ORPHAN Cures Act a "welcome and bipartisan fix" to the IRA's negative impact on drug development

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."