Drug Development for Rare Diseases - Public Workshops

Drug development for rare diseases is challenging, complex, but absolutely necessary. FDA has special programs to manage and encourage the drug development for rare diseases. 

According to "Rare Diseases at FDA" website, all three divisions (CDER, CBER, and CDRH) have special programs to support the drug development in rare diseases areas:

• CDER's Rare Disease Program
• CBER's Rare Disease Program

At "Regulatory Education for Industry (REdl)" meeting this week, there was a session "Partnering Across FDA to Advance Therapies for Rare Diseases" featuring three prosentations by FDA officers:

• Dr Retzky presented "FDA Rare Disease Updates: Incentives and Resources"
• Dr Lee presented "CDER's Perspective on Working Together with Our Rare Disease Partners within CDER and across FDA"
• Ms Vaillancourt presented "CBER's Perspective on Working Together with Our Rare Disease Partners across FDA"
• Dr Sherafat presented "Design Considerations for Clinical Trials in Rare Diseases"

FDA/CDER has an ARC program (Accelerating Rare disease Cures) aiming to drive scientific and regulatory innovation and engagement to accelerate the availability of treatments for patients with rare diseases. In year 1 of the ARC program, two important public workshops had been organized (webcasts can be watched following the links below):

• FDA/NIH Regulatory Fitness in Rare Disease Clinical Trials conference, May 16-17, 2022

CDER’s Rare Diseases Team and National Center for Advancing Translational Sciences
Focus on academic investigators and those looking to learn how to bridge the gap between academic investigation and the regulatory aspects of drug development

• FDA and Duke Margolis Virtual Public Workshop: Translational Science in Drug Development: Surrogate Endpoints, Biomarkers, and More, May 24-25, 2022

Focus on translational science and the development of surrogate endpoints