Below is a statement from Florida Congressman Gus Bilirakis (R) regarding a bill titled “Creating Hope Reauthorization Act of 2024.”
U.S. Representatives Gus Bilirakis (R-FL), Anna Eshoo (D-CA), Michael McCaul (R-TX), Lori Trahan (D-MA), Michael Burgess (R-TX), and Nanette Barragán (D-CA). introduced the Creating Hope Reauthorization Act of 2024. This important legislation will reauthorize the Creating Hope Act of 2011, which expanded the FDA priority review voucher program to incentivize pharmaceutical companies to develop new drugs for children with rare pediatric diseases.
Children are not little adults, and despite their significant need many kids throughout the country are suffering with rare diseases that don’t have treatments or cures because of the small population impacted by their individual condition and the high cost of bringing these specific treatments to market,” said Rep. Bilirakis. “The Rare Pediatric PRV program has been an effective tool for incentivizing the development of new and improved treatments for children with cancer and other 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.
“Pediatric cancer is a leading cause of death among children, yet pharmaceutical companies avoid developing pediatric cancer drugs because of the small market size and high risks associated with studying and testing drugs in children,” said Rep. Eshoo. “This bill provides incentives for research and development by providing pediatric cancer drug developers with vouchers to speed up reviews by the Food and Drug Administration of new drug products. The Creating Hope Reauthorization Act will save children’s lives.”
“Since I started the Childhood Cancer Caucus in 2005, I have met with countless cancer patients and advocates who have asked me for one thing: hope,” said Rep. McCaul. “At no cost to taxpayers, the Creating Hope Act has already led to the development of dozens of treatments for rare pediatric diseases and continues to assure millions of children and their families that help is on the way. I urge my colleagues to pass its reauthorization to help keep hope alive as we work to end this disease once and for all.”
“As a mom to two young girls, all of my work in Congress is centered around creating a better future for them and the next generation – and improving our country’s health care is no exception. When it comes to beating pediatric cancer or other pediatric rare diseases, we need to use every tool at our disposal and make sure big pharmaceutical companies are playing their part,” said Rep Trahan. “I am proud to be part of this bipartisan group introducing the Creating Hope Reauthorization Act on behalf of thousands of kids bravely battling cancer and rare diseases each year.”
“Receiving a cancer diagnosis for a child is an experience no parent ever wishes to face. The Creating Hope Act stands as a beacon of hope, bolstering pediatric cancer research and paving the way for groundbreaking treatments to reach children in need,” said Rep. Michael C. Burgess, M.D. “Its passage is not just a legislative milestone; it’s a lifeline for young patients.”
“A cancer diagnosis is devastating news for any family to hear – including mine,” said Rep. Barragán. “Most cancers are considered rare because they occur in fewer than 15 out of 100,000 people each year, and all forms of childhood cancers are categorized as rare diseases. The majority of rare diseases do not have a FDA-approved treatment, and children are disproportionately represented among the rare disease patient population. The Creating Hope Act has incentivized the development of new drugs to treat childhood cancers and other rare pediatric diseases. I am proud to join my colleagues to reauthorize this critical program and ensure children have access to innovative, FDA-approved treatments.”
“As many as half of those living with a rare disease are children, and developing treatments for these patient populations is particularly challenging. The significant uptake in pediatric rare disease research and drug development in recent years demonstrates the importance of the incentives created by the rare pediatric disease priority review voucher program for our community. More than 95% of rare diseases still lack any FDA approved treatments and without a timely program reauthorization, we risk losing the vital progress we have made towards finding treatments for so many pediatric rare diseases. We are thankful to Reps. McCaul, Eshoo, Bilirakis, Barragan, Burgess, and Trahan for introducing this important bill, and urge Congress to quickly reauthorize the Rare Pediatric Disease PRV program before the September 30, 2024, deadline,” said Peter Saltonstall, President and CEO of National Organization for Rare Disorders (NORD).
“Nearly 3 out of 4 rare diseases originate in childhood and for the vast majority of children impacted by these diseases, there is no FDA approved treatment. The creation of the Pediatric Priority Review Voucher injected hope and innovation incentives into the pediatric drug development pipeline. We must continue to advance solutions for the estimated 30 million Americans living with rare diseases. The EveryLife Foundation for Rare Diseases extends our profound appreciation to Representatives McCaul, Bilirakis, Burgess, Barragán, Eshoo, and Trahan for leading the Creating Hope Reauthorization Act of 2024. This legislation ensures the sustainability of a critical incentive driving progress in drug development for devastating rare diseases that predominantly affect children, all without burdening taxpayers. We eagerly anticipate Congress’ action on this important legislation prior to the expiration of the Rare Pediatric PRV Program,” said Jamie Sullivan, MPH Vice President of Policy for EveryLife Foundation.
Background:
Children comprise as many as half of those living with rare diseases, yet their treatment options are extremely limited. Treatments intended for adults are often too harsh for children, leaving them with life-altering complications even after their disease is cured. The Creating Hope Act works to solve this problem by expanding the cost-neutral Food and Drug Administration priority review voucher (PRV) program, which allows pharmaceutical companies to expedite FDA review of more profitable drugs in return for developing treatments for rare pediatric diseases. Since the bill’s passage in 2011, 46 PRVs have been awarded for 35 different rare pediatric diseases. The Creating Hope Reauthorization Act of 2024 would reauthorize the Creating Hope Act — which expires this September — for another four years.
