PROTECT for Rare Act

This bill amends Medicare, Medicaid, and private health insurance laws to require coverage and an expedited appeals process for drugs used to treat or manage rare diseases or conditions affecting 200,000 or fewer people in the United States, based on peer-reviewed medical literature or approved labeling, effective January 1, 2027.

Sec. 2, Coverage of certain drugs used in treatment or management of a rare disease or condition: The bill has three parts. (a) Medicare: It expands the definition of "medically accepted indication" for Medicare Part B and Part D drugs to include uses for rare diseases supported by peer-reviewed medical literature or clinical guidelines, as long as the drug is not listed as not indicated in standard compendia or contraindicated by the FDA. This applies to items and services furnished on or after January 1, 2027. (b) Medicaid: It revises the definition of "medically accepted indication" for Medicaid covered outpatient drugs to include uses for rare diseases supported by peer-reviewed medical literature, clinical guidelines, or an expert identified by a medical society, provided the drug is not listed as not indicated in compendia or contraindicated by the FDA. This applies to covered outpatient drugs furnished on or after January 1, 2027. (c) Private Health Insurance: It requires group health plans and health insurance issuers to provide an expedited appeals process for denials of coverage for drugs or biological products used to treat or manage rare diseases, if the use is supported by FDA-approved labeling or peer-reviewed literature and is not contraindicated. This applies to plan years beginning on or after January 1, 2027.