Predictive Modeling of Heterogeneous Treatment Effects in RCTs

How often has the 2020 Predictive Approaches to Treatment Heterogeneity (PATH) Statement been cited in published predictive models from randomized clinical trials (RCTs), how well have these reports adhered to PATH Statement recommendations, and how credible are their findings of heterogeneity of treatment effects (HTE)?In this scoping review of 65 reports of predictive models analyzing 162 RCTs, more than one-third (24 reports) identified credible, clinically important HTE. Risk-based modeling was more likely than effect modeling to meet criteria for credibility (87% vs 32%).The findings of this scoping review illustrate the value of predictive modeling of HTE for making RCT results more useful and suggest that external validation in independent patient cohorts can greatly increase credibility, especially for effect models.

Corresponding Author: Joe V. Selby, MD, MPH, Division of Research, Kaiser Permanente Northern California (emeritus), 3101 Benvenue Ave, Berkeley, CA 94705 (jvselby@outlook.com).

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