In late October, we co-hosted a pediatric oncology workshop with PhRMA and BIO, convening a diverse group of key stakeholders and experts in pediatric oncology drug development to discuss scientific and operational challenges of developing new drugs for children with cancer and to explore potential approaches to solving them. For the first time, this workshop brought together key stakeholders in oncology drug development for childhood cancer – patient groups, academia, many of the country’s leading pediatric oncologists, FDA, NIH, and biopharmaceutical companies – who expressed an unprecedented, collective commitment to work together for the benefit of children with cancer.
Full summary: Workshop on Novel Approaches for Pediatric Drug Development for Unmet Medical Need: Addressing Scientific and Operational Challenges in Pediatric Oncology
Workshop presentations included unique perspectives on challenges encountered in efforts to accelerate the development of new drugs for childhood cancer, including five real-life case studies from biopharmaceutical companies. The meeting continued with a working discussion on how the identified challenges might be overcome and how to accelerate the availability of innovative medicines for children with cancer.
Participants highlighted the importance of continued stakeholder discussions of the prioritization of diseases, molecular targets and therapeutic strategies as keys to improving the development of new oncology drugs for childhood cancer. Participants agreed that while several scientific and regulatory strategies could begin to address these challenges, other approaches will be needed, for example, better leveraging of pre-clinical data to inform initial testing in children and novel clinical trial designs, such as biomarker-directed master protocols as in the NCI’s Pediatric MATCH trial.
At the meeting’s conclusion, workshop participants agreed on the following next step:
“The attendees of the Pediatric Oncology Workshop resolved to pursue the creation of a pre-competitive public-private partnership that will explore potential approaches to solving the identified challenges.”
The scope of a pre-competitive public-private partnership will require clear definition as the attendees expressed the need for the new forum to allow for global collaboration. The pre-competitive public-private partnership could address many of the scientific and operational barriers identified in the workshop. In addition, workshop participants agreed to explore how to better leverage existing cooperative infrastructures to expedite the evaluation of drugs for children with cancer.
This workshop marks a new phase in multi-stakeholder engagement and presents an exciting opportunity for collaboration to advance the environment for the development of new and better therapies for treating children with cancer.