Patients left behind: Rare dermatologic conditions miss the orphan drug development boom

Abstract

Abstract Orphan drugs are indicated for treatment of uncommon conditions and are granted seven years of exclusivity by the FDA. As a result of the designation, there has been a considerable increase in rare disease research, pharmaceutical development, and market expenditures, however, we propose that rare dermatologic diseases (RDD) have been relatively neglected. To investigate the extent of active clinical research for RDD, we performed a search within a clinical trial database and found that of 428 rare dermatologic diseases, only 41 diseases (10%) were undergoing a total of 255 active clinical trials. Of those trials, 104 (41%) were industry-funded. Only 21 (5%) of RDD diseases were undergoing Phase 2 and 3 clinical trials, suggesting that few RDD were in late stage clinical development or likely of reaching regulatory approval.