Interview with Prof. Janet Woodcock: From Regulatory Leadership to Rare Disease Innovation
On August 7, 2026, the Rare Disease and Orphan Drugs Journal (RDODJ) interviewed Prof. Janet Woodcock, a distinguished leader in drug regulatory science and former senior FDA official, focusing on the evolving challenges and future of rare disease drug development. The interview explored key issues including clinical evidence generation for rare and ultra-rare diseases, regulatory approaches for emerging therapies, and the future of gene and cell therapy development.
Interview Questions
1. After decades of leadership at the FDA, what motivated you to focus more on rare disease drug development in your recent academic work?
2. In your recent article, you argue that the major challenge in rare disease drug development is no longer discovering potential therapies, but generating sufficient clinical evidence. What changes in the current development and regulatory system do you believe are most urgently needed to address this gap?
3. For ultra-rare diseases, randomized controlled trials are often impractical. What types of evidence should regulators and clinicians be prepared to accept while still maintaining confidence in a therapy's safety and effectiveness?
4. As gene and cell therapies continue to emerge, do you think traditional clinical development paradigms are still appropriate for rare diseases, or is a fundamentally different framework needed?
5. Looking back on your remarkable career in regulatory science, what achievement do you believe has had the greatest impact on patients, and why?
6. What advice would you offer to young investigators entering the field of rare disease research and regulatory science?
About the Interviewee:
Prof. Janet Woodcock is a prominent figure in drug regulatory science and pharmaceutical development. She began her career at the FDA in 1986 and went on to serve in several senior leadership positions, including Director of the Center for Drug Evaluation and Research (CDER), Deputy Commissioner and Chief Medical Officer, and Principal Deputy Commissioner. She also served as Acting Commissioner of Food and Drugs from January 2021 to February 2022. Throughout her career, Prof. Woodcock has played a significant role in advancing drug development and regulatory science. Her work has included initiatives related to risk management, pharmaceutical quality, drug safety, the Critical Path Initiative, the Sentinel Network, and patient-focused drug development. She has received numerous honors recognizing her contributions to public health, drug development, and regulatory innovation.
Editor: Daisy Ren
Production Editor: Xingyue Luo
Respectfully Submitted by the Editorial Office of Rare Disease and Orphan Drugs Journal
