RARE Act

The Retaining Access and Restoring Exclusivity (RARE) Act would clarify how the seven-year market exclusivity period works for drugs treating rare diseases. Currently, when a pharmaceutical company gets FDA approval for a drug to treat a rare disease, it receives seven years of market exclusivity. This bill would specify that this exclusivity protection applies only to the specific approved use or patient population for which the drug was approved, not to the entire rare disease category. This change responds to a 2021 court decision that interpreted the law more broadly, which prevented FDA from approving a different manufacturer's version of the same drug for a different patient population within the same rare disease. The bill would allow FDA to approve competing treatments for different patient groups or uses within the same rare disease, potentially giving patients more treatment options. Supporters argue this would prevent pharmaceutical companies from blocking access to innovative treatments for rare disease patients, especially children, while still maintaining incentives for drug development.