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Activity Number: 575 - Trial Design with Subpopulation Heterogeneity: What Is the Role of Real-World Evidence?
Type: Topic Contributed
Date/Time: Thursday, August 6, 2020 : 3:00 PM to 4:50 PM
Sponsor: Biopharmaceutical Section
Abstract #312778
Title: Evaluating Off-Label Use of Targeted Cancer Treatments: Generating RWE for Precision Medicine Using CancerLinQ Discovery (CLQD) Data
Author(s): Elizabeth Garrett-Mayer* and Melinda Kaltenbaugh
Companies: American Society of Clinical Oncology and American Society of Clinical Oncology
Keywords: real world data; real world evidence; cancer; missing data

Many cancer clinical trials exclude patient (pt) groups for whom treatments (trts) are prescribed after regulatory approval (e.g., pts older than 65 years old). Generalizability of trial results to pt populations that are not represented in trials assumes that trts have similar efficacy and adverse event profiles, but reasons for exclusion of pts from trials are often due to concerns over decreased efficacy or increased chance of adverse events. When trts are given to cancer pts who are unlike pts in trial populations, there are additional uncertainties regarding safety and efficacy. Real world data (RWD) from patients who have been treated with anti-cancer trts can be used to assess efficacy and safety profiles for pts treated outside of clinical trials. Analysis of RWD allows exploration of pt subgroups and pt groups poorly represented in trials but who may receive trts after drug approval. However, RWD differs substantially from clinical trial or traditional observational study data. Some of the challenges include missing data and inconsistency of collection of key endpoints of interest. Overcoming and addressing these challenges with an example in CLQD will be discussed.

Authors who are presenting talks have a * after their name.

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