Confronting the Challenges of Rare Disease

 

Confronting the Challenges of Rare Disease

The Orphan Drug Act of 1983 brought increased awareness to the need for new treatments for rare disease patients and provided incentives to pharmaceutical manufacturers of orphan treatments, including tax credits for the costs of clinical research, seven years of patent exclusivity, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees. Of the 3,500 drug designations issued by the Office of Orphan Products Development since 1983, more than 500 have resulted in marketing approval. Still, only 5 percent of the estimated 7,000 known rare diseases have a therapy approved by theU.S. Food and Drug Administration (FDA), leaving substantial unmet medical needs for patients.