Session (CTI)

Issue: Despite adoption of the ICH E9(R1) addendum and the estimand framework by the FDA and other regulatory bodies, its role in shaping patient-centered objectives in cancer research remains unclear throughout the continuous evidence lifecycle from clinical development through post-approval research. Overview: This session will reinforce how the estimand framework can facilitate generation of meaningful patient-centered evidence and will identify current opportunities and challenges for cancer research. Dr. Park will provide an overview of the estimand framework and discuss the role of the estimand framework for both clinical trial and real-world evidence (RWE) studies (8-minutes). Dr. Sharaz will discuss how regulators and health technology assessment (HTA) bodies view patient-reported outcomes (PROs) with a focus on the role of data collection post-treatment and post-progression to supplement traditional survival and progression endpoints (10-minutes). Dr. Skaltsa will discuss the methodological challenges as well as the current advancements with regard to analyzing PROs collected in oncology trials in line with the estimand framework, during the treatment period and beyond (10-minutes). Dr. Roydhouse will discuss considerations for comparing patient-reported tolerability of different therapies, and how comparative tolerability endpoints can supplement other PRO and non-PRO endpoints in oncology (10-minutes). The session will reflect on future directions of methodological research based on the estimand framework for patient-centered research including the communication of estimands to patients, regulators, HTA bodies, and other diverse stakeholders (5-minutes). The session will conclude with an interactive discussion with audience participation to explore current challenges and opportunities ahead for patient-centered research and the estimand framework (17-minutes).