Sponsors (2)
The bill increases access to and speeds coverage of drugs for rare diseases by allowing literature- and guideline‑supported uses to count as medically accepted indications and creating faster appeals, improving treatment access and lowering out‑of‑pocket costs for beneficiaries while raising federal/state spending, insurer costs, and risks from coverage of therapies with limited evidence and potential program‑integrity gaps.
Medicare and Medicaid beneficiaries gain broader coverage for drugs used to treat rare diseases when supported by peer‑reviewed literature or clinical guidelines, expanding access to therapies.
Part D enrollees and other beneficiaries will have more rare‑disease uses deemed 'medically accepted' for coverage, which should reduce out‑of‑pocket costs for patients needing those drugs.
Participants in group and individual health plans (and Medicaid enrollees) gain expedited appeals for denials of coverage for qualifying rare‑disease drugs, speeding access to needed therapies.
Federal taxpayers and Medicare/Medicaid budgets may face higher spending as more rare‑disease drug uses are covered, increasing program costs and fiscal pressure.
Relying on peer‑reviewed literature and clinical guidelines (rather than only FDA indications) may result in coverage of uses with limited evidence, risking patient exposure to therapies with uncertain benefit.
Insurers and plan sponsors may face higher pharmacy benefit costs and utilization, which could translate into higher premiums or more aggressive cost‑management that affects enrollees.
Based on analysis of 2 sections of legislative text.
Changes Medicare and Medicaid rules so certain drug uses for rare diseases (≤200,000 people) qualify for coverage if supported by peer‑reviewed literature, guidelines, or a medical society expert and not excluded by compendia/FDA labeling.
Expands Medicare and Medicaid coverage rules so that certain uses of drugs for rare diseases (those affecting 200,000 or fewer people in the U.S.) can qualify as "medically accepted" when supported by peer‑reviewed medical literature, clinical guidelines, or an expert identified by a medical society — provided the use is not listed as “not indicated” in specified compendia or contraindicated in FDA labeling. The change applies to Medicare Part A/B items and services furnished on or after January 1, 2027, and to Medicare Part D for plan years beginning on or after January 1, 2027; the Medicaid subsection is replaced but the effective date for Medicaid is not specified in the excerpt. The result is broader, standardized criteria for coverage of rare‑disease drug uses that are supported by scientific literature or professional guidance, which may increase access for people with rare conditions, change payer prior‑authorization and coverage practices, and affect costs for federal and state health programs and plan sponsors.
Introduced December 17, 2025 by Thomas Roland Tillis · Last progress December 17, 2025