Dr. Jason Mercer attended the annual Food and Drug Law Institute (FDLI) annual conference May 17-18 in Washington, DC.  The FDLI annual conference addresses complex legal, regulatory, compliance, and policy issues faced by FDA-regulated industry.  The conference fosters in-depth discussions of timely topics in the industry by bringing together experts from the federal government, industry, law firms, patient and consumer advocacy groups, consulting organizations, and academia.  This year’s conference featured presentations on the FDA’s strategic priorities for the upcoming year from Dr. Robert Califf, FDA Commissioner and Mark Raza, FDA Chief Counsel.  In depth discussions highlighted recent congressional actions and recent court cases and the impacts on the FDA, industry, and public health.  Sessions on rare disease drug development, regulation of cell and gene therapies offered helpful insights into current industry and FDA approaches in these highly important and evolving areas of therapeutic product development.

Dr. Lisa VanLuvanee attended the FDA CDER & JHU CERSI Workshop:  Addressing Challenges in the Design and Analysis of Rare Disease Clinical Trials: Considerations and Tools on May 2-3. In this workshop, FDA and Johns Hopkins University’s Center for Excellence dynamically discussed the challenges of drug development for rare diseases.  Topics included clinical trial design and interpretation issues resulting from a small number of patients with individual disorders, the lack of drug development and regulatory precedent for many rare diseases, and the lack of established endpoints.  The pros and cons of real-world evidence (RWE), natural history studies, and registries were also noted.  The meeting recording, slides, and other information are provided via a link on FDA’s website here: https://www.fda.gov/drugs/news-events-human-drugs/fda-cder-jhu-cersi-workshop-addressing-challenges-design-and-analysis-rare-disease-clinical-trials

Dr. Lisa VanLuvanee attended the Duke-Margolis Center / FDA workshop on Rare Disease Endpoint Advancement: Novel Endpoints for Rare Disease Drug Development on June 7-8. The Duke-Margolis Center for Health Policy and FDA convened to discuss the challenges and opportunities in rare disease endpoint development.  Of specific interest, the Rare Disease Endpoint Advancement (RDEA) Pilot Program was highlighted.  The goal of the RDEA program is to foster more rapid rare disease product development through enhanced Sponsor-FDA collaborations on the efficacy endpoint development process.  The RDEA program is also intended to provide greater transparency of evolving science through FDA presentations, guidance documents, public workshops, and a public-facing website.  Although only 1 RDEA proposal request is being granted in 2023, FDA will accept up to 3 RDEA proposals each year for fiscal years 2024-2027.  Facet is excited to see the FDA’s efforts to collaborate with Sponsors on rare disease development and is optimistic for the future of product development in this space.  The meeting recording, slides, and other information are publicly available on Duke’s website here: https://healthpolicy.duke.edu/events/rare-disease-endpoint-advancement-pilot-program-workshop-novel-endpoints-rare-disease-drug

NEW FACET TEAM MEMBER. Facet welcomes Dr. Marcoita Gilbert, an accomplished regulatory professional with 10 years of technical expertise in nonclinical drug development and applied toxicology.  Marcoita has a Ph.D., in pharmacology, toxicology, and physiology from the Brody School of Medicine at East Carolina University.  She also holds 3 BS degrees (neuroscience, chemistry, and biology).  Prior to joining Facet, she served as a Director of Regulatory Affairs at Cardinal Health, and formerly held roles as an expert toxicologist at both Cato and Camargo.  We are excited to welcome Marcoita to the Facet team as a Product Development Champion!