1. Orphan Drug Policy Changes and Industry Incentives
The bill revives the Orphan Cures Act, which exempts drugs with multiple rare disease indications from Medicare price negotiations under the Inflation Reduction Act (IRA). Previously, only drugs treating a single rare disease were exempt, which industry leaders argued discouraged further research into additional rare indications. After the IRA, the percentage of drugs with an initial orphan designation that received a second designation dropped by 48% (from 12.1% to 6.3%).
The Congressional Budget Office (CBO) estimates this exemption will cost taxpayers nearly $5 billion over the next decade.
