California Life Sciences Supports the ORPHAN Cures Act to Advance Rare Disease Research

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FOR IMMEDIATE RELEASE: Feb 5, 2025

South San Francisco, CA – Today, California Life Sciences (CLS) is pleased to see the introduction of the Optimizing Research Progress Hope and New (ORPHAN) Cures Act (H.R.946). The legislation – a bipartisan initiative championed by Representatives John Joyce, M.D. (R-PA) and Don Davis (D-NC) along with others – strengthens the pipeline of treatments for rare diseases.

This act comes at an urgent time – fewer than 10% of the 7,000+ known rare diseases have FDA-approved treatments. The ORPHAN Cures Act tackles the unintended consequences of the Inflation Reduction Act (IRA), which discourages continued research into rare diseases by making products prematurely eligible for Medicare price setting after just one rare disease designation.

“When it comes to rare disease research, every scientific advancement builds upon previous discoveries. This legislation ensures that the iterative nature of scientific progress isn’t hindered, allowing for the development of rare disease treatments for patients suffering from conditions that have no available treatments,” said Mike Guerra, CEO and President of CLS. “Promoting continued research will allow the life sciences sector to protect the pipeline of potential treatments for millions of Americans struggling with rare diseases.”

California’s strong life science ecosystem has been a global leader when it comes to the high-risk, high-reward research necessary for developing treatments for small patient populations. The ORPHAN Cures Act will ensure that California, and the United States at large, remains at the forefront of the scientific development of treatments and cures where there is unmet medical need.

No time should be wasted. CLS urges Congress to act now and advance this legislation to allow the life sciences industry to develop treatments for patients suffering from rare diseases, including the majority who await their first treatment option.

 

About California Life Sciences
California Life Sciences (CLS) is the state’s leading advocacy organization for the life sciences, promoting the sector and its diverse innovation pipeline. For more than 30 years, CLS has served the community by supporting companies of all sizes, from early-stage startups to established biotechnology, pharmaceutical, and medical technology industry leaders.

 

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For media inquiries, please contact:
Katie Kiss
Senior Director, Marketing
California Life Sciences
[email protected]