Convening Experts in Oncology to Address Children's Health (COACH)

Identifying pediatric oncology targets to be accelerated through preclinical testing for therapeutic development

The Problem
Cancer is the leading cause of death by disease in children under 15, yet study of cancer drugs in children has lagged due to clinical trial challenges and lack of guidance on data needed to justify the suitability of drugs in children.
The Solution
This project will convene a multi-stakeholder group on a quarterly basis for collaboration meetings, reviewing aggregated cancer research data to provide evidence-based guidance and help prioritize decisions for the preclinical testing.

Overview

While there are over 1,000 drugs in development for adult cancers, only a handful of these drugs are currently being tested in pediatric cancers. One promising avenue for more effective pediatric cancer drug development is to focus on earlier and improved assessment of the potential relevance of drugs developed for adult cancers. To best determine which of these emerging adult therapies should be prioritized for pediatric development, a better understanding of a therapy’s ability to target relevant pathways in a range of pediatric cancer needs to be conducted, which often means intensive preclinical testing.

To aide in the identification of which pathways are relevant to pediatric cancer, the FDA generated the Relevant Molecular Target List, which consists of 200+ distinct molecular targets of interest in pediatric cancers and is part of the FDA Reauthorization Act of 2017 (FDARA). The list is based on a detailed examination of peer-reviewed publications and publicly available databases looking for any data that a target could be playing a role in one or more pediatric cancers. To begin identifying which of these 200+ molecular targets might prove to be the most critical to developing new therapies for specific childhood cancers, a systematic review of existing data around targets is needed. In collaboration with NCI and FDA, the FNIH is convening key stakeholders and knowledge leaders in the pediatric oncology field in quarterly Convening Experts in Oncology to Address Children's Health (COACH) meetings to discuss the highest priority targets that represent the most significant preclinical testing needs in pediatric cancer on the Relevant Molecular Target List. The findings of these discussions will help decide which targets to focus testing efforts. In addition, the meeting outputs from COACH will ultimately inform pediatric treatment development as therapies that address the identified high priority targets emerge within the field.

At each meeting, a panel of experts from all project partners and key industry and clinical knowledge leaders for specific targets, armed with a systemically reviewed package of publicly available data for each clinical and preclinical target from publications, databases, and meeting proceedings, will discuss up to three targets of significant preclinical and clinical need. A summary report of each discussion and recommendations for needed testing for each target will be released to attendees after the meeting. After review by the meeting attendees, the summary will be released to the broader community.

These are invitation-only meetings. If your organization is interested in partnering with us on this effort, please reach out to the FNIH project contacts.

Goals

  • Convene Quarterly Collaboration Meetings in Preclinical Pediatric Oncology to foster discussion and collaboration across the pediatric oncology field.
  • Identify pediatric oncology targets that should be accelerated through preclinical testing.
  • Publish summary reports to share progress on target analysis.
  • Assist in guiding a streamlined way forward to more effective pediatric cancer drug development.