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Activity Number: 475 - Advancing Innovative RWE Analytics in Drug Development
Type: Topic Contributed
Date/Time: Wednesday, August 10, 2022 : 2:00 PM to 3:50 PM
Sponsor: Biopharmaceutical Section
Abstract #323022
Title: Defining Causal Questions for a Single-Arm Trial with an External Control Arm: An Application of the Target Trial Framework in Oncology
Author(s): Lisa Hampson* and Evgeny Degtyarev
Companies: Novartis Pharma AG, Switzerland and Novartis Pharma AG
Keywords: Causal Inference; Estimand; External Control Arm; Real-World Data; Target Trial Framework

Single-arm trials (SATs) may be used to support regulatory submissions in settings where there is a high unmet medical need and highly promising early efficacy data undermine the equipoise needed for randomization. In this context, patient-level real-world data (RWD) may be used to create an external control arm (ECA) to estimate causal treatment effects. Naive comparisons of the SAT with its ECA will yield biased estimates when groups are imbalanced with regards to (un)measured prognostic factors. Several methods are available to adjust for measured confounding, but the interpretation of such analyses is challenging unless the causal question of interest is clearly defined and the estimator is aligned with the estimand. In this presentation, we use a case-study of a pivotal SAT from late treatment line in oncology to illustrate how a combination of the target trial and the ICH E9(R1) estimand frameworks can be used to define the target estimand and avoid common methodological pitfalls. We find that use of these frameworks facilitates discussions amongst internal and external stakeholders, as well as an early assessment of the adequacy of the available RWD.

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

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