ORPHAN Cures Act

This proposal changes how Medicare can negotiate prices for prescription drugs that treat rare diseases. It says that any time a medicine qualified as an “orphan drug” for a rare disease won’t count toward the age limits (7 years for drugs, 11 years for biologics) that make a drug eligible for negotiation. It also says orphan drugs are excluded from negotiation even if they are approved to treat more than one rare disease, not just a single one. The Medicare negotiation program is set to start in 2026, so this would adjust those rules before and as they take effect .

What this could mean for you: medicines for rare diseases may be less likely to face Medicare price negotiations, which supporters say could encourage more research and approvals for rare conditions. However, it could also mean some higher-cost rare disease drugs stay expensive for longer for people on Medicare.

• Who is affected: people with rare diseases, Medicare patients, and drug makers.
• What changes: time on the “orphan drug” label doesn’t count toward negotiation eligibility; drugs for one or more rare diseases are excluded from negotiation.
• When: applies to the Medicare negotiation program beginning in 2026.