Butterfield’s Bill Supporting Children with Cancer and Other Rare Diseases Passes Congress

Dec 22, 2020
Press Release

WASHINGTON, D.C. – Today, Congressman G. K. Butterfield, along with his fellow co-chairs of the Childhood Cancer Caucus, Representatives Michael McCaul (TX-10), Jackie Speier (CA-14) and Mike Kelly (PA-16) released the following statement on the inclusion of the Creating Hope Reauthorization Act in the omnibus appropriations package: 

“The Creating Hope Reauthorization Act reauthorizes the rare pediatric disease priority review voucher program at the Food and Drug Administration (FDA).  This program plays a critical role in incentivizing life-saving and life-changing research and therapy development for a particularly vulnerable and underserved patient community—children with rare diseases,” said Congressman Butterfield.  “There are over 7,000 rare diseases, half of which impact children, few of which have FDA approved treatments.  Ensuring this bill’s inclusion in the omnibus spending package demonstrates Congress’s commitment to providing hope for every child who faces the devastation of a cancer or a life-threatening rare pediatric disease diagnosis.  As co-chair of the Childhood Cancer Caucus, I celebrate this progress and look forward to the president signing this reauthorization into law.  I will continue to support the needs of this vulnerable population and their families.” 

“Before the Creating Hope Act, there were only two drugs developed to specifically treat pediatric cancer.  Since the bill’s original passage, 22 new drugs for rare diseases in kids had been approved by the FDA, including two drugs for childhood cancers with more expected on the way,” said Congressman McCaul.  “The reauthorization of this voucher program will continue to produce life-saving treatments for children in need.  I am proud to join my colleague, G. K. Butterfield, in advocating for this critical piece of legislation.”

“Every 3 minutes a family must hear the earth-shattering news that their child has cancer.  No child should be left behind because the market for treatments for pediatric rare diseases and cancers is too small,” said Congresswoman Speier.  “Every child is worth fighting for.  Thanks to the Creating Hope Act, we’ve made meaningful progress incentivizing research into life-saving treatments and cures for children, and with this reauthorization we’ll continue to build on these efforts.”

“America’s children are our future, and nothing is more important than their health.  I am so grateful that the Creating Hope Reauthorization Act, which encourages the development of cures and treatments for rare diseases that affect our kids, will be enacted,” said Congressman Kelly.  “Thank you to my colleagues in the Childhood Cancer Caucus for standing up for our brave little warriors as they fight for their lives and futures.  Together, we can and will end pediatric cancer.”


The Alliance for Childhood Cancer:

“The Alliance for Childhood Cancer is very pleased to see the Creating Hope Reauthorization Act included in the Fiscal Year 2021 appropriations package, and the Alliance applauds Congressman Butterfield, Congressman McCaul, Senator Casey, and Senator Collins for their leadership in the fight against childhood cancer.  While recent decades have seen dozens of new FDA-approved cancer therapies, only a handful of treatments for childhood cancers have been approved over the same period.  The Creating Hope Act expanded the FDA priority review voucher program to incentivize pharmaceutical manufacturers to invest in drugs with indications for rare pediatric diseases.  Through this program, the FDA has awarded vouchers for two pediatric cancer drugs, dinutuximab (for high risk neuroblastoma) and CAR T-cell therapy tisagenlecleucel (for relapsed acute lymphoblastic leukemia), offering new hope for children with cancer.  The Creating Hope Act Reauthorization Act reauthorizes this important program that expands treatment options for children with cancer and their families and continues to advance the research that brings us closer to curing childhood cancers.”

National Organization for Rare Disorders (NORD):

“NORD supports the reauthorization of this important incentive that is designed to bring much-needed treatments to pediatric patients with rare diseases.  NORD appreciates the hard work by Members of Congress and their staffs to advance this legislation.”

EveryLife Foundation for Rare Diseases:

"The EveryLife Foundation for Rare Diseases applauds the critical Congressional action that led to the extension of the Pediatric Rare Disease Priority Review Voucher (PRV) Program at the FDA as proposed in the Creating Hope Reauthorization Act.  This innovative and cost-effective program has been a powerful incentive for the development of treatments for rare diseases and has enabled life-saving treatments to reach children with rare diseases faster without adding new costs to taxpayers.  Children and families with devastating and life-threatening rare diseases are grateful to you for continuing the PRV program."



The Creating Hope Act incentivizes the pharmaceutical industry to develop drugs for children with cancer and other life-threatening illnesses through the rare pediatric disease priority review voucher program at the U.S. Food and Drug Administration (FDA).  Historically, children with rare pediatric diseases have lacked treatments and therapies because the market it so small. The rare pediatric disease priority review voucher program was created to correct that market failure and provide a compelling incentive for biotech and pharmaceutical companies to invest in bringing rare pediatric disease treatments to market. Since the program’s creation in 2012, the FDA has awarded 22 priority review vouchers as part of this program.