The Coalition for Pediatric Medical Research: Recommended Pediatric Priorities for the Cancer Moonshot Initiative

March 03, 2016


David A. Williams, MD, president of Dana-Farber/Boston Children's Cancer and Blood Disorders Center, and other members of the Coalition for Pediatric Medical Research met with the staff of Vice President Joseph Biden, leader of the federal government's cancer moonshot. The coalition members urged Biden's staff to ensure that pediatric cancer is represented on the moonshot and presented the following recommendations:

Pediatric Cancer: Overview

  • Cancer is the leading cause of death due to disease among children in the United States.
  • The majority of childhood cancer survivors experience harmful, life-long complications from their treatments.
  • Children with cancer are being left behind in the revolution that is occurring in precision medicine.


  • The biology and types of childhood cancers are different than those in adults and require distinct investigation and therapies.
  • Childhood cancers are a compilation of more than 150 types of cancer, yet pediatric cancers as a whole receive a small fraction of total NCI/NIH funding.
  • While industry is the primary funder of R&D in adult cancers, it is largely absent as a funder of childhood cancers, making childhood cancer almost entirely dependent upon federal and philanthropic resources.

Pediatric Cancer Opportunities


  • Significantly increase NIH/NCI support for pediatric cancer research through a broad range of strategies and tactics:
    • Dedicated RFAs (e.g., novel approaches to currently "undruggable" targets such as fusion oncoproteins; immunotherapy; biology of resistance; molecular predictors of risk and outcomes, including prevention strategies)
    • Novel funding mechanisms (e.g., National Pediatric Research Network law)
    • Incentives to extend application of precision "omic" and other leading edge technologies to a broad array of high-risk and rare childhood cancers.
    • Leverage ongoing approaches to big data sharing for pediatric cancer research.

Therapy Development

  • Extend current and develop novel industry incentives to accelerate development of pediatric cancer therapies:
    • Fast Track and related accelerated pathways
    • Creating Hope Act and other incentive models
  • Amend PREA (Pediatric Research Equity Act) so that determinations are based on a drug's mechanism of action rather than indication.
  • Institute a public-private partnership to accelerate discovery and development of pediatric cancer therapies (See IOM Report Making Better Drugs for Children with Cancer).
  • Consider incentives to provide support for necessary infrastructure (e.g., compound libraries, biorepositories, animal models).
  • Increase support for multi-institution networks, including the Children's Oncology Group and the Clinical and Translational Science Awards (CTSA) to advance clinical and translational research.
  • Establish a fund to support pediatric cancer drug development for agents where there is significant promise in children but not in adults.

Training & Support

  • Increase funding for pediatric oncology T32 and new training awards, and create set-asides for pediatric cancer physician-scientists.
  • Strengthen the pediatric component of the CTSA program to accelerate pediatric clinical and translational research broadly, including in pediatric cancers.

Media Inquiries

Ellen Berlin

Kristen Dattoli

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