Comment on “Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment”

Abstract

This is a commentary paper for "Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment" by Dr. Levenson. Levenson et al and its companion papers provide clear and detailed explanations of the biostatistical considerations when using RWD/RWE in regulatory submissions. We will discuss some points of concern we particularly focused on, and will also introduce the research conducted by our working group in Japan. The working group addressing biostatistics aspects puts special emphasis on 1) the transparency of conducting clinical trials utilizing RWD and 2) possible biases relative to conventional clinical trial settings.

Strategies to Address Current Challenges in Real-World Evidence Generation in Japan

“…In the design phase, this information can be acquired from data dictionaries and pre-analysis investigation of data samples to assess data completeness. In the context of evaluating treatment effectiveness, the following process has been proposed: (i) choose the estimand by specifying the hypothesis, (ii) opt for a trial emulation framework and define the variables for meeting the “exchangeability” assumption, and (iii) specify the analytical method at the design stage [ 36 ]. In this regard, examples of trial emulation using observational data were reported in the literature [ 37 , 38 ].…”