Despite groundbreaking advances in pediatric cancer research, many promising therapies never reach the children who need them most.
Catherine Bollard, MD, is senior vice president and chief research officer at Washington, D.C.-based Children’s National Hospital. She is also director of the hospital’s Center for Cancer and Immunology Research.
As a member of the nonprofit think tank Access4Kids, her work focuses on brainstorming new pathways to bring pediatric drug therapies, including cell and gene therapies for the treatment of cancer, into the market and out of the pediatric cancer drug “valley of death.”
She spoke to Becker’s about her vision to break pediatric drug therapies out of the traditional drug development model.
Editor’s note: Responses have been lightly edited for clarity and length.
Question: Can you briefly describe pediatric medicine “valley of death” and what it means for hospital and health system leaders?
Dr. Catherine Bollard: The “valley of death” is a term frequently referenced in relation to complex biologic therapies [such as] cell and gene therapies that don’t fit the traditional pharma model. It refers to the fundamental gap that exists between promising preclinical research and the successful clinical translation to phase 1 and maybe even phase 2 clinical trials and commercialization and regulatory approval. In the pediatric setting, these drugs fail to become commercialized due to a lack of funding to perform trials given the rare patient population, regulatory hurdles and manufacturing challenges.
This creates a position where drugs that are developed specifically for pediatric patients and cure children with cancer, immune mediated and genetic diseases still end up failing to commercialize. Therefore children are denied access to potentially lifesaving therapies.
Q: The proposed Pediatric Advanced Medicines Biotech model represents a new approach to advancing pediatric cell and gene therapies. What role should hospitals and health systems play in driving the development, funding and commercialization of these treatments to avoid reliance on traditional pharmaceutical industry models?
CB: The not-for-profit Pediatric Advanced Medicines Biotech entity could be supported by philanthropists, venture philanthropists and hospitals. With hospital and academic support, we can manufacture the novel cell and gene therapy products in academic good manufacturing practice facilities, and partner with regulators to help “ferry” these products across the “valley of death” to commercialization outside the traditional biopharmaceutical model.
This PAMB would lead late-stage development and ultimately, commercialization. Critical to this success would be access to academic GMP facilities to manufacture the cell and gene therapies, which would require support from the pediatric hospitals and health systems.
