Dear Friends of Kids v Cancer,
Thank you. It has been a banner year.
We have created almost one billion dollars in incentives for new drug research for children
With your support, we have mobilized almost a billion dollars for research and development of drugs for seriously ill children.
Moreover, with the valuation of a Creating Hope Act pediatric priority review voucher reaching $350 million, we are witnessing a sea of change in biotech and pharmaceutical companies who are now embracing business plans built around pediatric cancer and pediatric rare disease drug development.
As you may recall, in 2012, we were successful in advocating for the passage of the Creating Hope Act. The Creating Hope Act created pediatric priority review vouchers as incentives for drug development for seriously ill kids. Under the Creating Hope Act, sponsors who develop drugs for seriously ill children, including children with cancer, and whose drugs receive FDA approval, receive a voucher. The vouchers are fully tradable. They provide the holder the rights to faster FDA review on any other drug, perhaps a blockbuster heart disease drug, for example.
$350 million for a pediatric cancer voucher
In March 2015, United Therapeutics earned the third voucher under the Creating Hope Act program for Unituxin for neuroblastoma, which was reported by the New England Journal of Medicine to be a major improvement in neuroblastoma treatment. United Therapeutics then turned around and sold the voucher to AbbVie for $350 million.
Academic pediatric researchers report that they are seeing dramatic increases in the level of interest companies are demonstrating on pediatric cancer drug development. Pharmaceutical executives who run pediatric programs are happy to report that their companies are now more invested in their efforts to develop more pediatric cancer drugs.
Kids v Cancer has been asked by the U.S. Government Accounting Office to provide evidence of the impact of the Creating Hope Act as part of a report required under the law. If you would like to work with us on this effort, please let us know.
We are asking for the reauthorization of The Creating Hope Act
The Creating Hope Act has demonstrated its potential to help change the landscape of pediatric cancer research. The Creating Hope Act was passed as a pilot program and, under the terms of the law, will sunset a year after the third voucher is issued, March 14, 2015. We are grateful for the leadership of Representatives McCaul, Butterfield and Van Holen, and Senators Casey and Isakson who introduced the Advancing Hope Act, H.R. 1537, and S. 1878 respectively, to reauthorize the Creating Hope Act.
We are pleased to report the Act was incorporated into the 21st Century Cures bill, H.R. 6, which passed the House, 344 to 77. We are waiting for the Senate to develop and pass its version of the 21st Century Cures Act as soon as possible to avoid the sunset of the voucher program.
We are pressing for the Kids Innovative Drugs Initiative to update the Pediatric Research Equity Act (PREA)
But incentivizing the development of drugs specifically designed for pediatric cancer is just the first step. There are 900 drugs in the adult cancer pipeline, many of which could benefit children but virtually none of them are being tested on kids or being researched for pediatric cancer indications.
To address this problem, Kids v Cancer developed the Kids Innovative Drugs Initiative (KIDS Initiative) to spur pediatric studies of drugs developed for adults.
The KIDS Initiative would update the Pediatric Research Equity Act (PREA), which was designed to require companies to test drugs being developed for adults on kids, but which has been interpreted in a way that does not cover pediatric cancer.
PREA requires companies developing drugs for adults to also develop those drugs for children if there are children who suffer from the same “indication” as the adults. However, “indication” pursuant to PREA has been interpreted as doing so only if the children suffer from the same disease. The problem, of course, is that children don’t tend to get breast cancer or prostate cancer, but the cancers they do get share many of the same molecular markers as adult cancers. By updating PREA to require companies to share drugs that act upon the same markers as those active in pediatric cancers, we expect to open up vast new opportunities for pediatric cancer drug development.
Over 150 pediatric advocacy groups support the KIDS Initiative, as have some of the leading cancer hospitals and research institutions. We continue to press for its passage into law.
We have established the Pediatric Cancer Drug Development Working Group
To further develop our legislative and regulatory proposals, we have established the Pediatric Research Drug Development Working Group. If you would like to be a member of the Working Group and participate in its discussions, please let us know.
We are helping children access unapproved, promising drugs through compassionate use access
Children with cancer face a catch 22: there are few pediatric cancer clinical trials with investigational drugs and at the same time it’s difficult to get a drug through a compassionate use application. We are continuing to address this challenge.
Earlier this year, Kids v Cancer was invited by Friends of Cancer Research to co-host a working group on compassionate use. Kids v Cancer staff also facilitated compassionate use applications for two children. And in addition, Elena Gerasimov wrote an excellent case study documenting what the pediatric cancer advocacy community did in the case of Josh Hardy, a young boy who successfully filed a compassionate use application. We look forward to developing opportunities for children with cancer and their physicians to access compassionate use of drugs more easily.
We are raising awareness
In our continued efforts to raise awareness of the issues affecting pediatric cancer research, Nancy Goodman and Katie Miller have been speaking widely, including at the CDDF–ENCCA– ITCC-SIOPE 3rd Paediatric Conference: Prioritization in Paediatric Oncology Drug Development (Vienna, Austria); the Boston Children’s Hospital Global Pediatric Innovation Summit (Boston MA); the 2015 Neuroblastoma Medulloblastoma Translational Research Consortium (NMTRC) Symposium, (Scottsdale Arizona); the Peter Drucker Institute (Clarmont, CA); Memorial Sloan Kettering Cancer Center, Art of Medicine (New York, NY); NYU School of Medicine and the New York Academy of Sciences Colloquium Pre-Approval Access: Can Compassion, Business, and Medicine Coexist? (New York, NY); National Organization for Rare Diseases, Friends of Cancer Research and Kids v Cancer Forum: Expanded Access in the Era of Breakthrough Therapy (Washington, DC); U.S. Food and Drug Administration Stakeholder Input on BPCA and PREA March 25, 2015 (Washington, DC), National Organization for Rare Diseases: Rare Diseases and Orphan Products Breakthrough Summit (Vienna, VA); Fastercures Partnering for Cures (New York, NY).
In addition, Kids v Cancer’s work on the Creating Hope Act pediatric priority review vouchers has been widely covered in major newspapers and business publications in the United States. Nancy has written letters to the editors for the Washington Post and New York Times and an op-ed for the Cancer Letter.
We are going to Europe
Kids v Cancer continues to be engaged with advocacy, regulatory and pharmaceutical executive counterparts in Europe as they develop their own agenda for promoting pediatric cancer drug development, engaging in conferences in Vienna and London over the course of the year.
We have established the Kids v Cancer Youth Board
Kids v Cancer has now formally instituted a Youth Board of dedicated and focused young men and women who have been volunteering for Kids v Cancer for several years.
The Youth Board Members run bake sales at the JCC of Manhattan; organize art and music projects for the children at NYU’s pediatric cancer clinic where Jacob was treated; participate in the Childhood Cancer Caucus lobby days on Capitol Hill; and serve as interns with Kids v Cancer at its Washington, D.C. headquarters.
We are building Kids v Cancer
The Kids v Cancer team – Katie Miller, Jenn Flynn, Georgina Rodriguez, Nancy Goodman and a large host of dedicated pro bono advisors and lawyers — welcomes our newest team member, Leika Uzcategui, as our Business Manager. Leika manages all of us, keeps our books, oversees special projects and events, as well as H.R. We’re thrilled to have Leika and her family as part of the Kids v Cancer team.
FDA and Peter Drucker Institute Awards
This year, Nancy received a Group Recognition Award from the US Food and Drug Administration as a member of the 2014 Patient Network Meeting: Pediatric Drug Development. She continues to serve on the NCI Board of Scientific Counselors and most recently the American Association of Cancer Research’s (AACR) Pediatric Cancer Working Group Steering Committee. In October, Kids v Cancer was honored to receive the 2015 Peter F. Drucker Award for Nonprofit Innovation as the most innovative non-governmental organization in the United States. In conferring this award, the Foundation noted the leverage Kids v Cancer achieves in generating hundreds of millions of dollars of pediatric cancer drug development on a shoestring budget.
Speaking of shoestring budget, we are deeply grateful for the support we have received from so many. We have come a long way, but we there is much to do. Thank you for all your support. We very much appreciate it.
Together we are Changing the Landscape of Pediatric Cancer.
Nancy Goodman and the Kids v Cancer team