Unless Congress makes a critical change, the Inflation Reduction Act (IRA) price controls could greatly reduce the number of new cures developed for rare diseases, writes Eric Dube, CEO of Travere Therapeutics and BIO Board member, in The Hill.
The problem: Orphan drugs for rare diseases are exempt from price controls—only if they’re approved for a single disease.
Why it matters: “One common reason companies can decide to pursue rare disease treatments is the promise that a successful drug might prove effective for multiple conditions,” writes Dube. “When a disease is rare, scientific progress that saves lives often depends on this approach.”
From personal experience: Dube started work in orphan drug development after surviving a rare cancer. Twenty years later, his company received accelerated approval for a rare kidney disease treatment; they’re seeking approval for a second rare kidney disease.
“The breakthroughs achieved might not have been possible” if these price controls existed a few years ago, Dube writes.
A possible cure: The ORPHAN Cures Act—sponsored by Representatives John Joyce, M.D. (R-PA) and Wiley Nickel (D-NC) and Senators Tom Carper (D-DE) and John Barrasso, M.D. (R-WY)—would allow orphan drugs to remain exempt from price controls, even if approved for use against a second disease.
The bottom line: “It’s not often that a minor change in a law’s text can save countless lives. But that’s why the ORPHAN Cures Act enjoys such broad, bipartisan support,” Dube writes. “For the sake of the 30 million Americans living with a rare disease, Congress needs to pass this bill as soon as possible.”
Read and share Dube’s opinion piece in The Hill.
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