Three Bilirakis Priorities to Help Children with Rare Diseases Pass House

As Co-Chair of the Rare Disease Caucus, Congressman Bilirakis has worked tirelessly to support rare disease patients and families by streamlining FDA processes and encouraging the development of treatments and cures for smaller patient populations. As part of that work, he co-authored the RARE Act, which offers a technical fix that provides regulatory certainty for rare disease drug development and eliminates ambiguity by codifying the FDA's long-standing interpretation that orphan drug exclusivity is to be awarded based on a product's FDA approved indication, rather than the entire disease. Bilirakis also co-leads legislation to reauthorize the Creating Hope Act of 2011which originally expanded the FDA Priority Review Voucher (PRV) program to incentivize pharmaceutical companies to develop new drugs for children with rare pediatric diseases. Since its inception, 53 PRVs have been awarded across 39 different rare pediatric diseases. Due to their small patient populations, pediatric rare diseases are too often left behind due to the small market size and high risks associated with clinical trials for these patients. The Creating Hope Reauthorization Act continues this critical priority review voucher program to incentivize companies to engage in this life-saving work for another five years. "The Rare Pediatric PRV program has been an effective tool for incentivizing the development of new and improved treatments for children with rare diseases. I'm proud to help lead the charge in offering hope to these families by ensuring this successful initiative continues to help rare disease patients nationwide, remarked Bilirakis. During the committee process, both of these Bilirakis bills were amended into the Give Kids a Chance Act, which requires companies to study combination products of pediatric cancers. This comprehensive legislative package passed the House unanimously last night.