Forbes November 4, 2025
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 potentially billions of dollars of cost savings for Medicare, and that granting manufacturers of rare disease drugs a “special exemption” is unnecessary for them to achieve financial success.
The Senators say their goal is to preserve incentives for “genuine rare-disease innovation”...
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