DRAFT November 15, 2014
LEGISLATIVE OPTIONS FOR SUPPORTING PEDIATRIC CANCER RESEARCH
There is a misperception that pediatric cancer has been largely solved and cured.
Nothing could be farther from the truth:
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Fact: Pediatric cancer is the #1 disease killer of children.
Fact: Pediatric cancer treatments have devastating toxicities because in order to
achieve survival, children receive higher doses of chemotherapies than adults
while they are more vulnerable to long term harm. Only about one third of
children diagnosed with cancer go on to lead long, healthy lives after treatment.
Fact: Though our society is witnessing a scientific revolution in cancer
treatments, this revolution has not reached children with cancer.
Fact: Of the approximately 900 drugs in the cancer pipeline, only a handful are in
clinical trials for children.
The pediatric cancer drug development process is broken at every point – from lack of
funding for basic research, to lack of access to drugs developed for adult cancers, to lack
of drugs developed expressly for pediatric cancers, to lack of care for pediatric cancer
survivors.
Addressing these shortfalls would give kids with cancer a chance to live and live
healthier lives. Together, these recommendations create greater transparency, more
available information, better access to drugs, more incentives for drug development
expressly for kids, stronger incentives for pediatric formulations and better patient care.
It is critical that the entire process be addressed.
ACCESS TO DRUGS DEVELOPED FOR ADULTS THAT COULD BENEFIT CHILDREN
Three Month Supplemental Exclusivity
The pipeline of approximately 900 drugs for adult cancer creates unique opportunities
for children with cancers, especially children with terminal cancers. However, too often,
terminal children are denied promising drugs developed for adult cancers until those
drugs are approved. Moreover, when development of unapproved adult drugs is
discontinued, there is often no opportunity to continue development on the pediatric
indication.
We propose several fixes to BPCA, with a three month supplemental exclusivity to
incentivize companies to comply with these fixes.
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For drugs for life threatening diseases for which there is a serious, unmet medical need,
BPCA should be tweaked so as to incentivize companies to undertake these obligations
by offering a three month supplemental exclusivity incentive.
1. Opt in:
a. By 60 days after the end of phase 1 adult meeting, the company will
submit a written request
b. The written request will include a provision that should the company
discontinue adult drug development, that the company will continue
pediatric drug development or, if requested by the NIH, transfer pediatric
IP and clinical supply to the NIH and agree to maintain supply.
2. Deadlines: (no deferrals for any reason)
a. By the end of phase 2 meeting on the adult side, the company will submit
phase 1 pediatric data
b. By end of phase 3 meeting for adult indication, the company will
complete phase 2 data on pediatric indication and complete pediatric
formulation
i. If the adult indication gets break through designation, then the
pediatric drug development only needs to open a phase 2 trial.
3. Success of trials: The drug must be approved for the pediatric indication to earn
supplemental exclusivity.
Bring PREA Up To Date With Scientific Innovations
To address these challenges, we urge Congress to bring the Pediatric Research Equity
(“PREA”) up to date with the new scientific strategies for drug development.
PREA was intended to benefit children by ensuring that drugs that would be used by
both adults and children would be approved for both adults and children. At the time
PREA was passed into law, drugs were developed and approved by indication (e.g., nonsmall cell lung cancer). However, now cancer drugs and other drugs are approved not
only by indication, but by specific targets and identified mechanisms of action (e.g., nonsmall cell lunch cancer and ALK inhibitor).
To bring PREA up to date with innovations in drug development, when there are
children who would be a part of a drug’s patient population because of targets or
mechanisms of action of their diseases, the drugs should be approved both for adults
and children.
PREA should be updated for life threatening diseases for which there is a serious, unmet
medical need so that :
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1. sponsors submit their pediatric study plan 60 days after the end of phase 1
meeting and
2. if there is a mechanism of action on the draft label of the end of phase 1
meeting, and if that mechanism of action is present in a pediatric population,
then PREA applies to that pediatric population.
The current exemption from PREA obligations for drugs that have orphan designations
has implications that have grown over time for drug development for pediatric cancer
and other pediatric life threatening indications.
Ensure that prioritization goals of the academic community are understood by
the FDA as FDA medical officers draft written requests
At least once a year, the FDA should hold an advisory committee meeting at which
outside experts will inform the FDA of what their research priorities are for each rare
pediatric disease for which the FDA will be authorizing a written request.
Written Requests
To create enforceability for the BPCA reforms, the written requests should be made
public to the greatest extent possible.
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