The Creating Hope Act


Two weeks after Kids v Cancer Founder Nancy Goodman’s eight year old son, Jacob, was diagnosed with medulloblastoma, a rare form of pediatric brain cancer, doctors gave him a round of chemotherapy. Jacob’s tumors did not respond. Why then, didn’t Jacob’s medical team change his chemotherapy protocol? Because in the last 30 years, no new drugs have been developed to treat Jacob’s form of cancer. In fact, in the past 20 years, only one drug has been expressly developed for any form of pediatric cancer. Jacob died on January 16, 2009 when he was ten years old.


There is a crisis in the state of drug development for pediatric cancers and other pediatric rare diseases.

 

 

In 20 years the FDA has initially approved only one drug for any childhood cancer • 1/2 of all chemotherapies used for children’s cancers are over 25 years old. • Research and development for new drugs from pharmaceutical companies comprises 60% of funding for adult cancer drugs and close to zero for childhood cancers. • However, the NCI spends 96% of its budget on adult cancers and only 4% of its budget on children’s cancers.

The Creating Hope Act of 2011 provides market incentives to pharmaceutical companies to develop drugs for pediatric rare diseases.

Get involved.
Help pass the Creating Hope Act.
We need your support.


PRESS INFORMATIONDETAILS OF THE CREATING HOPE ACT

Creating Hope Act of 2011 – A Priority Review Voucher for Pediatric Rare Diseases

Why create a pediatric rare disease priority review voucher?
Over the past 20 years, there have been almost no drugs developed specifically for pediatric cancers or other pediatric rare diseases. Pharmaceutical companies currently lack the financial incentives to develop new drugs because the market for drugs for rare pediatric diseases is so small. The only drugs available to children are hand-me-down drugs that were developed for adult diseases. This is problematic because, for example, pediatric cancers are fundamentally different from adult cancers in the nature of their causes, the appropriate approaches to their treatment and the impact of their late effects.

The FDA has approved only one new drug expressly developed for a pediatric cancer in the past 20 years (vs. approving hand-me-down drugs developed for adult cancers).

  • A recent Institute of Medicine Report found that half the drugs used for pediatric cancer treatment are over 25 years old.
  • Still, children in the United States suffer from more than 6,000 rare diseases (defined as having a prevalence of 200,000 or less).  Of those 6,000 diseases, only 200 have drug treatments.

The Creating Hope Act
The Creating Hope Act of 2011 would encourage the creation of new drugs for underserved children who suffer from serious and rare medical conditions, including life-threatening cancers, by providing a voucher to pharmaceutical companies who develop such drugs. This voucher could be used to secure expedited FDA approval for any other drug — particularly a blockbuster drug– so that that drug could be delivered to market faster. The voucher would constitute a strong, market incentive for pharmaceutical companies to develop drugs for children with serious and rare diseases, such as cancer. The Act builds on the “FDA Amendments Act of 2007,” which established a voucher for drug development for neglected tropical diseases.

Which drugs designed for pediatric rare diseases would qualify for a voucher?
The priority review voucher program would award a voucher to a company that gains FDA approval for a new drug application designed for a pediatric rare disease. To be eligible for a priority review voucher, the new drug application for the pediatric rare disease must be:

  • Eligible for priority review by the FDA
  • A new drug  that has not been previously approved by the FDA
  • For a pediatric indication
  • For a disease that has received Orphan Disease Act designation

Why would a pharmaceutical company choose to exercise or buy a voucher?
The voucher would reduce the FDA review time for the non-pediatric rare disease drug that would not otherwise receive an FDA priority review by 4-12 months. The sooner companies put a drug on the market, the faster they begin to earn revenue.

Earlier market entry also gives pharmaceutical companies a greater advantage over competitor companies.

How would vouchers reward pharmaceutical companies that want to develop drugs for rare pediatric diseases?

  • Experts have estimated a voucher could potentially be worth hundreds of millions of dollars.
  • Pharmaceutical companies that do not have a top-selling drug could sell the voucher to another company.
  • Pharmaceutical companies could use the prospect of receiving a voucher to raise additional capital.

How would the voucher work?
For example, if Pharmaceutical Company A received FDA approval for a drug for a pediatric rare disease, it would also receive a priority review voucher. Pharmaceutical Company A could keep the priority review voucher or sell it to Pharmaceutical Company B, which was planning to submit a new drug application for hair loss to the FDA for approval. Pharmaceutical Company B could then exercise the voucher and receive a priority review for the hair loss drug instead of the standard review the new drug application would have otherwise received.

Description of the legislation
The Creating Hope Act would extend Section 524 of the Federal Food, Drug, and Cosmetic Act (FDCA), enacted in 2007, which creates a priority review voucher for tropical diseases, by extending it to another set of neglected diseases: pediatric rare diseases.

  • Extension to pediatric rare diseases: This legislation includes rare pediatric disease within the scope of the program. This category encompasses any disease that is “rare” within the meaning of the Orphan Drug Act (affects less than 200,000 people, or the cost of development would exceed revenue) is recognized in the medical community as affecting a pediatric population and is a new drug that has not received FDA approval for an adult indication.
  • Closing a loophole: This legislation would prevent companies from receiving a voucher for tropical disease products that they already market in other countries. This change will ensure that the program rewards only innovative treatments.
  • Unlimited transferability of vouchers: A voucher may now be transferred unlimited times provided that the transferee, in each instance of transfer, notifies the FDA of the change in ownership. This change enables drug companies to maximize the value of the voucher in the marketplace.
  • Optional upfront priority review designation process: Under the current law, sponsors do not know whether their new drug application will qualify for a voucher until the time of FDA approval. The proposed legislation permits sponsors of both tropical disease drugs and rare pediatric disease drugs to seek a designation that the new drug would qualify for a voucher, should it be approved, even before they submit their new drug application.
  • Adds Chagas disease to the list of neglected tropical diseases: Chagas disease is responsible for more deaths in Central and South America than every other parasite-borne disease, including malaria. Yet, despite its profound impact, research and development of new treatments is severely underfunded. The addition of Chagas to the list of eligible diseases fulfils the intent of the original authors.
  • Reporting and marketing requirements: The Creating Hope Act requires that the sponsor submit a statement of good faith intent to market the eligible drug, as well as a report describing the demand and distribution of the ultimate product. These provisions will help to ensure that the drugs at issue get to the patients that need them in the first place.

 

List of Endorsers

Aeras Global TB Vaccine Foundation
American Childhood Cancer Organization
American Porphyria Foundation
American Society of Clinical Oncology
American Society of Pediatric Hematology and Oncology
American Society for Tropical Medicine and Hygiene
Association of Pediatric Oncology Social Workers
Bio Ventures for Global Health
Biotechnology Industry Organization (BIO)
Chai Lifeline
Children’s Brain Tumor Foundation
Children’s Cause for Cancer Advocacy
Children’s Hospital Association of Texas
Children’s Hospital of Philadelphia
Children’s Hospital of Pittsburgh at UPMC
Children’s Medical Center Dallas
Children’s Oncology Group (COG)
CSL Behring
CureSearch
Friends of Scott Foundation
Genzyme
GlaxoSmithKline
Kakki’s Every Life Foundation
Kids v Cancer
Leukemia and Lymphoma Society
Madison’s Foundation
Max’s Ring of Fire
Medicines for Malaria Venture (MMV)
Merck
Metronomix
National Adrenal Disease Foundation
National Association of Children’s Hospitals
National Brain Tumor Society
National Children’s Cancer Society (NCCS)
National Tay-Sachs and Allied Diseases Fund
National Organization for Rare Diseases (NORD)
Novartis
People Against Childhood Cancer (PAC2)
Pennsylvania BIO
Rady Children’s Hospital
Rally Foundation, The
Rodney White Children’s Foundation
Sarcoma Foundation of America
Seany Foundation, The
Shire
Sickle Cell Disease Association of America (SCDAA)
St. Baldrick’s Foundation
TB Alliance
Texas Children’s Hospital

 

  1. Institute of Medicine. “Making Better Drugs for Children with Cancer.” 2005. p 9. http://www.nap.edu/catalog/11259.html

  2. Henry G. Grabowski, David B. Ridley, and Jeffrey L. Moe. “Priority Review Vouchers to Encourage Innovation for Neglected Diseases.” In: K. Eggleston, ed. Prescribing Cultures and Pharmaceutical Policy in the Asia-Pacific. Brookings Institution Press. 2009. http://faculty.fuqua.duke.edu/~dbr1/research/priority.pdf

  3. Waseem Noor. “Placing Value on FDA’s Priority Review Vouchers.” In Vivo. September 2009. http://www.imscorprep.com/tl_programs/documents/IMS0909iv.pdf

  4. David B. Ridley, Henry G. Grabowski, and Jeffrey L. Moe. “Developing Drugs for Developing Countries.” Health Affairs. 2006. Vol. 25, No. 2: 313-24. http://content.healthaffairs.org/cgi/content/abstract/25/2/313

Comments are closed.