Orphan drug exemption would erase Medicare pilot savings
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- A Harvard University researcher found that excluding rare disease treatments from Medicare drug-pricing pilot programs would wipe out much of the savings the policies are designed to deliver on retail drugs.
- Biotech companies are lobbying the Trump administration to exclude orphan disease treatments from two Medicare pilots: Global Benchmark for Efficient Drug Pricing (GLOBE) and Guarding U.S. Medicare Against Rising Drug Costs (GUARD).
- The GLOBE and GUARD pilots are part of President Trump's "most-favored nation" policy, which aims to lower U.S. drug prices to the levels seen in other wealthy countries.
- Some of the largest drugmakers globally that struck individual most-favored nation pricing deals with the White House have said they were exempted from the pilots, though deal details have not been made public.
- The exact scope of the pilot programs remains unsettled, leaving uncertainty about which therapies and companies will ultimately participate.
Why it matters: If the orphan drug carve-out is granted, two Medicare pilot programs central to Trump's most-favored nation pricing agenda could lose much of their projected savings before they begin — handing biotech lobbyists a win that cuts against the policy's stated goal of reducing Medicare drug costs.
