RA/QA News Roll: Late July 2024

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 headline news, Jeff Shuren, the longtime head of FDA’s device division, plans to retire from federal service at the end of this month. As the head of CDRH, Shuren was one of the FDA’s most influential, senior, and tenured regulators. Michelle Tarver, deputy center director for Transformation at CDRH, will serve as interim Director of CDRH. There is currently no information about where Shuren will be headed next. The FDA is currently seeking a replacement. The agency has posted a job opening with a maximum salary of $400,000 per year under its 21st Century Cures Act authority.

Also, the FDA has just revealed its new FY2025 user fee rates for drugs, devices, generic drugs, over-the-counter medicines, biosimilars, and compounding outsourcing facilities. Paid subscribers got our full breakdown:

The FDA's Fiscal Year 2025 User Fees: A Quick Guide

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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 Guidances, Regulations, and Related Documents

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• The FDA has released draft guidance on facilitating the development of drugs for pediatric inflammatory bowel disease. It details recommendations for clinical trials, including study population, design, efficacy, and safety considerations. It emphasizes rigorous scientific approaches and the use of adult study data to inform pediatric drug development programs for treating pediatric ulcerative colitis and Crohn's disease. 💊

• The FDA has issued revised final guidance on Drugs for the Treatment of Partial Onset Seizures, focusing on the extrapolation of efficacy from adults to pediatric patients aged 1 month and older. It provides criteria for formulation development, efficacy, clinical pharmacology, and safety considerations. The guidance supports using data from adult studies to inform pediatric drug development, focusing on achieving similar drug exposure levels and assessing tolerability. 💊

• The FDA has published a new FAQ document addressing the most common questions received in response to its final rule on Laboratory Developed Tests (LDTs). The document also clarifies that LDTs for Highly Pathogenic Avian Influenza will fall under the agency's enforcement discretion approach, meaning they won’t be regulated as in vitro diagnostics. 💉

• The FDA has released revised final guidance on Application User Fees for Combination Products. It outlines the process for determining fees based on whether a single application or separate applications are submitted. It also explains the conditions under which user fee waivers, reductions, or exemptions might apply, particularly under the Prescription Drug User Fee Act (PDUFA) and Medical Device User Fee Amendments (MDUFA). 💊

• The FDA has released a new guidance document that addresses eligibility standards for blood donors, with a focus on athletes whose training regimens result in abnormal blood pressure and pulse readings that would otherwise disqualify them from donating. 💊

• The FDA has released a new final guidance document that provides recommendations on assessing electronic health records and medical claims data to support regulatory decision-making for drugs and biologics. 💊

• Another new final guidance document provides recommendations on managing changes to container closure systems and their components, specifically focusing on glass vials and stoppers. It outlines best practices for ensuring the integrity and safety of these systems when modifications are made. 💊

• A revised CBER policy on regulatory meetings with sponsors of drug or biologics applications includes new information on “converting meeting types.” This latest update provides additional guidance on how to change the type of meeting requested to better suit the regulatory needs and schedules of both CBER and the sponsors. 💊

FDA General

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• In a new FDA blog post, CDER Director Patrizia Cavazzoni and CBER Director Peter Marks discuss plans to establish a new Rare Disease Innovation Hub, also known as "the Hub." This center of excellence will focus on "products intended for smaller populations or for diseases where the natural history is variable and not fully understood." The Hub aims to serve as a "single point of connection and engagement," enhance inter-center collaboration, and advance regulatory science. While CBER and CDER are acting as co-leads, CDRH, the Oncology Center of Excellence, the Office of Combination Products, and the Office of Orphan Products Development will also collaborate. Marks initially previewed the Hub at the Drug Information Association (DIA) annual meeting in late June. Additionally, CDER has announced its intention to hire an Associate Director of Rare Disease Strategy to act as the "director of strategic coalitions" for the Hub. 💊 💉

• In a blog post from FDA Digital Health Center of Excellence (DHCoE) Director Troy Tazbaz and Digital Health Specialist John Nicol, the focus is on a “lifecycle management approach” for artificial intelligence. The post introduces an “AI Lifecycle (AILC) Concept” with a detailed map of “key activities” for each phase of the AI lifecycle. It previews future activities for the DHCoE, including the development of guidance, checklists, and other policies. One guidance document under development by the CDRH is titled “AI/ML software functions: lifecycle management considerations and premarket submission recommendations,” closely aligning with the AILC Concept presented in the blog post. 💉

• The FDA has extended the comment period for its new Platform Technology Designation Program by an additional 30 days. 💊

• An upcoming medical device sterilization town hall meeting will focus on bioburden, bacterial endotoxin, and packaging integrity testing for sterile medical devices. 💉

• The FDA announced that it has renewed the charter of its Pharmacy Compounding Advisory Committee for an additional two years. 💊

• A newly announced Town Hall meeting for CBER’s Office of Therapeutic Products, which regulates cell and gene therapies, will focus on “CMC readiness for late-stage INDs” submitted for cell therapies. CMC stands for chemistry, manufacturing, and controls and refers to the ability to make a product. 💊

• A newly announced virtual workshop on FDA and NIH “regulatory do’s and don’ts” is designed to convey “tips from FDA” related to developing new cancer drugs. 💊

• An on-demand webinar recently focused on the reorganization of CDER’s Office of Pharmaceutical Quality. OPQ Director Michael Kopcha assured that stakeholders should experience "little to no immediate impact" from the organizational changes, explaining that most changes are aimed at "fostering internal improvements." 💊

• A new FDA blog post focuses on tackling the rising burden of common chronic diseases. FDA Commissioner Robert Califf argues that “gaps in evidence generation explain part of the gap between the tremendous growth in effective medical products and our lagging health outcomes” for common chronic diseases. He notes that the FDA is working to “simplify clinical trials and streamline them with routine patient care.” 💊 💉