RA/QA News Roll: Mid February 2025
Here's what's happening at FDA and across the broader life science RA/QA space.
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.
In response to subscribers' requests for news from other agencies, we’ve added a section covering recent EMA happenings.
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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 federal government began firing tens of thousands of federal employees last Thursday who were still in their probationary periods, which can last up to two years and includes recently promoted staff. While official details on the impact on the FDA remain limited, multiple news reports indicate that HHS agencies started to be affected on Friday. However, the FDA’s workforce may be partially shielded from the layoffs due to two key factors: many of its employees are funded through user fees, and a significant portion work on critical public health matters. Under a recent executive order, such staff are theoretically exempt from certain reduction-in-force actions.
The Senate officially confirmed Robert F. Kennedy Jr. as Secretary of HHS yesterday morning. According to POLITICO, Kennedy and his team have already outlined a list of priority actions and are expected to move swiftly in implementing changes. In support of his agenda, Congress' Make America Healthy Again (MAHA) Caucus is preparing legislation to advance his initiatives. Notably, the confirmation process saw little opposition from healthcare companies and public health organizations, many of whom remained silent, believing they had little chance of blocking his appointment and hoping to preserve credibility for future policy negotiations. With Kennedy confirmed, legislators can now move forward with scheduling hearings for other key HHS leadership positions. This includes President Donald Trump’s nominee to lead the FDA, Marty Makary.
Despite widespread concern among life sciences professionals over the potential policy and practical impacts of FDA staffing reductions, few organizations have publicly defended the agency. However, earlier this week, the advocacy group Friends of Cancer Research (FOCR) issued a statement warning that a “weakened FDA and NIH will hurt patients, science, and the economy.” “Fewer resources mean longer wait times for new drugs, slowing down breakthroughs for cancer, rare diseases, and other life-threatening conditions,” the group wrote. “[…] Smart investments—not capricious cuts—are necessary to keep America at the forefront of innovation and ensure that patients continue to receive the safest, most effective treatments.” It remains to be seen whether other public health organizations will follow FOCR’s lead and speak out.
The National Treasury Employees Union (NTEU), along with other government employee unions, has filed a lawsuit in the U.S. District Court for the District of Columbia challenging the Trump administration’s efforts to restructure federal agencies and reduce staff without congressional approval. The lawsuit argues that these actions violate the Constitution by bypassing Congress’ legislative authority and that the administration violates federal laws governing due process for reduction-in-force (RIF) decisions.
Steven Grossman, former head of the Alliance for a Stronger FDA, has provided a detailed primer on the budget reconciliation process and its potential effects on the FDA. His analysis offers insights into how funding decisions could shape the FDA’s operations and regulatory priorities in the coming months.
Under the order, agencies will be permitted to hire only one employee for every four who leave federal service, with some exceptions for public safety roles. The policy could create pressure on FDA staff considering departure, as vacancies may remain unfilled, a concern already circulating among agency employees.
Additionally, the order requires agencies to submit a report within 30 days assessing whether any divisions or subcomponents should be eliminated or consolidated.
Following a court order, the FDA has reinstated several guidance documents and webpages that were previously removed after an executive order was published. Among the restored materials are the FDA’s Diversity Action Plan and its guidance on the study of sex differences in the clinical evaluation of medical products, which includes numerous references to the term “gender.” While it is unclear how long these documents will remain online, they are currently accessible once again.
Multiple reports indicate the White House is drafting an executive order that would require the agency to cut a “certain percentage” of its workforce. According to The Wall Street Journal and The Washington Post, the proposed cuts are under discussion, while BioCentury reports that they could halve FDA staff—a drastic reduction, especially considering that many FDA employees are not taxpayer-funded but instead supported by industry-paid user fees designed to ensure efficient medical product reviews. However, an important caveat remains: The White House has denied these plans exist, and they could change substantially before being finalized—if they move forward at all.
A WIRED Magazine report yesterday revealed that Marty Makary, President Donald Trump’s nominee for FDA commissioner, and Jay Bhattacharya, Trump’s nominee for NIH director, are both members of the editorial board for the newly established Journal of the Academy of Public Health. The journal’s board includes several scientists who were critical of the U.S. government’s response to the COVID-19 pandemic, suggesting a potential alignment with viewpoints that challenged mainstream public health policies during the crisis.
FDA General
For the first time in years, the FDA’s CBER has updated its webpages with new data, revealing a significant increase in Regenerative Medicine Advanced Therapy (RMAT) designations in fiscal year 2024. According to the newly published data, CBER received 59 RMAT designation requests and approved 43—more than double the previous record of 18 designations granted in a single year. The update marks a rare publication from CBER under the Trump administration, highlighting the growing interest and approval rate for regenerative medicine therapies. 💊
Former FDA Commissioner Robert Califf has quickly transitioned to a new role, rejoining Duke Health as a faculty member in its Division of Cardiology. In a quietly published blog post last week, Duke Health announced that Califf will focus on a variety of key issues, provide guidance to colleagues, and support younger faculty as they navigate their early careers. His return marks a continuation of his long-standing involvement in academia and medical research.
Judge Tanya Chutkan of the U.S. District Court for the District of Columbia ruled against Vanda Pharmaceuticals in its lawsuit against the FDA and Teva Pharmaceuticals. Vanda had alleged that the FDA violated federal law by approving a generic version of its drug Hetlioz, used to treat non-24-hour sleep-wake disorder. The judge found that Vanda failed to sufficiently demonstrate that the FDA’s actions violated the Administrative Procedure Act. This case offers notable insights for those following generic drug litigation—particularly regarding braille labeling requirements. 💊
Pharmaceutical industry trade groups PhRMA and BIO have weighed in on the FDA’s recent workshop on patient-focused drug development, offering recommendations for improving the agency’s processes.
PhRMA identified several opportunities to streamline the submission and evaluation of patient experience data (PED), as well as FDA’s communication regarding its use in regulatory decisions. “PhRMA strongly encourages CDER and CBER to take steps to clarify expectations for reviewers around how PED should be evaluated in regulatory review and decision-making and subsequently discussed in review documents to mitigate inconsistencies in documents developed by different review divisions,” the group wrote.
BIO suggested that the FDA develop a repository of patient-focused drug development (PFDD) case studies to highlight best practices and lessons learned in PED submissions. The organization also recommended that the FDA provide clearer guidance on submitting PED in a tabular format, which was noted at the workshop as a frequent source of confusion for sponsors.
As the U.S. moves to withdraw from the WHO, STAT asks the key question: Will FDA officials participate in an upcoming meeting to determine which influenza strains should be targeted in next year’s vaccines? The FDA traditionally plays a crucial role in these discussions as one of three essential regulatory labs with a vote. Despite the uncertainty surrounding U.S. involvement, WHO has reportedly still extended an invitation to FDA representatives. However, it remains unclear whether they will attend. 💊
This week, the FDA received a petition urging it to work with Takeda Pharmaceuticals to ensure continued access to Soticlestat, a drug used to treat CDKL5 Deficiency Disorder (CDD), Lennox-Gastaut Syndrome (LGS), and Dravet Syndrome (DS). “This decision jeopardizes the health and stability of patients for whom Soticlestat has been a life-changing therapy, offering unparalleled seizure control and an improved quality of life,” the petitioner wrote. The petition calls on the FDA to explore pathways that would allow continued patient access to the drug, either through the New Drug Application program or other accelerated regulatory approval mechanisms. While the FDA cannot compel Takeda to continue manufacturing Soticlestat, it could take steps to facilitate production by other companies. 💊
A new petition from the law firm Buchanan Ingersoll & Rooney urges the FDA to "strictly apply" its product-specific guidance for thioguanine tablets (marketed as Tabloid), a chemotherapy drug used to treat acute nonlymphocytic leukemias. The petition argues that an in vivo bioequivalence study should be required before approving a generic version, citing concerns over myelosuppression, a potentially life-threatening side effect. Tabloid is marketed by Waylis Therapeutics. (The FDA regularly receives petitions from brand-name drug sponsors or their attorneys requesting that the agency impose specific requirements before approving generic versions of their drugs. While the FDA almost always rejects these petitions, they can still influence how regulators scrutinize certain issues.) 💊
The FDA issued a warning advising diabetes patients who rely on smartphone alerts for their medical devices to be cautious, as software updates may disable or mute these alerts, potentially leading to serious health risks. “In some cases, missing these alerts may have contributed to serious harm, including severe hypoglycemia, severe hyperglycemia, diabetic ketoacidosis, and even death,” the agency stated. Because digital health products often run on devices not originally designed for medical use, the FDA recommends that patients turn off automatic operating system updates to prevent unexpected changes and regularly check smartphone alert settings at least once per month to ensure notifications remain active. 💉
Governing
The House of Representatives’ budget resolution indicates that Republican lawmakers are pursuing significant government spending cuts, potentially impacting the FDA. By March 27, congressional committees must submit recommendations on legal changes to meet targeted savings. The House Energy and Commerce Committee (E&C)—which oversees the FDA—has been assigned the most significant reduction target of any committee, tasked with identifying $880 billion in savings between 2025 and 2034. To achieve these cuts, E&C will likely focus on reductions to Medicaid spending, a move that could have ripple effects on healthcare funding and regulatory agencies like the FDA.
The Senate Health, Education, Labor and Pensions (HELP) Committee, which oversees FDA-related matters, has finalized its subcommittee assignments. Sen. Roger Marshall (R-Kan.) will serve as chair of the Primary Health and Retirement Security Subcommittee, with Sen. Ed Markey (D-Mass.) as the ranking member. The Republican members of the subcommittee are Sens. Paul, Collins, Murkowski, Mullin, Hawley, Banks, Husted, and Moody, while the Democratic members include Sens. Murray, Baldwin, Murphy, Hassan, Hickenlooper, Kim, and Rochester.