Small fix to IRA, big boost for rare disease

Biotechnology Innovation Organization (BIO)

January 9, 2024

A small fix to the Inflation Reduction Act could boost rare disease R&D—and voters support it. Plus, researchers say they found the gene that allows wheat to grow in extreme temperatures. (487 words, 2 minutes, 26 seconds)

A small fix to IRA, a big boost for rare disease

A small change to the Inflation Reduction Act (IRA)—supported by voters—would make a big difference for patients with rare diseases, writes BIO Chair Dr. Ted Love in RealClearHealth.

Developing orphan drugs for rare diseases is challenging: “Such small patient populations create enormous financial and practical barriers to developing cures and therapeutics,” writes Dr. Love.

They’re exempt from price controls—but only if they’re approved to treat one disease. However, nearly a quarter of orphan drugs approved since 2003 “were later approved to treat additional diseases,” he says.

“As a result, the IRA discourages researchers from investigating whether existing orphan drugs have additional uses or at best, it incentivizes delaying launching the drug as long as possible. This would deal an enormous blow to the rare-disease research community,” he explains.

A solution:The ORPHAN Cures Act, which would change the IRA’s incentive structure to encourage follow-on investment—and has bipartisan backing in the House and Senate.

What voters want: 94% of American voters consider rare disease drug research important, and 85% think it’s critical for the government to “expand incentives for rare disease drug development,” finds a poll conducted by Morning Consult for BIO.

The bottom line: “It’s hard to believe the IRA’s architects actually intended to derail research into rare diseases. Yet that’s exactly what’s happening. Lawmakers can reverse course and correct the problem by passing the ORPHAN Cures Act,” Dr. Love concludes.

Researchers find gene that enables wheat to grow in extreme temperatures

Editing one gene in wheat could create a new crop able to reproduce in extremely cold or warm weather, researchers announced last week.

Why it matters: Wheat meiosis—when chromosomes from parent cells pair to create seeds for the next generation—normally occurs at temperatures between 17-23C. Increasing this range would make it possible to grow wheat in extreme temperatures.

The discovery: Researchers at the John Innes Centre for plant science in the U.K. say they have proven DMC1, a meiotic gene in wheat, is responsible for allowing reproduction at extreme temperatures. They conducted experiments using CRISPR-Cas9 technology to remove DMC1 from wheat.

The next step: Finding variations of DMC1, and different dosages and expressions of the gene, to increase the temperature range for wheat meiosis.

Gene editing is already climate-proofing wheat,developing a new variety that can better tolerate drought. But regulatory acceptance is essential to this crop becoming a widely used solution.

What we need now: Smarter, streamlined regulation to allow biotech solutions that can help us mitigate and adapt to climate change, as BIO has explained.

BIO Beltway Report

President Biden’s Tuesday: No official events scheduled. Meanwhile, Health and Human Services (HHS) Secretary Xavier Becerra announced the appointment of Stacy Sanders to the role of Chief Competition Officer, a new role created to help “lower health care and prescription drugs costs by promoting competition,” explains the agency.

What’s Happening on Capitol Hill: With a deal reached Sunday, Congress has until Friday, January 19 to hammer out the details and avert a partial government shutdown.