RA/QA News Roll: Late June 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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A couple of headlines we wanted to call out first:
The FDA’s recent warning that "clinical and bioanalytical studies conducted by Synapse Labs are unacceptable." This contract research organization, based in India, was found to have significant data integrity concerns. As a result, the FDA is mandating that studies conducted by Synapse Labs be repeated by the companies that sponsored the research. The FDA has been aware of these concerns since at least February, and the European Medicines Agency issued a similar public warning to the industry in March 2024. This directive affects hundreds of products. The FDA has requested that companies with affected products respond within 30 days with their plans to either re-conduct studies and resubmit data within the next year or voluntarily withdraw the affected drugs from the market.
The Supreme Court has overturned the Chevron Doctrine, a 1984 legal decision that allowed regulatory agencies significant discretion in interpreting ambiguous or unclear legislation to create regulations. This decision could potentially empower life sciences companies to challenge FDA regulations, including those like the Laboratory Developed Test regulation, in instances where congressional intent or clarity is lacking.
Here are some key items from the past couple of weeks.
💊 = Pharma, biologic, and biotech.
💉 = Medical device, medtech, and diagnostics.
FDA Guidances, Regulations, and Related Documents
The White House has recently released the FDA’s Diversity Action Plan draft guidance document following an extensive review process. This revised document represents a comprehensive rewrite of the agency’s previous draft guidance on the topic and is double the size in terms of page count. Notably, the guidance states that waivers from the requirements for submitting a Diversity Action Plan will only be granted in exceptional cases, whether in full or in part. Read our guidance breakdown here. 💊
The FDA has updated a table outlining when specific oversight applies to various types of in vitro diagnostics under its final Laboratory Developed Test (LDT) regulation. (Contact us if you still need to access consultants to plan and manage your compliance transition following the FDA’s final rule to end LDT enforcement discretion. Qualified consultants are already in high demand.) 💉
The FDA has issued a new Small Entity Compliance Guide designed to assist small businesses in adhering to CDRH's LDT final rule. This guide simplifies the final rule's requirements without introducing additional information. Its primary aim is to provide a more accessible format for understanding and implementing the regulatory guidelines. 💉
The FDA has released new guidance focusing on considerations for demonstrating interchangeability with a reference product. A significant update is the agency's shift away from requiring switching studies for all interchangeable determinations, reflecting an evolved scientific approach. More on that here. 💊
The FDA has released a final guidance document detailing its assessment of goal date decisions under the Generic Drug User Fee Amendments. GDUFA Dates represent target deadlines for FDA decisions on applications, aligned with commitments outlined in the GDUFA Commitment Letter. A critical aspect of setting these dates involves evaluating the readiness of generic drug manufacturing facilities for inspection. The final guidance outlines the FDA's process for determining facility readiness. 💊
The FDA has updated its guidance on circumstances that constitute delaying, denying, limiting, or refusing a drug or device inspection. Initially published a decade ago following the FDA Safety and Innovation Act (FDASIA), which originally applied only to drug-related inspections, the latest guidance now also encompasses devices. 💉 Read our in-depth guidance breakdown here:
The FDA has issued a new report focusing on enhancing clinical study diversity, specifically centered on a workshop conducted in November 2023. The report serves as a summary of the meeting and clarifies that it does not offer guidance or represent the FDA's current stance on the subject. 💊
Digital Health Center of Excellence Director Troy Tazbaz has published a new blog post highlighting AI's potential. The post outlines upcoming topics that will delve deeper into AI concepts, including standards, best practices, and operational tools; quality assurance in laboratories; transparency and accountability; and risk management for AI models in healthcare. 💉
The CDRH has officially released its updated List of Recognized Standards for medical devices through a Federal Register announcement. This update solidifies the changes previously mentioned in their newsletter a few weeks ago. 💉
The FDA's Office of New Drugs has released a new report summarizing reforms and changes implemented in its regulatory program for new drugs since 2017. Often overlooked structural changes are detailed in the report and an accompanying blog post. The report also outlines upcoming enhancements, such as an Information Request-focused dashboard aimed at improving lifecycle tracking and search capabilities within the NDRP. 💊
A new version of Form FDA 3989, which pertains to the PMR/PMC Annual Status Report for Drugs and Biological Products, has been released. This form is specifically designed to document post-marketing requirements and commitments associated with these products. 💊
FDA General
The FDA has announced a new webinar on July 23 focused on its Office of Trade and Global Partnerships. This office manages issues related to international trade, mutual recognition agreements, and information sharing with global counterparts. 💊 💉
The FDA has approved significant modifications to the Risk Evaluation and Mitigation Strategies (REMS) for two CAR-T products, TECARTUS and YESCARTA, developed by Kite Pharma, a subsidiary of Gilead Sciences. The updates allow the removal of a previous warning concerning the management of cytokine-release syndrome (CRS) and neurological toxicities. According to the FDA, these changes aim to alleviate the burden on healthcare providers who administer these products. 💊
The FDA has announced plans to hold a meeting on July 22 focused on best practices for meeting management under the PDUFA VII user fee program. The workshop aims to discuss various topics, including the submission of meeting requests, efficient time management, finalizing meeting agendas, developing and submitting meeting background packages, and lessons learned from the COVID-19 pandemic, including the use of virtual meeting platforms. 💊
The FDA has published a request for input regarding non-device software functions. Due to federal law limitations, certain device functions cannot be regulated by the FDA. The agency is developing a report to monitor these functions and is seeking public input on the matter. 💉
According to Tamy Kim from FDA's Oncology Center of Excellence, The Pink Sheet reports (paywalled) the FDA's new diversity action plan guidance restricts waivers from requirements to "very rare" cases. This marks one of the initial insights into the approach for granting waivers under the diversity plan. 💊
An interview with Michelle Tarver, CDRH’s deputy director for transformation, discusses the agency's Home as a Health Care Hub Initiative. Read more about the initiative here. 💉
CDER Director Patrizia Cavazzoni and CBER Director Peter Marks authored a blog post about the START Pilot Program called Support for Clinical Trials Advancing Rare Disease Therapeutics. This initiative draws inspiration from Project Warp Speed, which expedited therapeutics development during the Covid-19 pandemic. The post details seven companies' participation in START, the products they are developing, and the specific rare diseases they aim to address. 💊
CDRH has proposed a new collection of information for its Voluntary Total Product Life Cycle (TPLC) Advisory Program (TAP) Pilot, launched in 2023. This initiative is a commitment under the Medical Device User Fee Act reauthorization, aimed at offering participants enhanced early-stage interactions to mitigate failure rates. Part of the program's requirements includes tracking outcomes and participant satisfaction, as outlined in the Federal Register notice detailing these aspects. 💉
CDRH has recently updated its webpage dedicated to the "Patient and Caregiver Connection" program. This initiative facilitates interactions between regulators and individuals—patients or caregivers—who can provide firsthand experiences related to medical conditions or medical devices. 💉
CDER and CBER have assessed the use of RWE in submissions over the last fiscal year. Out of the submissions reviewed, 10 protocols and 4 new product applications included RWE. Detailed information on how RWE contributed to these applications can be found on their respective web pages. 💊
CDRH has announced an upcoming medical device sterilization town hall meeting (July 10) focused on discussing regulatory, design, and testing considerations related to changes in sterilization methods. The meeting will include a mock pre-submission for a fictional medical device. This initiative is significant as both the EPA and FDA are encouraging companies to transition away from ethylene oxide-based sterilization methods following the EPA's recent final rule. 💉
Governing
Senator Robert Casey (D-Pennsylvania) has introduced new legislation to extend the rare pediatric disease priority review voucher program. The program is slated to expire at the end of the federal fiscal year unless renewed. 💊