The passage of the Orphan Drug Act (ODA) in 1983 has provided important regulatory and economic incentives to develop novel medicines for patients suffering from rare diseases that may not be commercially attractive enough to offset the research and development costs of small patient populations.  We have analyzed 497 novel drugs approved between 2010 and 2022, of which 220 were given orphan designation status under the ODA.  We discuss trends over this time period of increasing numbers of drugs approved with orphan designation, with approximately 50% of approved drugs per year from 2017-onwards receiving this designation.  The majority of drugs approved with this designation are for oncology (140 drugs, 30% of all approved drugs), followed by infection (68 drugs, 14%) and neuroscience (62 drugs, 13%) as the top three therapy areas.  We also discuss potential risks for long development times, and provide example case studies of successful development and launch of novel drugs for rare diseases.

Analysis of orphan designation status for FDA approved drugs, 2010-2022

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