Rare disease drugmakers are lobbying the Trump administration to exclude their treatments from Medicare pilots aimed at cutting brand-drug prices. The Rare Disease Company Coalition met with the White House Office of Management and Budget to discuss two pilot programs tied to a most-favored-nation approach. The groups say the pilots could disrupt access for patients relying on therapies that often serve small populations and follow distinct development and pricing dynamics. The coalition’s engagement signals a near-term policy confrontation between affordability initiatives and the economics of rare-disease drug development. The outcome remains tied to policy design and implementation details, with rare-disease stakeholders seeking carve-outs before pilots finalize and scale.