Investigating the landscape of US orphan product approvals

Kathleen L. Miller and Michael Lanthier, Orphanet Journal of Rare Diseases 2018 13:183, doi:10.1186/s13023-018-0930-3

The Orphan Drug Act was enacted in 1983 to encourage the development of drugs for rare diseases. Previous research has attempted to examine the impact of the Act by assessing either the number of orphan designations that have been granted or the number of new orphan drugs approved for marketing. This study provides a more in-depth understanding of the effect of the Orphan Drug Act by investigating all types of drug approvals with an orphan designation, along with multiple characteristics of the drugs, over the entire 35 years of the Act. These orphan approvals include: new molecular entities (new drugs approved first for a rare disease), secondary indications (an expansion from the first approved indication), and new formulations.

Investigating the landscape of US orphan product approvals