ORPHAN Cures Act

This legislation, titled the ORPHAN Cures Act , seeks to amend the Social Security Act to expand and clarify the exclusion for orphan drugs from the federal Drug Price Negotiation Program . Its primary goal is to incentivize the development of treatments for rare diseases by adjusting how these drugs are treated under the program. Specifically, the bill mandates that any period a drug was designated as an orphan drug will not be counted when determining its eligibility for price negotiation, effectively extending its market exclusivity. Furthermore, it broadens the definition of an orphan drug to encompass those designated for one or more rare diseases or conditions , rather than just a single one, and clarifies this definition by referencing the Federal Food, Drug, and Cosmetic Act. These changes aim to provide greater protection and incentives for manufacturers developing therapies for underserved rare disease populations.