S. 3551 — PROTECT for Rare Act

This section expands Medicare Part B coverage of drugs and biologicals under the definition of medical and other health services (42 U.S.C. 1395x(t)(2)) to include those used in the treatment or management of a disease or condition affecting 200,000 or fewer individuals in the United States, if supported by peer-reviewed medical literature (including clinical guidelines) and not listed as not indicated in specified compendia or contraindicated in FDA labeling (effective January 1, 2027). It also expands the definition of a medically accepted indication for covered Part D drugs to include such rare disease or condition uses (effective for plan years beginning on or after January 1, 2027) and revises the Medicaid definition of medically accepted indication for covered outpatient drugs to include uses supported by peer-reviewed medical literature, clinical guidelines, or an expert identified by a medical society treating the rare disease or condition, if not listed as not indicated in specified compendia or contraindicated in FDA labeling (effective January 1, 2027). Additionally, this section requires group health plans and health insurance issuers under the Public Health Service Act and ERISA to provide an expedited appeals process pursuant to PHSA §2719 for coverage denials of FDA-approved or -licensed drugs or biological products used for rare diseases or conditions, if supported by FDA labeling or peer-reviewed literature (including clinical guidelines) not unfavorably reviewed in specified compendia or listed as contraindicated in FDA labeling.