Optimizing Oncology Trials for Regulatory Success: PRO Measurement Strategy
Only 8.3% of oncology drugs approved by the FDA between 2010 and 2020 included patient-reported outcome (PRO) data in their labeling - a missed opportunity to capture the patient experience beyond traditional tumor response and survival endpoints. The FDA's guidance on patient-reported outcomes in cancer trials fundamentally changes how sponsors should design and implement COA measurement strategies, yet uptake remains limited due to implementation uncertainties and complexity concerns.
Dr. Todd Everhart, Clinical Vice President of Internal Medicine at Signant Health, provides strategic guidance for navigating this evolving regulatory landscape. Board-certified in Internal Medicine and a Fellow of the American College of Physicians with over 25 years of clinical experience, Dr. Everhart translates FDA recommendations into practical measurement strategies that generate submission-ready evidence while optimizing the patient and site experience.
Essential implementation strategies:
- Designing PRO measurement approaches aligned with FDA's five core domains: disease symptoms, symptomatic adverse events, side effect impact, physical function, and role function
- Implementing modular assessment strategies with domain-specific frequency requirements for optimal data quality
- Leveraging decentralized trial methods to enable at-home PRO completion that reduces clinic burden
- Balancing comprehensive data collection with participant retention and protocol adherence
Critical viewing for oncology clinical operations teams, regulatory affairs professionals, and medical directors working to enhance labeling claims and competitive positioning.
