ORPHAN Cures Act a “welcome and bipartisan fix” to the IRA’s negative impact on drug development 

September 18, 2023  | BIO

Representatives John Joyce, M.D. (R-PA) and Wiley Nickel (D-NC) today introduced the Optimizing Research Progress Hope And New Cures (ORPHAN Cures Act). BIO President and CEO, Rachel King, made the following remarks: 

“This legislation is a welcome and bipartisan fix to the Inflation Reduction Act’s (IRA) negative impact on the development of drugs to treat rare diseases.   

“Encouraging R&D for drugs to treat rare diseases is difficult as is. By definition, orphan drugs benefit small patient populations, making investment in this space incredibly risky. But there is tremendous need for these treatments. Fewer than 5 percent of rare diseases have an FDA approved treatment. We have repeatedly warned that the IRA — by subjecting drugs that can treat more than one rare disease to government price controls — creates even more barriers to investment into follow-on research and development for orphan drugs.   

“The new bill, introduced by Representatives John Joyce, M.D. (R-PA) and Wiley Nickel (D-NC), changes the incentive structure within IRA to encourage follow-on investment into orphan drug development. Reversing IRA’s perverse incentives will eliminate the significant barrier for scientists to usher in new waves of rare disease drug innovation – all to the benefit of the thousands of patients currently suffering from rare diseases for which no treatments exist.   

“BIO looks forward to working with lawmakers to get the Optimizing Research Progress Hope And New Cures (ORPHAN Cures Act) across the finish line as quickly as possible.”

Georgia Life Sciences Joins National Coalition Urging Congressional Action on Key Innovation Policies

December 10, 2025
Georgia Life Sciences has joined 43 state and regional life sciences organizations in signing a national Council of State Bioscience Associations (CSBA) letter calling on Congress to take immediate action on three bipartisan policy priorities that are essential to sustaining U.S. leadership in biomedical innovation and supporting patients nationwide. With Congress back in session and several critical programs at risk of expiring, the letter urges congressional leaders to advance the following provisions without delay: 1. Reauthorize the Rare Pediatric Disease Priority Review Voucher (PPRV) Program The PPRV program has been instrumental in incentivizing the development of therapies for children with rare and life-threatening conditions. Its lapse threatens to slow or halt research that families across the country are counting on. 2. Extend the SBIR/STTR Programs The Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) programs provide crucial early-stage capital for emerging biotech and medtech companies—many of them in Georgia. Without reauthorization, hundreds of innovative small businesses face uncertainty, jeopardizing new therapies, diagnostics, and technologies in the pipeline, 3. Advance PBM Transparency Reforms Greater transparency and accountability within pharmacy benefit manager (PBM) practices are needed to ensure that savings reach patients and employers. Reforming PBM operations is essential to strengthening access and affordability across the healthcare system. A Unified Message from the Life Sciences Community The sign-on letter reflects broad, bipartisan alignment across the national life sciences ecosystem: researchers, entrepreneurs, investors, patient advocates, and state associations all share a common message— these programs underpin America’s global competitiveness and are vital to patients who rely on continued scientific progress. Georgia Life Sciences has shared the letter with members of Georgia’s congressional delegation and will continue engaging with policymakers to emphasize the importance of swift action.

GLS Joins Georgia Partners in Calling for Federal PBM Reform

December 7, 2025
Georgia Life Sciences is proud to stand with patient advocates, providers, and employers across the state in urging Congress to take meaningful action on pharmacy benefit manager (PBM) reform. We recently signed on to a joint letter to Georgia’s Congressional Delegation stressing the urgent need for transparency, fair practices, and policies that ensure savings flow directly to patients.

GLS’ President and CEO, Maria Thacker Goethe in December's issue of Georgia Trend

December 4, 2025
Georgia Life Sciences’ CEO, Maria Thacker Goethe shares her perspective on the value of Georgia’s research universities in this month’s issue of Georgia Trend. “Georgia research institutions have felt a very significant impact from the federal rollbacks, specifically in NIH and NSF funding. Thacker Goethe’s message about the impact of research cuts is simple: Disruption shatters the foundational trust researchers have in grant continuity….” To read the full article, click here .
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