Making accurate and interpretable treatment decisions for binary outcomes

• Main Authors: Shen, Lingjie, Geleijnse, Gijs, Kaptein, Maurits
• Format: Journal Article
• Language: English
• Published: 20.11.2023
• Subjects: Statistics - Methodology
• DOI: 10.48550/arxiv.2311.11765

Optimal treatment rules can improve health outcomes on average by assigning a treatment associated with the most desirable outcome to each individual. Due to an unknown data generation mechanism, it is appealing to use flexible models to estimate these rules. However, such models often lead to complex and uninterpretable rules. In this article, we introduce an approach aimed at estimating optimal treatment rules that have higher accuracy, higher value, and lower loss from the same simple model family. We use a flexible model to estimate the optimal treatment rules and a simple model to derive interpretable treatment rules. We provide an extensible definition of interpretability and present a method that - given a class of simple models - can be used to select a preferred model. We conduct a simulation study to evaluate the performance of our approach compared to treatment rules obtained by fitting the same simple model directly to observed data. The results show that our approach has lower average loss, higher average outcome, and greater power in identifying individuals who can benefit from the treatment. We apply our approach to derive treatment rules of adjuvant chemotherapy in colon cancer patients using cancer registry data. The results show that our approach has the potential to improve treatment decisions.