMed’s longstanding opposition to third-party servicing of medical devices, maintaining that only manufacturers and their authorized designees should be permitted to service FDA-approved products to ensure their continued safety and efficacy. 💉
Catch up on our recent posts:
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 over 2,500 resources worldwide, over 225 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 contract and FTE talent through us:
Resources in 62 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.