Propensity Score Methods for Analyzing Observational Data Like Randomized Experiments: Challenges and Solutions for Rare Outcomes and Exposures

Am J Epidemiol. 2015 Jun 15;181(12):989-95. doi: 10.1093/aje/kwu469. Epub 2015 May 20.

Abstract

Randomized controlled trials are the "gold standard" for estimating the causal effects of treatments. However, it is often not feasible to conduct such a trial because of ethical concerns or budgetary constraints. We expand upon an approach to the analysis of observational data sets that mimics a sequence of randomized studies by implementing propensity score models within each trial to achieve covariate balance, using weighting and matching. The methods are illustrated using data from a safety study of the relationship between second-generation antipsychotics and type 2 diabetes (outcome) in Medicaid-insured children aged 10-18 years across the United States from 2003 to 2007. Challenges in this data set include a rare outcome, a rare exposure, substantial and important differences between exposure groups, and a very large sample size.

Keywords: confounding; discrete-time failure analysis; inverse probability of treatment weighting; marginal effects; observational study; propensity score matching; randomized experiments.

Publication types

  • Evaluation Study
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adolescent
  • Antipsychotic Agents / adverse effects
  • Causality
  • Child
  • Confounding Factors, Epidemiologic
  • Data Interpretation, Statistical*
  • Diabetes Mellitus, Type 2 / chemically induced
  • Epidemiologic Research Design*
  • Female
  • Humans
  • Intention to Treat Analysis
  • Longitudinal Studies
  • Male
  • Matched-Pair Analysis
  • Models, Statistical
  • Observational Studies as Topic*
  • Propensity Score*
  • Randomized Controlled Trials as Topic

Substances

  • Antipsychotic Agents