A study of Fermaglich and Miller (2023) evaluated trends in orphan drug designations and approvals after the Orphan Drug Act of 1983 was approved in the US. The authors use FDA data to evaluate between 1983 and 2022 and find that:
During the 40 years of the ODA, 6,340 orphan drug designations were granted, representing the development of drugs for 1,079 rare diseases. Additionally, 882 of those designations resulted in at least one FDA approval for use in 392 rare diseases. Much of this development has focused on oncology, as seven of the ten most designated and approved diseases were rare cancers.
Of the orphan drug designations (approvals), the top five disease areas were:
- Oncology: 38% of designations (38% of approvals)
- Neurology: 14% (10%)
- Infectious Disease: 7% (10%)
- Metabolism 6% (7%)
- Hematology: 5% (8%)
More details on orphan drug designations and approvals can be found in the full document. here.
