Inclusion of the Orphan Cures Act in the One Big Beautiful Bill legislation implies that certain high-cost drugs with orphan indications will either no longer be eligible for Medicare price negotiation or will have their eligibility deferred. getty
Senators Welch (D-VT), Cortez Masto (D-NV) and Wyden (D-OR) introduced legislation last month to repeal a provision in the One Big Beautiful Bill exempting all orphan-only drugs from Medicare price negotiation. The senators seek to replace it with a policy that would only exempt “true rare-disease drugs” that account for less than $400 million in annual Medicare spending.
The legislators’ objective partly aligns with arguments made by researchers against having orphan drug exceptions. They maintain that these stipulations preclude potentia

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