Butterfield introduces bill to support children with Cancer and other rare diseases

Statement

Today, Congressman G. K. Butterfield, along with the other co-chairs of the Childhood Cancer Caucus, Representatives Michael McCaul (TX-10), Jackie Speier (CA-14) and Mike Kelly (PA-16) introduced the Creating Hope Reauthorization Act. The Caucus Leadership is joined by original co-sponsors Representatives Eliot Engel (NY-16), Gus Bilirakis (FL-12), Robin Kelly (IL-02), Richard Hudson (NC-08), Bobby Rush (IL-01) and Markwayne Mullin (OK-2). 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).

Pediatric cancer remains the number one disease that leads to the death of American children. While survival rates have improved for some types of pediatric cancers, thousands of children are lost to cancer each year and many more encounter life threatening complications related to harsh chemotherapies. Children have significantly fewer treatment options than adults and oftentimes must rely on treatment regimens developed for adults because pediatric-specific treatments simply do not exist for many pediatric cancers and rare diseases. Children are not little adults and despite their significant need, pharmaceutical companies have had trouble developing treatments for pediatric cancer and rare diseases because of the small population and high cost of bringing these specific treatments to market. To combat this, the Creating Hope Act incentivizes drug innovation by offering pharmaceutical and biotech companies a voucher that can be used for priority FDA review of an additional drug of their choosing in return for the development of a treatment for a rare pediatric disease.

"It is critical that children and their families have a permanent solution to address the great need to increase treatment options and possibly save lives. Every child who faces the devastation of a cancer diagnosis or a life-threatening disease deserves our best effort to provide support and ultimately, hope. As the co-chair of the Rare Disease Caucus and co-chair of the Childhood Cancer Caucus, I am proud to introduce this bill and to have the strong support of so many of my colleagues." -Rep. G. K. Butterfield (NC-01)

"Since the founding of the Childhood Cancer Caucus we have dedicated our time to uncovering every rock and pushing appropriations every step of the way to secure more funding for childhood cancer. With cancer being the leading cause of death among children, what could be a more noble cause than this? I'm grateful for everyone who gathered here today in support of this important cause -- to not just advocate, but actively make a difference in so many lives." -Rep. Michael McCaul (TX-10)

"Every three minutes a family hears the devastating diagnosis that their child has cancer. Our childhood cancer warriors deserve our finest resources and investment to discover the cures of tomorrow and improve quality of life. I'm proud to join my co-chairs of the Childhood Cancer Caucus in introducing the Creating Hope Reauthorization so that we can spur innovation in the fight against rare pediatric diseases and eradicate childhood cancer once and for all." -Rep. Jackie Speier (CA-14)

"There is more hope than ever in the fight against childhood cancer, and today we affirm our commitment to beat it! Thank you to the countless individuals who are in this arena every day, from doctors and scientists discovering new cures, policy-makers supporting their efforts, and the brave children who are battling each and every day for their lives. We can and will win this war for our kids!"-Rep. Mike Kelly (PA-16)

Endorsed By:

3/32 Foundation, Adam's Angels Ministry, Alex's Army Childhood Cancer Foundation, Amanda Riley Foundation, Association of Pediatric Hematology Oncology Nurses, Braden's Hope For Childhood Cancer, Bridge the Gap - SYNGAP Education and Research Foundation, Brooke's Blossoming Hope for Childhood Cancer, Brown University/Hasbro Children's Hospital, Caleb's Crusade for Childhood Cancer, Cancer Care Manitoba, CancerFree KIDS, Carson Leslie Foundation, CHEMOWARRIOR: the Eli Sidler Foundation, Children's Hospital of Philadelphia, Chloe's Journey of Faith Foundation, Christina Renna Foundation Inc., Circle of Care, Conner Cares, Curtana Pharmaceuticals, DADA2 Foundation, Daniel's House Publications, DBSA, EveryLife Foundation for Rare Diseases, FFAME (Family, Friends and Allies of ME/cfs, Foster Tree Service, Gabriella's Smile Foundation, Georgetown Lombardi Comprehensive Cancer Center, GIC Group/Global Food Safety Forum, Gold Rush Cure Foundation, Grace Way Foundation, Grandparents in Action, Harvard Medical School/ Massachusetts General Hospital, International Society of Paediatric Oncology (SIOP), Jack's Angels, Jeffrey's Voice, Joey's Wings Foundation,Jonah's Just Begun, Julia's Grace Foundation, Kids vs Cancer, Kids Without Cancer, Kier's Kidz, Lucy's Love Bus, Mackey Children's Cancer Foundation, MIB Agents, Michael Mosier Defeat DIPG Foundation, NanoValent Pharmaceuticals, National MPS Society, National Organization for Rare Disorders® (NORD), NF2 BioSolutions, Noah's Bandage Project, Noah's Hope, Oral Health Nursing Education and Practice, Pediatric Assessment, Learning & Support, Pediatric Cancer Foundation, Pediatric Valley Medical Center, Perlmutter Cancer Center at NYU Langone Health, Rally Foundation for Childhood Cancer Research, Rett's Roost, Sadie Keller Foundation, Samuel Jeffers Childhood Cancer Foundation Section 32, St. Jude Children's Research Hospital, Stephen T Marchello Scholarship Foundation, Super Sam Foundstion, Swifty Foundation, Syndax Pharmaceuticals, Team G Childhood Cancer Foundation, Teen Cancer America, The Cooper Max Foundation, The Cure Starts Now North Texas, The Dandy Horse, Inc., The Jesse Heikkila Foundation, The Maeve McNicholas Memorial Foundation, The National Children's Cancer Society, The Nicholas Conor Institute, The Progeria Research Foundation, The Rare Childhood Cancer Advocacy Group, The Scott Carter Foundation, The Steven G AYA Cancer Research Fund, The Valerie Fund, This Star Won't Go Out, Toby Knapp Radio, Wade's Army, We Believe Foundation, Wylie's Day Foundation, Y-mAbs Therapeutics Inc., Biotechnology Innovation Organization.

Background:

The Creating Hope Act was passed in 2012 and was re-authorized until 2020 as a part of the 21st Century Cures Act of 2016.
The Creating Hope Reauthorization of 2019 would permanently reauthorize this life-saving program.
The FDA has awarded vouchers for two pediatric cancer drugs: Unituxin (for high-risk neuroblastoma) and Kymriah (a CAR T for relapsed ALL Leukemia) and for 16 other pediatric rare disease medicines. There are also voucher programs for medical countermeasure drugs and tropical disease drugs.
While the priority review voucher program is permanent for medical countermeasure drugs and tropical disease drugs, it is not yet permanent for pediatric rare disease drugs.