Orphan drug exemptions could kill Medicare drug savings

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- Harvard University researcher finds excluding orphan disease therapies from Medicare drug-pricing pilots would wipe out much of the retail drug savings, undermining the policy’s impact
- Biotech companies are lobbying the Trump administration to exclude rare disease treatments from the GLOBE and GUARD Medicare pilot programs aimed at lowering U.S. drug prices
- Trump administration is considering scope changes to the GLOBE and GUARD pilots, part of a 'most-favored nation' strategy to align U.S. drug prices with other wealthy countries
- Major global drugmakers that struck most-favored nation pricing deals with the White House may have been exempted from the pilots, though details of those agreements remain undisclosed
Why it matters: Exempting orphan drugs could erase the financial foundation of Medicare’s drug-pricing pilots, which rely on broad participation to generate savings. If large manufacturers are already excluded through private deals, the public goal of lower prices risks being undermined by backroom arrangements benefiting industry.




