Some methods for heterogeneous treatment effect estimation in high dimensions

Stat Med. 2018 May 20;37(11):1767-1787. doi: 10.1002/sim.7623. Epub 2018 Mar 6.

Abstract

When devising a course of treatment for a patient, doctors often have little quantitative evidence on which to base their decisions, beyond their medical education and published clinical trials. Stanford Health Care alone has millions of electronic medical records that are only just recently being leveraged to inform better treatment recommendations. These data present a unique challenge because they are high dimensional and observational. Our goal is to make personalized treatment recommendations based on the outcomes for past patients similar to a new patient. We propose and analyze 3 methods for estimating heterogeneous treatment effects using observational data. Our methods perform well in simulations using a wide variety of treatment effect functions, and we present results of applying the 2 most promising methods to data from The SPRINT Data Analysis Challenge, from a large randomized trial of a treatment for high blood pressure.

Keywords: causal inference; machine learning; personalized medicine.

Publication types

  • Validation Study

MeSH terms

  • Algorithms
  • Biostatistics / methods*
  • Causality
  • Computer Simulation
  • Decision Making*
  • Electronic Health Records / statistics & numerical data
  • Humans
  • Machine Learning / statistics & numerical data
  • Observational Studies as Topic / statistics & numerical data
  • Patient-Specific Modeling / statistics & numerical data
  • Precision Medicine / statistics & numerical data
  • Propensity Score
  • Randomized Controlled Trials as Topic / statistics & numerical data
  • Regression Analysis
  • Treatment Outcome*