Top row (left to right): Carol Vallett, Eva May, Nevine Zariffa. Bottom row (left to right): Hillary Andrews, Ashita Batavia, Marc Theoret, and Alex Wyatt)
On December 5, Friends of Cancer Research (Friends) hosted a public webinar, “Friends Annual Meeting 2024 Recap: Sharing Next Steps and Opportunities with Advocates,” to provide advocates with an overview of discussions from the 2024 Annual Meeting, outline the path forward, and facilitate a dialogue with the goal of capturing the community’s voice and promoting broader educational access to patient/advocacy groups.
A Common Strategy for Using ctDNA as an Intermediate Endpoint in Prospectively Designed Trials
The webinar delved into the promising role of circulating tumor DNA (ctDNA) as an intermediate endpoint in oncology drug development, positing that it could streamline the regulatory approval process for new therapies. Panelists emphasized the necessity of generating large, standardized data sets in efforts to validate change in ctDNA levels as an intermediate endpoint for overall survival (OS). Carol Vallett, Friends Advisory Advocate, underscored the importance of balancing flexibility in ctDNA data collection timelines with the need for uniform reporting frameworks to enable robust meta-analyses. Carol also discussed how intermediate endpoints can accelerate treatment progress while ensuring results are grounded in data that demonstrates safety and efficacy, which is a critical factor given the urgency for patients.
Interim Overall Survival Evaluations and Implications for Trial Designs and Analysis Plans
The discussion also addressed the U.S. Food and Drug Administration’s (FDA) essential function in the validation of early endpoints, navigating the complexities inherent in assessing immature overall survival (OS) data along with intermediate OS data. Marc Theoret, Oncology Center of Excellence (OCE), U.S. FDA, provided insights on the challenges in navigating the disconnect between short-term endpoints and assessing intermediate OS at the same time. The panelists discussed approaches to strategically interpret early survival analyses, as immature data could lead to incorrect assumptions about safety or efficacy, thereby delaying approvals.
Enhancing Post-Marketing Studies with Pragmatism
The discussion highlighted the importance of pragmatic trial designs to reduce patient and investigator burden, especially in underrepresented populations. The panelists discussed how integrating pragmatic trial elements, such as integration of data from electronic health records (EHR), with clinical trial data can help streamline patient enrollment and data collection to make clinical trials more accessible and representative of real-world populations. Eva May, Friends Advisory Advocate, echoed this sentiment by noting her excitement in her working group participation and the importance of inclusivity in clinical trial design, as pragmatic elements can enhance patient-centricity in research by improving strategies for patient enrollment and retention. May also stressed the need for culturally competent community engagement plans to foster equity in healthcare outcomes and reiterated it is critical to incorporate patient voices in research.
