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Dynamic Treatment Regimes.

ABSTRACT: A dynamic treatment regime consists of a sequence of decision rules, one per stage of intervention, that dictate how to individualize treatments to patients based on evolving treatment and covariate history. These regimes are particularly useful for managing chronic disorders, and fit well into the larger paradigm of personalized medicine. They provide one way to operationalize a clinical decision support system. Statistics plays a key role in the construction of evidence-based dynamic treatment regimes - informing best study design as well as efficient estimation and valid inference. Due to the many novel methodological challenges it offers, this area has been growing in popularity among statisticians in recent years. In this article, we review the key developments in this exciting field of research. In particular, we discuss the sequential multiple assignment randomized trial designs, estimation techniques like Q-learning and marginal structural models, and several inference techniques designed to address the associated non-standard asymptotics. We reference software, whenever available. We also outline some important future directions.

SUBMITTER: Chakraborty B

PROVIDER: S-EPMC4231831 | biostudies-literature | 2014

REPOSITORIES: biostudies-literature

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Dynamic Treatment Regimes.

Chakraborty Bibhas B Murphy Susan A SA

Annual review of statistics and its application 20140101

A dynamic treatment regime consists of a sequence of decision rules, one per stage of intervention, that dictate how to individualize treatments to patients based on evolving treatment and covariate history. These regimes are particularly useful for managing chronic disorders, and fit well into the larger paradigm of personalized medicine. They provide one way to operationalize a clinical decision support system. Statistics plays a key role in the construction of evidence-based< ...[more]

PMID: 25401119

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Project description:Precision medicine is currently a topic of great interest in clinical and intervention science. A key component of precision medicine is that it is evidence-based, i.e., data-driven, and consequently there has been tremendous interest in estimation of precision medicine strategies using observational or randomized study data. One way to formalize precision medicine is through a treatment regime, which is a sequence of decision rules, one per stage of clinical intervention, that map up-to-date patient information to a recommended treatment. An optimal treatment regime is defined as maximizing the mean of some cumulative clinical outcome if applied to a population of interest. It is well-known that even under simple generative models an optimal treatment regime can be a highly nonlinear function of patient information. Consequently, a focal point of recent methodological research has been the development of flexible models for estimating optimal treatment regimes. However, in many settings, estimation of an optimal treatment regime is an exploratory analysis intended to generate new hypotheses for subsequent research and not to directly dictate treatment to new patients. In such settings, an estimated treatment regime that is interpretable in a domain context may be of greater value than an unintelligible treatment regime built using 'black-box' estimation methods. We propose an estimator of an optimal treatment regime composed of a sequence of decision rules, each expressible as a list of "if-then" statements that can be presented as either a paragraph or as a simple flowchart that is immediately interpretable to domain experts. The discreteness of these lists precludes smooth, i.e., gradient-based, methods of estimation and leads to non-standard asymptotics. Nevertheless, we provide a computationally efficient estimation algorithm, prove consistency of the proposed estimator, and derive rates of convergence. We illustrate the proposed methods using a series of simulation examples and application to data from a sequential clinical trial on bipolar disorder.

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Dynamic Treatment Regimes.

Publications

Dynamic Treatment Regimes.

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