California Life Sciences Urges Congress to Support Rare Disease Patients


California Life Sciences (CLS) is pleased to support the Optimizing Research Progress Hope and New (ORPHAN) Cures Act, introduced by Reps. John Joyce, M.D. (R-PA) and Wiley Nickel (D-NC). The bipartisan legislation would fix the unintended consequence created by the Inflation Reduction Act (IRA) that hinders the development of orphan drugs and disincentivizes research into additional rare disease indications.

“The ORPHAN Cures Act is an important piece of legislation that will spur and support the riskiest biomedical innovation into life-saving treatments and cures for patients that desperately need them,” said Mike Guerra, CEO and President of CLS. “Fewer than ten percent of rare diseases have an FDA approved treatment, and there is a critical need for investment in rare disease research to provide hope to the thousands of patients currently suffering from conditions without available treatments.”

“With the tremendous scientific breakthroughs we have seen in the past decades, it is easy to forget that science is most often an iterative process that builds on prior learnings. The ORPHAN Cures Act would allow our sector to continue to build on these successes in the rare disease space, as opposed to cutting this process short by making these products eligible for Medicare price setting after one rare disease indication.”

CLS urges Congress to quickly advance this legislation to ensure that our membership, which includes the most innovative companies in the world, can continue to invest in additional research for rare disease patients.