John Joyce and Wiley Nickel seek to amend the Inflation Reduction Act in support of rare disease research.

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The Importance of the ORPHAN Cures Act for Rare Diseases


Reps. John Joyce (R-Pa.) and Wiley Nickel (D-N.C.) highlighted the significance of their proposed bill that aims to amend the Inflation Reduction Act (IRA) to support ongoing research for treatments of rare diseases.

Discussion at The Hill Event

During The Hill’s event “Science & Policy, The Future of Cancer Care,” sponsored by AstraZeneca, Joyce and Nickel shed light on the ORPHAN Cures Act.

Key Amendment

If approved, the ORPHAN Cures Act would modify the IRA to ensure that orphan drugs treating rare diseases are not subject to Medicare price negotiations. These drugs are vital for conditions that would otherwise lack financial backing for development.

Enhanced Protection

The current IRA excludes orphan drugs from Medicare negotiation, but solely for medications targeting a single disease. The proposed amendment aims to safeguard drugs that treat “one or more rare diseases or conditions.”

Bipartisan Support

The bipartisan nature of the bill was emphasized by Joyce, who stressed the importance of defining rare diseases beyond their perceived rarity. Diseases like sickle cell disease and childhood cancers fall under this category.

Necessity for Amendment

Nickel elaborated on the urgency for modifying the IRA, citing the hasty nature of its enactment and the potential to hinder research into rare diseases. The proposed changes aim to ensure continued progress in this critical area.

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Read More of this Story at – 2024-05-09 21:44:00

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