Carolyn Shore

Oct 2, 2024 | jamanetwork.com | Carolyn Shore |Jeffrey Kahn |Johns Hopkins

Rare Disease Innovation at the FDA—Opportunities for Implementation Over the past 4 decades since the Orphan Drug Act was passed in 1983, there have been tremendous advances in research, innovation, and regulatory policies that have spurred development of new treatments for rare diseases, but less than 5% of rare diseases have approved treatments on the market.1 More must be done to meet the needs of people living with rare diseases.