On December 27, 2020 Congress passed the Creating Hope Reauthorization Act. Under to this reauthorization, after September 30, 2024 FDA may only award vouchers for approved rare pediatric disease product applications if a company has a rare pediatric disease designation for the drug granted by the FDA by September 30, 2024.
Pursuant to the Creating Hope Act, a company that develops a drug for a pediatric rare disease – and receives FDA approval – also receives a voucher. The voucher comes with rights to faster FDA review of any future drug, enabling the voucher holder to receive an FDA “priority review” instead of a “standard review.” The voucher is transferable.
Pediatric vouchers have been sold for as much as $350 million and are now trading at $100 million. Over $2 billion of vouchers have been traded. Since the enactment of the Creating Hope Act, the FDA has approved 34 drugs expressly for pediatric rare diseases, two of which are pediatric cancer drugs.
Nancy Goodman, CEO of Kids v Cancer, stated: “I thank Representative G.K. Butterfield and Representative Michael McCaul, Senator Michael Bennet and Senator Susan Collins for their dedication to children with cancer and their commitment to shepherding the Creating Hope Reauthorization Act through Congress.
The Creating Hope Act has help children like Emma Whitehead, who was treated with the first FDA approved drug for a gene therapy, Novartis’ Kymriah, for B-cell precursor ALL. Kymriah earned a voucher.
The Creating Hope Act has made possible new pediatric cancer drug development for companies like Y-mABs Therapeutics. Y-mABs is using the Creating Hope Act pediatric priority review voucher program to develop exciting new therapies for children with neuroblastoma and other pediatric cancers.
Goodman said, “My son, Jacob, died of medulloblastoma, a pediatric cancer, ten years ago. I and every parent who lost a child to cancer, or who has cared for a child with cancer, asks Congress to pass the Creating Hope Reauthorization Act before its sunset on September 30, 2020. Now is the time for the Creating Hope Act to be permanently reauthorized. There is clear evidence of its impact. Moreover, there are two other voucher programs that were passed or modified after the passage of the Creating Hope Act that are permanent: a program for tropical disease drugs and a program for medical countermeasure drugs. It is time for the pediatric priority review voucher program to be permanent as well.”
“We thank Congress for reauthorizing the Creating Hope Act pediatric voucher program until September, 2024.”
The Creating Hope Act Permanent Reauthorization was endorsed by the following organizations and hospitals:
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,
Cancer Free 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 v 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 Foundation,
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.