Non-oncologic orphan drug approvals across the world: Types of evidence required and time to approval

Abstract

Large time gaps are frequently observed between approvals of orphan products by different jurisdictions. A retrospective review of publicly accessible information for new orphan medicines approvals between 2021 and 2022 was performed to quantify the types of evidence required and timeframes for initial and subsequent approvals in six different regions. We identified 53 orphan products: 91% were approved in two or more regions, and 69% relied upon the same/highly similar types of evidence for marketing authorization. Time between subsequent marketing authorizations varied widely, with a mean of 1 year for the second, increasing to 3 years for the third through sixth authorizations. A high degree of consistency in the evidence accepted across different regions was found, and global collaborative programs might help accelerate timelines.

Keywords: drug development; marketing authorization; new drug approval; orphan drug; rare diseases.