These congressmen want to modify the Inflation Reduction Act to foster rare disease research

Reps. John Joyce (R-Pa.) and Wiley Nickel (D-N.C.) discussed Thursday why they’re pushing to pass their bill that would alter provisions of the Inflation Reduction Act (IRA), with the congressmen saying the legislation is needed to ensure continued research into treatments for rare diseases.

Joyce and Nickel discussed the ORPHAN Cures Act while speaking at The Hill’s event “Science & Policy, The Future of Cancer Care,” sponsored by AstraZeneca.

The ORPHAN Cures Act, if passed, would amend the IRA to ensure that orphan drugs are excluded from Medicare price negotiations. Orphan drugs are medications that treat rare diseases that drugmakers would have no financial incentive to develop without financial assistance from the government.

The IRA already excludes orphan drugs from Medicare negotiation, but this provision only applies to medication that treats a single disease. An orphan drug would lose its distinction if it was approved to treat another condition. 

The ORPHAN Cures Act would amend the language in the IRA to exclude drugs that treat “one or more rare diseases or conditions.”

The bill has bipartisan support, and Joyce told The Hill’s Editor-in-Chief Bob Cusack that passing it is “so important” because “maybe rare diseases aren’t that rare.”

“We have to define what a rare disease is. A rare disease is a disease that affects 200,000 or fewer Americans,” Joyce said. “‘Rare diseases’ is perhaps a bit of a misnomer because rare diseases are diseases that we all recognize; like sickle cell disease, like all childhood cancers, like diseases like Duchenne muscular dystrophy.”

Nickel further explained why the legislation modifying the IRA was necessary.

“It should get some attention. And the Inflation Reduction Act was done very hastily. There wasn’t a lot of focus on how the work they did in the last Congress could disincentivize the research into these rare diseases,” Nickel said.

“It’s just one of the tweaks, tune-ups to the Inflation Reduction Act to help it continue to move forward and away that’s helpful for everyone,” he added.