PROTECT for Rare Act

The PROTECT for Rare Act (S 3551) requires Medicare, Medicaid, and private health insurers to establish an expedited appeal process for coverage denials of drugs treating rare diseases or conditions affecting 200,000 or fewer U.S. individuals. It expands coverage criteria by allowing insurers to consider peer-reviewed medical literature and clinical guidelines - not just FDA-approved labeling - as valid justification for treatment, while excluding uses listed as contraindicated in approved drug labeling or medical references. The law applies to all covered drugs used for rare conditions and takes effect for coverage decisions starting January 1, 2027. This directly affects patients with rare diseases, healthcare providers seeking coverage approvals, and insurers managing drug benefit denials.