Rare disease drugmakers lobby Trump on Medicare price pilots
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- Rare Disease Company Coalition met with the White House Office of Management and Budget last week to lobby for excluding treatments for rare diseases from Trump's Medicare brand-drug-price pilots
- The two pilot programs at issue are part of President Trump's plan to get drugmakers to lower US prices to levels charged in other wealthy countries — a policy generally referred to as most-favored nation
- Carving out orphan drugs would shrink the pilots, which already exclude at least some major drugmakers, per the article's framing
- The coalition is composed of biotech companies developing rare-disease treatments, the same market long covered by the orphan-drug designation regime
Why it matters: Excluding orphan drugs would shrink the pilots — which already carve out at least some major drugmakers — and narrow the slice of the brand-name market the administration can actually pressure on pricing. For rare-disease patients, the carve-out preserves existing treatment pipelines but could leave them outside the discounted-pricing framework the pilots are designed to deliver.
