Analysis questions expanded orphan drug exemptions under recent legislation

Ed Silverman, a Pharmalot columnist, examined a provision in the One Big Beautiful Bill Act that keeps certain orphan drugs out of Medicare price negotiations.

The Inflation Reduction Act, which began allowing Medicare to negotiate drug prices three years ago, originally exempted orphan drugs that had only one FDA‑granted orphan designation.

The newer Orphan Cures Act, incorporated into the Big Beautiful Bill Act, expands that exemption to medicines with multiple orphan indications and also pushes back the timelines when an otherwise eligible orphan drug might be considered for negotiation.

The analysis concludes that the broader exemption may not be warranted, raising concerns that it could limit the effectiveness of Medicare’s pricing leverage on rare‑disease treatments.