John Joyce and Wiley Nickel Aim to Promote Rare Disease Research by Amending the Inflation Reduction Act

By Better Today On May 9, 2024

TL/DR –

Reps. John Joyce (R-Pa.) and Wiley Nickel (D-N.C.) are pushing for a bill that would modify the Inflation Reduction Act (IRA) to protect research into treatments for rare diseases. The proposed ORPHAN Cures Act would ensure that orphan drugs, which treat rare diseases, are excluded from Medicare price negotiations even if they treat more than one condition. The congressmen argue that the bill, which has bipartisan support, is necessary to prevent disincentivizing the research into these diseases, many of which are more common than their label suggests.

Reps. Joyce and Nickel Advocate for Alterations to Inflation Reduction Act

During a recent event, Reps. John Joyce (R-Pa.) and Wiley Nickel (D-N.C.) discussed their push to pass the ORPHAN Cures Act. An alteration to the Inflation Reduction Act (IRA), this new legislation aims to secure ongoing research for rare disease treatments.

At “The Future of Cancer Care,” an event sponsored by AstraZeneca, Joyce and Nickel detailed the ORPHAN Cures Act. If successful, this act would amend the IRA, ensuring orphan drugs, essential for rare disease treatment, remain exempt from Medicare price negotiations.

Currently, the IRA does exclude orphan drugs from Medicare negotiation, but solely for medication treating a single disease. A drug would lose its orphan status if approved to treat another condition. The proposed ORPHAN Cures Act seeks to amend this language, excluding drugs that treat “one or more rare diseases or conditions.”

The bipartisan bill is vital, according to Joyce, as “maybe rare diseases aren’t that rare.” He clarified, “A rare disease is a disease that affects 200,000 or fewer Americans. ‘Rare diseases’ is perhaps a bit of a misnomer because rare diseases are ones we all recognize; like sickle cell disease, childhood cancers, diseases like Duchenne muscular dystrophy.”

Nickel further explained the necessity of modifying the IRA. He claimed the original IRA was enacted hastily, potentially disincentivizing research into rare diseases. Nickel described the ORPHAN Cures Act as one of the necessary “tune-ups” to the IRA, beneficial to everyone.