This legislative proposal, titled the PROTECT for Rare Act, seeks to significantly improve access to medications for individuals with rare diseases by amending federal health care laws. It broadens the criteria for what constitutes a "medically accepted indication" for drugs across Medicare, Medicaid, and private health insurance, specifically including those used to treat or manage diseases affecting 200,000 or fewer individuals in the United States. Under the bill, both Medicare Part B and Part D would cover drugs for rare diseases if their use is supported by peer-reviewed medical literature and not specifically contraindicated. Similarly, Medicaid's definition of "medically accepted indication" would be updated to include such uses, provided they are backed by peer-reviewed literature, clinical guidelines, or expert opinion. These changes are set to take effect on January 1, 2027. For private health insurance, the bill mandates that group health plans and health insurance issuers establish an expedited process for appealing coverage denials of drugs or biological products for rare diseases. This expedited review applies to FDA-approved or licensed drugs whose use is supported by their labeling or peer-reviewed literature, and not unfavorably reviewed or contraindicated. This provision aims to ensure more equitable and timely access to necessary treatments for rare conditions.
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Timeline
Introduced in Senate
Read twice and referred to the Committee on Finance.
Introduced in Senate
Read twice and referred to the Committee on Finance.
PROTECT for Rare Act
USA119th CongressS-3551| Senate
| Updated: 12/17/2025
This legislative proposal, titled the PROTECT for Rare Act, seeks to significantly improve access to medications for individuals with rare diseases by amending federal health care laws. It broadens the criteria for what constitutes a "medically accepted indication" for drugs across Medicare, Medicaid, and private health insurance, specifically including those used to treat or manage diseases affecting 200,000 or fewer individuals in the United States. Under the bill, both Medicare Part B and Part D would cover drugs for rare diseases if their use is supported by peer-reviewed medical literature and not specifically contraindicated. Similarly, Medicaid's definition of "medically accepted indication" would be updated to include such uses, provided they are backed by peer-reviewed literature, clinical guidelines, or expert opinion. These changes are set to take effect on January 1, 2027. For private health insurance, the bill mandates that group health plans and health insurance issuers establish an expedited process for appealing coverage denials of drugs or biological products for rare diseases. This expedited review applies to FDA-approved or licensed drugs whose use is supported by their labeling or peer-reviewed literature, and not unfavorably reviewed or contraindicated. This provision aims to ensure more equitable and timely access to necessary treatments for rare conditions.