Orphan Drug, Rare Pediatric Disease and Expedited Program Designations

What You Will Learn

Substantial progress continues in the development of treatments for rare diseases or orphan products. In 2020, 32 novel drugs and biologics were FDA approved with orphan drug designation status. Notably, 1 of 5 (20%) novel biologic approvals in the Center for Biologics Evaluation and Research (CBER)—although not orphan designated—was used in rare diseases. In the Center for Drug Evaluation and Research (CDER), 31 of 53 (58%) novel drug approvals were orphan designated products. Unfortunately, 1 in 10 (25 to 30 million) Americans is affected by rare diseases (more than half of which are children), and approximately 95% of the more than 7,000 rare diseases have no treatments available.

To advance the development of rare disease treatments, the FDA Office of Orphan Products Development grants orphan drug and rare pediatric disease designations (ODD and RPD) to eligible products for the prevention, diagnosis, and treatment of rare diseases. Additionally, CBER and CDER provide four expedited program and approval pathways—Fast Track Designation (FTD), Breakthrough Therapy Designation (BTD), Accelerated Approval, and Priority Review Voucher Designation (PRV)—to facilitate and expedite the development and review of drugs to address the unmet medical need in the treatment of serious and life-threatening conditions. Because most rare diseases are serious or life-threatening, with a high unmet medical need, most qualify for at least one expedited program.

This rare disease webinar provides an overview of the FDA’s compendium of rare disease and expedited programs and provides sponsors with recommendations to leverage these opportunities and speed the development timeline.

Attendees will learn how to:

• Features and benefits of ODD, PRD, FTD, BTD, Accelerated Approval, and PRV
• Qualifying criteria for each designation or approval pathway
• When to submit a designation request
• Real world examples highlighting expedited drug development timeline with designations
• Key differences between FDA and European Medicines Agency (EMA)
• Rare disease and expedited programs, including EMA orphan drug and PRIority MEdicines (PRIME) designations