CCCA Presents at NIH-FDA Joint Leadership Council Meeting

JUNE 2, 2010

FOR IMMEDIATE RELEASE

Silver Spring, MD -- Lori Salley, Executive Director of the Children's Cause for Cancer Advocacy (CCCA), presented on the challenges of developing new therapies for children with cancer at today's public meeting of the newly formed Joint Leadership Council, a partnership of the National Institutes of Health (NIH) and Food and Drug Administration (FDA).

The NIH-FDA Joint Leadership Council aims to use their agency collaboration to better ensure that the latest science and biomedical research is integrated into the regulatory review process and the approval of new therapies.

CCCA's presentation, titled "Challenges in Pediatric Cancer Research and Care," emphasized that although childhood cancer deaths have declined dramatically over the past 30 years due to intensifying chemotherapy regimens among a set of cytotoxic drugs developed for adults, progress has slowed in the last decade. Survival rates for a number of cancers remain low, and chemotherapy treatments have reached their maximum intensity for use in children. Further, two-thirds of the nation's 328,000 childhood cancer survivors suffer from at least one side effect as a result of their treatment and their long-term mortality is affected as a result.

The CCCA presentation, created by Susan Weiner, PhD, CCCA's President and Founder, laid out the barriers to bringing new molecular, personalized therapies to children, which include the many varied and rare types and subtypes of childhood cancers that are different than adult cancers, the length of time it takes to complete Phase III trials, and the federal research protections that constrain the procedures that can be done to children. Related to these unique challenges, the biotechnology and pharmaceutical industry has limited engagement in the development of new pediatric oncology drugs, and regulatory incentives are focused on testing of adult drugs in children, not the development of pediatric-specific therapies.

CCCA made the following suggestions for ensuring that children with cancer have access to safe, effective, modern therapies:

• Facilitate public/private partnerships to discover and develop new drugs to be brought to NCI cooperative drugs for evaluation;
• Create an interagency task force charged with publicizing NIH resources available for the discovery and development of new pediatric therapies;
• Reauthorize and modify the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act to reflect contemporary science, including a pediatric incentive for biologics;
• Promote research on minimally invasive biomarkers to get safer, more effective drugs to kids;
• Increase research on novel statistical strategies to both reduce children's exposure to ineffective treatments and reduce sample sizes necessary for reliable conclusions;
• Ensure that FDA personnel assigned to pediatric oncology matters understand the unique issues and complex research constraints with this population; and
• Create a mechanism that fosters collaboration among FDA, NCI, and pediatric oncology experts with the authority to advise researchers on strategies to bring modern treatments to children with cancer.

CCCA is a leading national advocacy organization that works to achieve access to less toxic and more effective therapies, expand resources for treatment, and address the needs and challenges of childhood cancer survivors and their families. CCCA works to ensure that these needs and perspectives are integrated into the highest deliberations of cancer policy.