Representatives John Joyce, M.D. (R-PA) and Wiley Nickel (D-NC) have recently introduced the Optimizing Research Progress Hope And New Cures (ORPHAN Cures Act). This legislation is a significant step in addressing the negative impact of the Inflation Reduction Act (IRA) on developing drugs to treat rare diseases. IBIO President & CEO John Conrad made the following remarks.

“We are pleased to endorse the Optimizing Research Progress Hope And New Cures Act, which represents a vital bipartisan effort to reduce barriers to research and innovation in the rare disease space. As the trade association representing over 91,000 bioscience jobs in Illinois, we recognize the significant need for investment in treatments for rare diseases. Fewer than 5 percent of rare diseases have an FDA approved treatment, leaving patients without options and researchers without adequate support.

The Inflation Reduction Act (IRA) has significantly deter investment in rare disease research. This legislation creates a disincentive for scientists to invest in follow-on research and development for orphan drugs that can treat multiple rare diseases, limiting progress and stifling innovation. The ORPHAN Cures Act will address these issues by changing the incentive structure within the IRA, encouraging further investment in orphan drug development.

We are dedicated to working with lawmakers to ensure that the ORPHAN Cures Act is passed as quickly as possible, as it represents a crucial step forward in the fight against rare diseases. We urge Congress to recognize the critical need for investment in rare disease research, and to support this bipartisan effort to right the wrongs of the IRA. By doing so, we can help usher in new waves of rare disease drug innovation and support the thousands of patients currently suffering from conditions without current treatments available.”