BioCentury
ARTICLE | Politics, Policy & Law

Pitched as savior for small biotechs, Orphan Cures Act boosts big pharmas

The single-orphan exemption had unanticipated impacts, so did expanding it

September 8, 2025 11:09 PM UTC

The biotech industry and patient advocates secured one of their top priorities in July when Congress included the Orphan Cures Act in the “big beautiful” budget and tax bill, expanding the orphan drug exemption from Medicare drug price negotiation.

Orphan Cures was supposed to offset unintended effects of the Inflation Reduction Act (IRA) that hurt patients with very rare diseases. Ironically, it has created another set of unanticipated effects — protecting some blockbuster drugs from Medicare price negotiation. ...