At CAC2 we love to celebrate when our members work together. We offer congratulations to Supporting Organization Day One Biopharmaceuticals and CAC2 member authors and reviewers who contributed to the white paper “Advancing Pediatric Cancer Research and Drug Development through Multi-Stakeholder Collaboration: A Framework Based on the Tovorafenib Journey.”
“Advancing Pediatric Cancer Research and Drug Development through Multi-Stakeholder Collaboration” highlights the specific collaborative efforts needed to address the limited treatment options for childhood cancer. Based on learnings from the research and development journey of tovorafenib, a treatment for pediatric low-grade glioma, the paper highlights features of successful collaborations among nonprofits, academia, and industry. The centerpiece of the white paper is the multi-stakeholder collaboration framework produced as an outcome of a focused workshop. Importantly, the framework can be applied to other pediatric and rare diseases. Access the White Paper here.
The paper draws out the lessons from the example of successful collaboration around tovorafenib. In 2005, a group of parents formed the PLGA Foundation to address the lack of treatments for pediatric low-grade astrocytoma. In collaboration with the Dana-Farber Cancer Institute and other family foundations, they supported research identifying the preclinical tovorafenib activity in pediatric low-grade glioma. This led to a clinical trial sponsored by Day One Biopharmaceuticals, and on April 23, 2024, the US Food and Drug Administration granted accelerated approval for tovorafenib.
Recognizing that the tovorafenib journey could serve as a model for advancing other pediatric cancer research, Day One’s Patient Advocacy team convened a workshop on January 27, 2023, to examine the dynamics of collaboration among NPOs, academia, and industry. Participants identified and defined key elements of this collaboration and the roles and responsibilities of each stakeholder group.
Crucially, the framework that emerged aligned on five essential elements for advancing pediatric cancer research and drug development:
- A core, multi-stakeholder group of individuals dedicated to a specific therapeutic area
- Strategic, long-term sustained funding on a focused research program
- An industry champion committed to pediatric cancer
- A multidisciplinary academic research program, including key stakeholders
- NPO involvement throughout the journey
The principles of collaboration outlined in this framework are relevant to pediatric oncology and apply to non-pediatric cancers and other rare diseases facing similar challenges in advancing research and new therapies. By understanding and applying these principles, stakeholders can continue to drive progress in pediatric cancer research and beyond.
Request to have a printed copy/ies of the paper sent to you at https://www.dayonebio.com/