The ASF and FAST recently joined other rare disease groups to ask the Food and Drug Administration (FDA) to establish a Rare Disease Center of Excellence in the Agriculture, Rural Development, Food and Drug Administration and Related Agencies Fiscal Year 2025 appropriations bill. The report language directs the FDA to create an Intercenter Institute for Rare Diseases that will serve as a cross-cutting, capacity-building, collaborative hub for rare disease activity at the FDA.
While interest in rare disease therapy development has increased since the passage of the historic Orphan Drug Act of 1983, the regulatory systems we have in place struggle to meet the unique challenges and complexities inherent in rare diseases. The last 40 years have yielded tremendous progress, going from 38 approved drugs to more than 1,200 approved indications for rare diseases. Despite significant scientific advancements, the rare disease community continues to face substantial obstacles in the development, review, and approval of safe and effective treatments. With over 10,000 rare diseases affecting more than 30 million Americans, the urgency for a streamlined and focused approach in regulatory science and review processes cannot be overstated. About 95 percent of rare disease communities still lack an FDA-approved treatment and significant unmet needs remain for the communities that do have an approved treatment.
We are proud to support such important work in the rare disease space.