Rare disease drugmakers seek carve-out from Trump pricing pilots
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- Rare Disease Company Coalition met last week with the White House Office of Management and Budget to lobby for excluding rare disease drugs from two Medicare price-cut pilot programs.
- Biotech companies are pressing the Trump administration to carve out treatments for rare diseases from programs designed to lower brand drug prices in Medicare.
- The two pilot programs are part of Trump's most-favored nation policy, which aims to match U.S. drug prices to levels charged in other wealthy countries.
- The lobbying target — Medicare's brand drug pricing pilots — is the key mechanism the administration is using to enforce most-favored nation pricing domestically.
Why it matters: If the coalition succeeds, rare disease drugmakers would sidestep Medicare's price-cap pressure entirely, protecting pricing power on therapies that often face little competition. If they lose, small-population drugs would be subject to the same international price-matching as mass-market blockbusters — a materially different revenue environment for the rare disease sector.
