Merck & Co., Inc. North Wales, Pennsylvania, United States
Background: With multiple initiatives attempting to replicate clinical trials with real-world data (RWD) beginning to report findings, the collective wisdom gathered from the exercises will help provide insights regarding 1) what research questions that may be best answered by clinical trial data or RWD; 2) factors that may contribute to the incomparability between RWD and clinical trials; and 3) approaches that may help increase the comparability between the two different kinds of data sources in a systematic way.
Objectives: 1. To summarize general patterns observed in various exercises of replicating clinical trials, focusing on: (a) Critical factors that can affect the feasibility of replication; (b) Research questions that may be better answered by RWD WS those by clinical trials 2. To discuss pharmacoepidemiologic and statistical methodologies that were employed in the replication exercises, focusing on: (a) Scenarios that may warrant specific type(s) of methodology; (b) Accounting for the clinical inclusion/exclusion criteria.3. To discuss applications of the learnings from the replication exercises
Description: The symposium will start with a brief introduction of the panelists, background, objectives, and the various exercises of replicating clinical trials with RWD (Solomon Iyasu, 5 minutes), followed by review of findings and lessons learned from the first 8 trials replicated as part of RCT DUPLICATE (Sebastian Schneeweiss, 20 minutes) and from OPERAND (Bill Crown, 20 minutes), focusing on summarizing specific patterns/scenarios in which the feasibility is high WS. low, and if there are corresponding methodologies to help enhance the comparability. In addition, replication results of cancer trial(s) and methodologies designed to enhance the effective sample size of RWD in clinical settings with stringent clinical inclusion/exclusion criteria, along with broad replication considerations and challenges based on real clinical examples, will also be discussed (Thomas Jemielita, 20 minutes). Applications of the learnings in future considerations of using RWE supporting regulatory submission will be considered and discussed (Kenneth Quinto, 20 minutes). The symposium will then end with a brief summary and conclusion (Solomon Iyasu, 5 minutes).