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The Impact of Single Indication Orphan Drugs on the Future of Rare Diseases

According to a new study by FDA and HHS researchers published in the January 2024 issue of Health Affairs , 65% of 491 novel drugs (NMEs) were approved for a single rare disease from 1990 through 2022. The research showed that 15% were approved for multiple rare disorders, while 20% had been approved for both rare and common diseases. 10% subsequently received a pediatric approval for an orphan condition. There have been more 800 orphan-designated drug approvals since the U.S. Orphan Drug Act was established in 1983.

Notably, research also shows that just 6% orphan drugs specifically indicated for rare diseases are among the top 200 branded drug sellers in the world. Indeed, the majority of branded high-revenue orphan drugs are indicated for oncology and other larger orphan conditions.

The research provides proof that the Orphan Drug Act of 1983 is aligned with the original intent of its policy sponsors, which was to encourage drug research and development for disorders that individually affect less than 200,000 people in the U.S. That said, we wonder if it is time to update the prevalence calculation for rare conditions that was used more than 40 years ago to determine the 200,000 lives threshold for the law given that the U.S. population has increased nearly 42% since 1983. Following the original logic used by policymakers then, that number would be closer to 275,000. What do you think?


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