TL/DR –
The ORPHAN Cures Act has been introduced by Reps. John Joyce (R-Pa.) and Wiley Nickel (D-N.C.) to alter the Inflation Reduction Act and ensure that orphan drugs, medications that treat rare diseases, continue to be excluded from Medicare price negotiations. The proposed amendment would adjust the Act’s language to exclude drugs that treat “one or more” rare diseases or conditions, rather than just a single disease. The lawmakers argue that the legislation is crucial for the continuation of research into treatments for rare diseases as these drugs would not be financially viable for development without government support.
Pushing for Changes in the Inflation Reduction Act
Reps. John Joyce (R-Pa.) and Wiley Nickel (D-N.C.) are advocating for amendments to the Inflation Reduction Act (IRA), specifically the ORPHAN Cures Act provisions, to reinforce the ongoing research for rare diseases treatments.
Discussion at The Future of Cancer Care Event
The proposed changes were discussed by the Congressmen at an event on science and policy hosted by The Hill and sponsored by AstraZeneca.
ORPHAN Cures Act Amendments
The ORPHAN Cures Act aims to refine the IRA, preventing orphan drugs – medications for rare diseases often financially supported by the government – from being included in Medicare price negotiations. The current IRA provisions allow orphan drugs exclusion only if they treat a single disease, which can change if the drug gets approval to treat another condition.
Refining the Definition of Orphan Drugs
The amendments will adjust the language in the IRA to exclude drugs treating “one or more rare diseases or conditions.” The bipartisan-supported bill, according to Joyce, is crucial as “rare diseases aren’t that rare.” Rare diseases are defined as affecting 200,000 or fewer Americans, including conditions such as sickle cell disease, childhood cancers, and Duchenne muscular dystrophy.
The Need for Legislation Modification
Nickel argued that the swift implementation of the Inflation Reduction Act overlooked potential disincentives for researching rare diseases. He termed the proposed changes as necessary “tune-ups” to the Act to make it beneficial for everyone.
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