Legislative WIN for the EB Community!

This week marks a significant milestone for children and families affected by Epidermolysis Bullosa (EB) and the broader rare disease community.  

After passing the Senate last week and the House this week, the federal budget, which includes two of debra of America’s top legislative priorities, the Accelerating Kids’ Access to Care Act and the Mikaela Naylon Give Kids a Chance Act, has now been signed into law by the President, securing a major victory for the EB Community. 

The Mikaela Naylon Give Kids a Chance Act reauthorizes the Rare Pediatric Disease Priority Review Voucher (RPD PRV) Program, which Congress allowed to expire last year. This program is a critical tool for accelerating the development of treatments for the estimated 15 million children in the U.S. living with a rare disease. 

Under this program, when a company receives FDA approval for a rare pediatric therapy, it can earn a “priority review” voucher. That voucher can either be used to fast-track the review of another drug or sold to another company, creating powerful incentives for investment in rare disease research. Since the program’s inception, more than 60 vouchers have been awarded for treatments targeting 40 pediatric diseases, all at no cost to taxpayers. Reauthorizing it helps ensure that innovative therapies, including those for EB, can continue moving from the lab to patients who urgently need them. 

Equally impactful is the Accelerating Kids’ Access to Care Act, which tackles dangerous delays in medical care when children must travel across state lines for specialized treatment. 

Currently, when providers treat out-of-state Medicaid patients, they must go through a separate credentialing process with the patient’s home state, a bureaucratic hurdle that can take months. For children with complex medical needs, including those requiring cutting-edge cell and gene therapies, these delays can be life-altering. 

This legislation will streamline credentialing for specialty providers, helping ensure that children with rare diseases receive timely, coordinated care when and where they need it most. 

This achievement would not have been possible without the passionate advocacy of our EB Community. Thank you for your persistence and your partnership.