DOI: 10.1101/19010827Nov 2, 2019Paper

Predictive approaches to heterogeneous treatment effects: a systematic review

MedRxiv : the Preprint Server for Health Sciences
A. RekkasDavid van Klaveren


Background: Recent evidence suggests that there is often substantial variation in the benefits and harms across a trial population. We aimed to identify regression modeling approaches that assess heterogeneity of treatment effect within a randomized clinical trial. Methods: We performed a literature review using a broad search strategy, complemented by suggestions of a technical expert panel. Results: The approaches are classified into 3 categories: 1) Risk-based methods (11 papers) use only prognostic factors to define patient subgroups, relying on the mathematical dependency of the absolute risk difference on baseline risk; 2) Treatment effect modeling methods (9 papers) use both prognostic factors and treatment effect modifiers to explore characteristics that interact with the effects of therapy on a relative scale. These methods couple data-driven subgroup identification with approaches to prevent overfitting, such as penalization or use of separate data sets for subgroup identification and effect estimation. 3) Optimal treatment regime methods (12 papers) focus primarily on treatment effect modifiers to classify the trial population into those who benefit from treatment and those who do not. Finally, we also identified pap...Continue Reading

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