PROTECT for Rare Act
Sponsored By: Senator Tillis, Thomas [R-NC]
Introduced
Summary
Expanded access to drugs for rare diseases by creating a rarity-based coverage pathway and faster appeals across Medicare, Medicaid, and private insurance for conditions affecting 200,000 or fewer people.
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- Medicare beneficiaries: Adds a rarity-based way to qualify a drug as a “medically accepted indication” if the use is supported by peer‑reviewed literature or clinical guidelines and not contradicted by compendia or FDA labeling. This applies to Part B and Part D uses furnished on or after January 1, 2027.
- Medicaid enrollees: Broadens the Medicaid definition of “medically accepted indication” to include rare‑disease uses backed by peer‑reviewed literature, clinical guidelines, or an expert named by a medical society, provided the use is not listed as not indicated in compendia or contraindicated in FDA labeling. This applies to covered outpatient drugs furnished on or after January 1, 2027.
- Private plan enrollees and ERISA-covered workers: Requires expedited appeals for denials of drugs used for diseases affecting 200,000 or fewer people when the use is supported by labeling or peer‑reviewed literature and not unfavorably reviewed in compendia or contraindicated. The expedited process applies to plan years beginning on or after January 1, 2027.
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Bill Overview
Analyzed Economic Effects
2 provisions identified: 2 benefits, 0 costs, 0 mixed.
More coverage for rare disease drugs
If enacted, Medicare and Medicaid would expand what counts as a medically accepted use for drugs that treat diseases affecting 200,000 or fewer people. Uses would qualify when supported by peer‑reviewed medical studies, clinical guidelines, or an expert from a medical society. Coverage would still be barred if a compendium lists the use as "not indicated" or if FDA labeling lists it as a contraindication. The change would apply to drugs and items furnished on or after January 1, 2027, and to Medicare Part D plan years beginning on or after January 1, 2027.
Faster appeals for rare drug denials
If enacted, group health plans and issuers would have to offer an expedited internal appeals process for denials of FDA‑approved or licensed drugs used to treat diseases affecting 200,000 or fewer people. The use would need to be supported by approved labeling or by peer‑reviewed literature and not be listed as "not indicated" in the compendia or as a contraindication in FDA labeling. This requirement would apply to plan years beginning on or after January 1, 2027.
Sponsors & CoSponsors
Sponsor
Tillis, Thomas [R-NC]
NC • R
Cosponsors
Sen. Heinrich, Martin [D-NM]
NM • D
Sponsored 12/17/2025
Roll Call Votes
No roll call votes available for this bill.
View on Congress.gov