COMMENTS BEFORE THE 21ST CENTURY CURES ROUNDTABLE OF CONGRESSMAN SHIMKUS AND CONGRESSMAN DAVIS
Executive Director, Kids v Cancer
Congressman Shimkus, Congressman Davis, thank you for inviting me here today. I come as Founder and Executive Director of Kids v Cancer and, more importantly, as a mother who lost her 10 year old son, Jacob, to a pediatric brain cancer.
My message to you today is a simple one. The exciting innovations you have been addressing in past Congressional round tables – Accelerated Approval and the introduction of biomarkers to facilitate trial completion, precision medicine to create tailored treatment plans for individuals, the integration of companion diagnostics – these innovations have occurred with little benefit to children with cancer and other life threatening diseases.
When my son Jacob was diagnosed with cancer at age eight, he received drugs that were 20 to 40 years old. His physicians knew they were unlikely to be effective, yet they gave these drugs to Jacob because they were the only treatments available.
Why is that? If we have the most innovative, resource rich pharmaceutical industry in the world, and if we are in the middle of exciting innovations to find new cures, why aren’t the advances benefiting children?
CREATING HOPE ACT
After Jacob died, I formed Kids v Cancer to address the challenge of drug development for children with cancer and other life threatening illnesses. Our first project, with the support and leadership of the entire pediatric cancer community, was to draft the Creating Hope Act to create incentives for drug companies to develop drugs for kids with cancer and other life threatening illnesses. The form of the incentives is a transferable priority review voucher. A company that develops a pediatric rare disease drug receives a voucher. The voucher is transferable and may be exercised to receive faster FDA review on another drug, perhaps a large market drug.
The Creating Hope Act was enacted into law in 2012 as 21 USC 360ff. And I’m proud to announce that last month, the first Creating Hope Act pediatric priority review voucher sold for $67.5M. Our program works.
Who won? Biomarin won because they sold the voucher and won the proceeds. Sanofi and Regeneron won because they were able to purchase the voucher and will use it for faster FDA review of a cholesterol drug. Kids with Morquio syndrome, a terrible pediatric disease, won when they got a new drug. And tax payers win because there is no cost to them.
We look forward to working with the FDA to implement the Creating Hope Act and to ensure that the Creating Hope Act covers pediatric cancers. We look forward to working with you, Congressman Shimkus and Congressman Davis, to permanently reauthorize the Creating Hope Act at the appropriate time.
ACCESS TO DRUGS
The Creating Hope Act is an important step in supporting pediatric cancer and pediatric rare disease drug development.
There are still, however, additional roadblocks to be addressed. We need more incentives to ensure that children have a chance of benefiting when drugs are primarily developed for adults. Although Best Pharmaceuticals for Children Act and Pediatric Research Equity Act were designed to respond this challenge, they are not effective in the area of pediatric cancer research.
A couple data points.
- There are only a handful of pediatric cancer trials anywhere that include an unapproved drug. More than 95% of all pediatric cancer trials use only approved drugs.
- Pediatric oncologists complain that it can take years to receive a supply of an unapproved drug for clinical or even preclinical study in children. And often they do not receive the drug.
In the area of oncology drug development, oncology drugs are now developed by mechanism of action rather than by organ – breast cancer or prostate cancer, for example.
One would think this would be of benefit to children because children’s cancers sometimes share targets with adult cancers. However, kids with terminal cancers do not have access to these promising unapproved targeted therapies, usually until if and when they are approved for an adult cancers and can be used off label. If the drug does not work for an adult cancer, it is not tested in a pediatric cancer. That is a loss.
There are numerous ways we can address these roadblocks and I look forward to working with you on them, Congressman Shimkus and Congressman Davis.
Losing Jacob to cancer did not occur once, it happens every day. It’s September and my surviving children, Ben and Sarah, are back at school. I wonder – would Jacob still be playing keyboard and singing in his 90’s rock band? Would he be sporting a buzz cut or long, shaggy hair? He had beautiful red hair. Ben and I hang Jacob’s old band posters in Ben’s bedroom. Ben likes them because he says he can think of Jacob when he first wakes up.
We Americans should be able to do better. Through legislative and regulatory reform, we can give children and teens with cancer and other life threatening illnesses a better chance.
Thank you for giving me the opportunity to speak. I look forward to working with you.