ORPHAN Cures Act

The ORPHAN Cures Act would revise the Social Security Act to broaden and clarify how orphan drugs are treated in Medicare’s Drug Price Negotiation Program. Specifically, it codifies that time periods during which a drug was an orphan drug should not count toward the elapsed-time calculation used to determine when a drug is subject to negotiation or other program milestones. It also expands and clarifies the definition of what qualifies as an orphan drug by aligning it with the FDA’s “rare disease or condition” standard, and by allowing the designation to cover one or more such diseases or conditions (instead of just one). In short, the bill strengthens protections for drugs that have or once had orphan status and tightens the definitional framework around which drugs are excluded from price negotiation. Potential impacts include a longer interval before certain drugs enter the negotiation program (or a longer phase during which price negotiation is not required), greater alignment with FDA orphan-drug definitions, and a broader scope for what counts as an orphan drug under the exclusion.