Generating New Treatments for Kids – It’s A Team Effort

The effort to change the laws that affect pediatric research may be the most challenging and important for our community and will require continued commitment, vision, collaboration and most importantly, unity.
— Children's Cause for Cancer Advocacy

For the past 17 years, the Children’s Cause for Cancer Advocacy has served as the authoritative voice of the childhood cancer community in public policy debates in Washington, framing discussions on research funding, treatment initiatives, access to care and treatment and survivorship.

The Children’s Cause brought that focus and leadership early on to help form the Alliance for Childhood Cancer, strengthening collaboration and cooperation among the many pediatric cancer groups to achieve significant policy milestones for kids and their families.  Now with 35 organizations in its ranks, the Alliance has increased the visibility of childhood cancer issues in the Washington legislative and policy arena.

Over the years, the Children’s Cause – with unity across the community – has worked to successfully pass the Caroline Pryce Walker Conquer Childhood Cancer Act, focused attention on childhood cancer research at the National Cancer Institute, helped to accelerate survivorship programs at the NCI, pushed to address chronic shortages of drugs used to tread childhood cancer and helped enact new laws intended to evaluate new therapies for children.

The drug development laws, the Best Pharmaceutical for Children Act (BPCA) and the Pediatric Research Equity Act (PREA), continue to be priorities for the Alliance.  The two laws act in tandem as both carrot and stick to encourage new drug development for childhood diseases. BPCA provides an incentive of six months of exclusivity – or patent extension – for drugs that are approved for use in children. 

PREA - the “stick” portion of the approach - requires pharmaceutical companies who are developing a drug for adult indications to also test the drug in children.  Two exceptions to this requirement constrain their impact for children with cancer.  First, pediatric studies of a drug can only be required in the same disease (“indication”) for which it is being studied in adults.  Since children do not develop lung, breast or prostate cancer, for example, drugs under development for adult cancers do not have to be tested in kids.  A second exemption from the PREA requirement applies to any drug being developed for a “rare” disease. Rare diseases are defined in US law as those that are diagnosed in fewer than 200,000 people per year in the US.   Unfortunately, most cancers meet the definition of a rare disease.   These exceptions have resulted in PREA having virtually no effect in stimulating more treatments for childhood cancer.

Families and childhood cancer advocates anxious for new treatments have been frustrated with the inadequacy of PREA and have pressed for remedies that stimulate more childhood cancer development from industry.   With a legislative opportunity coming up in the next year through a larger must-pass bill, the Children’s Cause co-chaired a workgroup of the Alliance and Coalition Against Childhood Cancer (CAC2) that developed a series of policy recommendations to encourage more childhood cancer drug development. These recommendations include changes to the PREA statute.

Alliance membership has rallied as in the past to support this effort and unified the community in a common objective. As strong as a unified childhood cancer community might be, moving Congress on this matter will require collaboration and agreement among a broad range of stakeholders, including a variety of patient groups (the laws cover more than just cancer), professional medical associations, research institutions, federal agencies and - most importantly - the biopharmaceutical industry.

In anticipation of a new Congress and legislation affecting the FDA, the Children’s Cause has begun a broad consensus-building process to solicit additional recommendations and suggestions by stakeholders that will create a bold, effective framework for the creation of new drugs for kids with cancer.

The Children’s Cause, along with the Alliance, has been committed to successfully spearheading policy initiatives that have advanced an agenda for childhood cancer that advances therapies and improves care.  The effort to change the laws that affect pediatric research, however, may be the most challenging and important for our community and will require continued commitment, vision, collaboration and most importantly, unity. 

Together, we look forward to improving the lives of children with cancer.