In this episode, Eric Hardter and guest Kelsey Lenoch discuss recent US FDA developments, including leadership changes at CBER and CDER, staffing impacts on orphan product reviews, and the new Rare Disease Evidence Principles aimed at accelerating approvals for rare and ultra-rare conditions.

They also explore evolving trial endpoints in oncology (including surrogate measures and ctDNA), transparency moves such as the public release of Complete Response Letters and the industry’s reactions, updates on the National Priority Voucher Program, and the FDA Pre-Check initiative to boost domestic drug manufacturing.

Tune in for an in-depth discussion offering timely updates and expert perspectives on what may be ahead for drug development stakeholders.

Welcome and Introductions (00:00:05) Recap and Recent US Regulatory Affairs Developments (00:01:00) Dr. Vinay Prasad’s Removal and Return to FDA CBER (00:01:20) New Appointments at FDA CDER (00:02:17) FDA Staffing Shortages and Impact on Orphan Products (00:04:22) Rare Disease Evidence Principles Announcement (00:05:29) FDA’s Rare Disease Innovation Hub and Ultra-Rare Disease Focus (00:07:19) Novel Endpoints in Oncology Drug Development (00:09:31) FDA/AACR Joint Meeting on Novel Endpoints (00:10:12) Challenges with Overall Survival as an Endpoint (00:10:36) FDA’s Evolving Guidance on Endpoints (00:11:04) Post-Licensure Requirements and Surrogate Endpoints (00:11:48) Case Study: Myelofibrosis Drug and Patient-Reported Endpoints (00:12:49) Standardization Challenges for New Endpoints (00:13:55) Circulating Tumor DNA as a Biomarker (00:14:51) FDA’s Release of Complete Response Letters – Phase Two (00:16:06) Pros and Cons of Public Complete Response Letters (00:17:19) Industry Pushback and Sponsor Transparency (00:19:22) Case Study: Lycos Therapeutics and Psychedelic Drug Approval (00:21:09) Accessing and Mining Complete Response Letter Data (00:22:34) National Priority Voucher Program Update (00:23:43) FDA Pre-Check Program for Domestic Manufacturing (00:24:26) Closing Remarks and Future Outlook (00:25:12)