The FDA Just Released a Ton of Guidance Documents
The agency is kicking off 2025 with a trove of new guidance just weeks before the new administration takes over. Here's a handy reference to what just dropped.
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We’re glad to be back in your inboxes after the holidays. Today, we have a special update for you.
As President-elect Donald Trump’s administration prepares to take over in two weeks, the FDA issued a huge batch of new draft and final guidance documents on Monday. The guidances address various topics, including the reliable application of AI in drug development and the criteria for determining when a confirmatory trial officially begins for accelerated approval.
Typically, the FDA stays pretty quiet during the holiday season, sharing only approvals. However, this year was a little different. We can’t remember when 20+ were released on the same day. Over the next few weeks, we’ll analyze and break these down for paid subscribers.
In the meantime, here’s a handy list of what the FDA just released so you can pick through it yourself with a few brief summaries of some of the most notable.
Using AI to support regulatory decision-making for drugs and biologics —The FDA has presented a 23-page draft detailing a new “risk-based credibility assessment framework” aimed at assisting drug manufacturers in validating the reliability of AI model outputs when used to generate data for agency decisions. This draft includes six examples of possible AI applications, such as decreasing reliance on animal studies, combining information from multiple sources like clinical trials and genetic databases, enhancing companies’ insights into disease manifestations, and analyzing extensive data sets to establish clinical trial endpoints or evaluate outcomes.
Considerations for complying with 21 CFR 211.110 — A new draft guidance from CDER, CBER, and the Center of Veterinary Medicine outlines considerations for ensuring batch uniformity and drug product integrity, emphasizing a scientific and risk-based approach to manufacturing controls. It introduces flexibility in in-process sampling and testing while promoting the integration of advanced manufacturing technologies, such as continuous manufacturing and process models, to enhance drug quality and supply chain reliability. The guidance highlights the importance of robust process monitoring, in-line testing, and quality control to maintain a state of control and ensure consistent product quality. It also addresses the challenges of relying solely on process models and encourages manufacturers to combine these with in-process testing for comprehensive control strategies. The FDA advocates early collaboration with regulatory teams to develop innovative approaches that modernize pharmaceutical manufacturing and maintain compliance.
Accelerated approval confirmatory studies — For a few years now, the FDA has been clear about its intention to expedite confirmatory trials for accelerated approvals. Delays in these trials can leave patients relying on therapies that offer limited or no clinical benefit. In a new 9-page draft guidance, the agency states that a drug will not be cleared for use if a confirmatory trial isn’t initiated before an accelerated approval. Additionally, the guidance indicates that when the FDA feels continued enrollment and retention challenges may arise post-marketing, enrollment completion before approval might be required. For development programs focused on rare diseases, where a sponsor is extending a trial and utilizing a surrogate or intermediate clinical endpoint, the FDA noted that such a trial would be considered underway as long as it is expected to finish in a timely manner.
Considerations for using tissue biopsies in clinical trials — The FDA has laid out specific recommendations for industry, investigators, and IRBs to ensure the ethical and practical use of biopsies for both adults and children. The guidance emphasizes a risk-benefit framework, requiring biopsies only when essential for trial objectives, such as determining participant eligibility, evaluating primary endpoints, or understanding treatment responses while suggesting optional biopsies for exploratory or future research purposes to reduce participant burden. For pediatric trials, the agency outlines additional safeguards, with stricter thresholds for non-therapeutic biopsies to ensure minimal risk or only a minor increase over minimal risk unless a direct clinical benefit is anticipated. The guidance also stresses the importance of robust informed consent practices, transparency in protocol design, and early collaboration with regulatory bodies to minimize risks and protect participant rights.
Final guidance on communications of unapproved/off-label uses — The agency finalized a 29-page guidance detailing its enforcement policy regarding the communication of scientific information about unapproved uses of approved medical products to healthcare providers. In this final version, the FDA noted revisions to its draft recommendations for source publications, offering more specificity and examples to better illustrate them. Additionally, the language concerning presentation considerations was clarified, and an extra example was included for further understanding.
The FDA’s CBER released five new draft guidance documents on reducing the risk of transmission of hepatitis B virus, hepatitis C virus, HIV, disease agents associated with sepsis, and mycobacterium tuberculosis in human cells, tissues, and cellular and tissue-based products (HCT/Ps).
Another CBER draft focuses on recommendations for determining the eligibility of HCT/Ps donors.
Here are quick links to the other guidances:
Evaluating sex-specific and gender-specific data in device clinical studies
Study of sex differences in the clinical evaluation of medical products
Validating IVDs for emerging pathogens during public health emergencies
Developing drugs and biological products for weight reduction
Talk to us if you need expert support understanding or operationalizing any of the policies in these guidances.
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