Making our Voices Heard at NCI

On Friday February 21, the Children's Cause for Cancer Advocacy (CCCA) was proud to participate in a full day meeting at the National Cancer Institute (NCI) with the Director's Consumer Liaison Group (DCLG).  The entire day was devoted to barriers to drug development in pediatric cancer research -- a first for this advisory board.   dclg-v2

The day began with  an overview of the state of pediatric cancer research, featuring top experts Dr. John Maris of the Children's Hospital of Philadelphia,  Dr. Malcolm Smith and Dr. Lee Helman both of NCI.  All three speakers addressed both the significant progress we have made in certain childhood cancers and the fact that we have made virtually no progress in others.  When asked to specify the most promising advancements, we heard about unprecedented discoveries in recent years, particularly in immunotherapies.

Unfortunately though, there are still many challenges.

Among the challenges identified were:

  • The need for greater access to biospecimens and the ethical considerations in conducting biopsies;
  • Too often childhood cancer clinical research is driven by regulatory requirements and are based on adult cancer priorities rather than scientific needs of childhood cancer research;
  • Long-term effects on childhood cancer survivors due to toxic therapies;
  • Losing the next generation of physician-scientists due to decreased federal funding

The conversation continued during lunch, with a lengthy discussion about the shortfalls of the current regulatory framework in both the US and Europe as well as industry incentives. Featured speakers included Dr. Peter Adamson of Children's Hospital of Philadelphia and Dr. Gregory Reaman of the FDA's Office of Hematology and Oncology Products. The Children's Cause recently commented on these issues to the FDA and was glad to see general agreement from others on these shared principles regarding better coordination between the US and Europe and needed changes to the Pediatric Research Equity Act which would allow FDA to require pediatric studies of oncology drugs based on particular pathways rather than the site of cancer in adults.

Later in the day, representatives from various advocacy organizations had the opportunity to present on the work they are doing.  Maureen Lilly, Executive Director of CCCA, discussed our particular work to promote changes to FDA regulations (as noted above) and the Caroline Pryce Walker Conquer Childhood Cancer Reauthorization Act, which would, among other things, improve biospecimen collection.  We were pleased, though not surprised, to see that our work is properly focused on filling in the gaps identified by the nation's top childhood cancer researchers.

Not only did we learn a lot from the presenters, but we noted how much the DCLG members learned both in terms of content and recognition of needing greater involvement of the childhood cancer community. They left the meeting committed to adding a pediatric cancer community representative to the DCLG and continuing the conversation not only with the advocacy community, but with industry representatives as well.  Materials from the meeting will be made available in the near future at:

CCCA is proud to remain a part of these ongoing discussions and ensuring that childhood cancer patients, survivors and families are represented at the highest level of policymaking conversations.