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)

• 00:00:13 – Introduction to Conversations in Drug Development

• 00:00:30 – Welcoming Professor Alan Boyd Back

• 00:01:43 – Reflecting on 20 Years of Boyds

• 00:02:49 – The Journey to Founding Boyds

• 00:06:05 – Early Challenges in Gene Therapy

• 00:07:26 – Manufacturing Gene Therapy Products

• 00:10:35 – Achievements in Cell and Gene Therapy

• 00:15:39 – Highlights from Two Decades

• 00:19:46 – Changes in Drug Development

• 00:23:32 – Evolution of Clinical Trials

• 00:28:17 – The Role of the Team at Boyds

• 00:31:43 – Personal Interests Beyond Medicine

• 00:38:12 – Future of Drug Development

• 00:38:42 – Closing Thoughts and Reflections

In the latest episode of Conversations in Drug Development, Dr Katherine Bowen and Dr Eric Hardter discuss the ever-changing US regulatory landscape under the new administration, including new leadership and staff turnover, and their impact on drug development. They examine the FDA's cautious approach to AI, efforts to reduce animal testing, and the new National Priority Voucher Program aimed at expediting drug approvals. Tune in for an in-depth discussion that offers timely updates and expert perspectives on what might be in store for drug development stakeholders.

EPISODE OUTLINE

00:00:14 – Welcome to Conversations in Drug Development 00:01:16 – US Regulatory Environment Update 00:02:35 – Changes in FDA Leadership 00:03:19 – The Impact of Staffing Changes 00:05:54 – New Perspectives with Vinay Prasad 00:07:50 – Future of Cell and Gene Therapy Approvals 00:10:03 – Market Reactions and Funding Concerns 00:11:45 – Implications of FDA Cuts 00:12:29 – Marty McCary’s Vision for the FDA 00:14:46 – Navigating Conflicting Perspectives 00:15:45 – FDA Workforce and Operational Changes 00:17:21 – Review Process and Efficiency Challenges 00:20:43 – ELSA: AI in FDA Operations 00:22:54 – Reducing Animal Testing in Drug Development 00:25:05 – National Priority Voucher Programme Insights 00:31:21 – Future Outlook and Next Steps