This bill changes how orphan drugs are defined and priced, helping to promote the development of treatments for rare diseases. It aims to make these drugs more accessible and affordable for patients who need them.
Optimizing Research Progress Hope And New Cures Act or the ORPHAN Cures Act This bill modifies certain provisions under the Medicare Drug Price Negotiation Program with respect to orphan drugs. The Medicare Drug Price Negotiation Program requires the Centers for Medicare & Medicaid Services to negotiate the prices of certain prescription drugs under Medicare beginning in 2026. Among other requirements, drugs must have had market approval for at least 7 years (for drug products) or 11 years (for biologics) to qualify for negotiation. The program does not apply to orphan drugs that are approved to treat only one rare disease or condition. The bill modifies these provisions so as to exclude any period in which a drug was an orphan drug from market approval calculations. It also excludes orphan drugs that are approved to treat more than one rare disease or condition from the program.
1. This bill expands the definition of orphan drugs to include those for multiple rare diseases. 2. It allows for longer time periods to be considered when pricing these drugs. 3. The bill clarifies how orphan drugs are treated under the Drug Price Negotiation Program. 4. It aims to encourage research and development of treatments for rare diseases.