RA/QA News Roll: Mid October 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.
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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
The FDA has released a new final guidance document on electronic systems, electronic records, and electronic signatures in clinical investigations. 💊 💉 Paid subscribers can read our full breakdown and recommendations for alignment:
The CDRH has launched a new webpage explaining its Total Product Life Cycle Advisory Program (TAP), which "facilitates engagement with non-FDA parties." TAP is designed to assist companies, particularly small businesses, by offering early, frequent, and strategic interactions with the FDA to help navigate the regulatory process for product commercialization. The program also facilitates communication with non-FDA experts. The webpage outlines TAP's role in engaging with patients and addressing issues related to clinical evidence development, clinical practice, new technology adoption, and reimbursement. Additionally, it includes a list of organizations that have agreed to be listed as TAP participants on the FDA’s website. 💉
The FDA has issued its FY 2025 Broad Agency Announcement (BAA) for Advanced Research and Development of Regulatory Science. Posted annually, the BAA outlines key areas of interest for the FDA in regulatory science, focusing on developing tools that can enhance the agency's ability to regulate medical products. This document provides valuable insights into the FDA’s priority focus areas and the problems it aims to address in the coming year. 💊 💉
The FDA’s Office of Women’s Health has released an updated “Research Roadmap” outlining seven priority areas for research deemed "essential." These priorities include studying how sex differences may impact medical product safety, improving methodologies to assess the safety and efficacy of FDA-regulated products, developing new biomarkers to detect and predict the severity and progression of conditions in women, and advancing methods and tools that consider sex and gender differences when evaluating new technologies. 💊
The CDRH has released its updated FY 2025 guidance agenda, which includes two lists of guidance documents planned for publication or finalization in 2025. The “A-list” features documents that are set for publication, while the “B-list” includes documents that may be published “as resources permit.” Additionally, an “Under Construction” list highlights guidances that are in progress but face inconsistent resources or have been deprioritized. Notable new guidances include those on labeling in vitro diagnostics, an enforcement discretion policy for certain Laboratory Developed Tests addressing unmet needs, AI-enabled device software functions, a revision of the device software functions and mobile medical applications guidance, and clinical evidence considerations for digital mental health treatment devices. 💉
FDA General
The FDA has launched its reorg. The website is being updated to reflect this reorganization. Key changes observed so far include the renaming of the FDA’s medical countermeasures office to the Office of Regulatory and Emerging Science, which will operate under the Office of the Chief Scientist. Additionally, the Office of the Chief Medical Officer (OCMO) has launched a new webpage and introduced several new suboffices, including the Office of Orphan Products Development. Moreover, the Office of Regulatory Affairs (ORA) has been renamed the Office of Inspections and Investigations (OII). The FDA is also updating its Staff Manual Guides (SMGs). 💊 💉
The FDA has announced new exemptions from certain provisions of the Drug Supply Chain Security Act, which are set to take effect next month. Earlier this summer, the FDA granted exemptions related to small dispensers and invited other trading partners to submit exemption requests by early August. Now, the agency has expanded these exemptions, specifically under the "enhanced" provisions of Section 582. Manufacturers and repackagers will have exemptions extended until May 2025, wholesale distributors until August 2025, and dispensers until November 2025. These exemptions are available only to entities that have either completed or made documented efforts to complete data connections with their immediate trading partners but still face data exchange challenges. 💊
The CDRH has expanded the capabilities of its Customer Collaboration Portal to accept and track new submission types. As of Nov. 1, device firms can now submit their Small Business Determination (SBD) requests electronically through the Portal, and all SBD requests must be submitted in this way. SBD designation allows firms to qualify for reduced user fees. The Portal’s Progress Tracker, known as the “Pizza Tracker,” has been expanded to include Premarket Approval and PMA Panel-Track supplements. Sponsors can now upload submissions and view their application status and milestones in near real-time. This marks the first major expansion of the Pizza Tracker since it began tracking new 510(k) submission types in 2022, part of the FDA’s commitments under the most recent device user fee reauthorization (MDUFA V). 💉
The FDA has posted its new Annual Forecast for Planned Monograph Activities, which outlines the monographs the agency intends to update or publish over the next three years. The forecast includes eight FDA-initiated proposed orders and one request for data on anticaries test methods. However, the forecast is nonbinding. 💊
A new Federal Register notice signals that the FDA plans to study the promotion of prescription drugs within a talk show format. The agency's Office of Prescription Drug Promotion (OPDP) aims to understand how "native advertising" of drugs impacts consumer comprehension of a drug's benefits and risks. The FDA also noted that it has recently issued compliance letters to pharmaceutical companies for misleading promotion of prescription drugs in talk show settings. 💊
A transcript from the recent FDA webinar covers labeling requirements for in vitro diagnostic products, including Laboratory Developed Tests (LDTs). The recorded webinar is available here. Understanding LDT labeling is critical for companies as the FDA moves to implement its final rule declaring that LDTs are considered IVDs. A slide deck from the webinar is also available. 💉
The FDA has announced that it has awarded seven new clinical trial grants under its Orphan Products Grants Program. The funded projects include studies of treatments for Cushing disease, microcystic lymphatic malformations, RAF dimer-driven thyroid cancer, pediatric aplastic anemia, retinoblastoma, Leber congenital amaurosis 5 (LCA5), and relapsed myeloid malignancies following allogeneic stem cell transplantation. 💊
The FDA has updated its list of potential signals of serious risks and new safety information identified between April and June of this year. These signals are updated quarterly, though on a significant lag. Among the drugs being evaluated, several cystic fibrosis medications are under review for their potential link to "increased intracranial pressure disorders," and several insomnia drugs are being assessed for "paradoxical drug reaction," which involves increased hyperactivity at the onset of sleep. Singulair is also being evaluated for a potential association with psychiatric disorders. 💊
At the recent Meeting of the Science Board to FDA, Commissioner Robert Califf outlined his top priorities for the agency, likely in his final months as commissioner. Califf provided "a broad overview" of his vision, emphasizing overarching priorities that impact all areas of the FDA, including misinformation, evidence generation, supply chain, and information technology issues. His No. 1 outstanding priority was "Advisory Committees," following recent efforts to reform the committee system. Addressing questions from the Science Board, Califf acknowledged widespread mistrust in government institutions, noting: "I don’t think the slide with FDA, if you look at public perception, is worse than the other agencies – but also it’s not better." 💊 💉