010: An Overview of Statistical Approaches and Operational Challenges Related to Non-Concurrent Controls in Platform Trials

Traditional, two arm randomized controlled trials are the gold standard for evaluating new therapies, but as technology has advanced, the need for flexible and efficient trial designs has increased. Master protocols, specifically platform designs, have been proposed as an option. Platform trials test multiple interventions against a common control arm, allowing for arms to enter and exit the study, and often this increases efficiency in evaluating efficacy for experimental arms. However, increased flexibility comes with challenges from both statistical and operational perspectives. Many challenges are related to proper utilization of control data in assessing efficacy. Should an arm be compared only to controls enrolled during the period in which it was active (concurrent controls), or can we leverage information from controls enrolled during other time periods (non-concurrent controls)? Several analytic frameworks have been proposed to address this question, and these decisions have important implications for operational aspects of the platform, such as data sharing. This presentation will discuss considerations for the use of non-concurrent control data and related challenges.

platform trials

non-concurrent controls

master protocols

adaptive design 

Study Design and Data Management

Conference on Statistical Practice (CSP) 2023