CGT's Top Regulatory Challenges Broken Down By Segment
By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On Twitter @ErinHarris_1
Our recent Cell & Gene Live, Tackling Cell & Gene Therapies’ Top 3 Regulatory Impediments, featuring expert panelists, Dr. Adora Ndu, Chief Regulatory Affairs Officer at BridgeBio and Dr. Nina Hunter, Vice President, Corporate Strategy at REGENXBIO, delivered just under 60 minutes of exceptionally valuable information. Indeed, during the presentation, Dr. Ndu and Dr. Hunter answered my questions about problems in the industry, specific FDA initiatives to overcome said problems, broader regulatory trends, and more. If you were unable to attend the live event or if you want to re-watch, the full-length version is available on demand.
We also know that your time is valuable, which is why we’ve broken down the full-length version into digestible segments that you can view on your own time. Enjoy.
Segment 1:
CGT’s Main Opportunities to Meet Regulatory Requirements
I always like to begin Cell & Gene Live events by level setting the conversation at hand before digging into the details. Here, I ask our experts to highlight the main opportunities where the cell and gene therapy industry is working to meet regulatory requirements. They cover accelerated and expedited designation of regenerative medicines, navigating the regulatory requirements around the root causes of genetic disease by repairing or replacing a patient’s genes, and much more.
Segment 2:
Challenge #1: The Changing Regulatory Landscape
By 2030, 60 CGTs are expected to reach regulatory approval. One of the biopharmaceutical industry’s most closely watched regulatory developments is the FDA’s reauthorization of the Prescription Drug User Fee Act (PDUFA) or PDUFA VII, as it provides resources for the FDA and CBER to have the capacity to provide feedback on development and review submitted applications, among other critical activities. During this segment, Dr. Hunter and Dr. Ndu break down how PDUFA VII, Fiscal Year 2023 Omnibus Appropriations Bill, and more will impact the cell and gene therapy field in the next few years.
Segment 3:
Challenge #3: Rare or Ultra-Rare Study Design Considerations and Use of Existing Data
There are more than 7,000 unique, rare diseases recognized in the United States, and 300 million people worldwide living with a rare disease; patients often experience a long journey to receive their diagnosis. In this segment, our expert panelists examine the regulatory requirements for safety and efficacy demonstration and identify the gaps in regulatory oversight that require clearer guidance.
Segment 4:
Challenge #3: Lack of Animal Models
Many cell and gene therapies are intended for diseases and conditions that primarily impact pediatric patients, requiring the prospect of direct benefit to individual subjects enrolled in the trial. In the absence of good animal models, there may be challenges demonstrating the prospect of direct benefit. During this segment, Dr. Hunter discusses novel approaches to consider going forward.
Segment 5:
Other CGT Regulatory Challenges
Here, Dr. Hunter and Dr. Ndu detail their own experiences with roadblocks with regulators and how they and their teams worked to overcome them.
During each Cell & Gene Live, our audience submits real-time questions for our expert panelists. Watch and listen as Dr. Hunter and Dr. Ndu provide detailed, insightful responses to our audience questions on everything from regulatory timelines to successful and unsuccessful strategies needed to convince the regulators that current regulation requirements are either inappropriate or alternatively excessively burdensome for cell and gene-based therapies. The Audience Q&A is always a can’t-miss.
Be sure to register for our next Cell & Gene Live, which will take place on Monday, February 13, 2023 at 11 am EST. Nurix’s Michael Blackton, Center for Breakthrough Medicines’ John Lee, and I will be back for Part 2 of our wildly popular discussion on scaling cell therapies, this time focusing on comparability and how establishing manufacturing, characterization, and controls can positively affect scalability. Registration is free. See you there!