title: Propensity Score Methods for Analyzing Observational Data Like Randomized Experiments: Challenges and Solutions for Rare Outcomes and Exposures.
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.
authors.forename: Michelle E
authors.initials: ME
authors.lastname: Ross
authors.forename: Amanda R
authors.initials: AR
authors.lastname: Kreider
authors.forename: Yuan-Shung
authors.initials: YS
authors.lastname: Huang
authors.forename: Meredith
authors.initials: M
authors.lastname: Matone
authors.forename: David M
authors.initials: DM
authors.lastname: Rubin
authors.forename: A Russell
authors.initials: AR
authors.lastname: Localio
mesh_terms: D000293:Adolescent; D014150:Antipsychotic Agents / Q000009:adverse effects; D015984:Causality; D002648:Child; D015986:Confounding Factors, Epidemiologic; D003627:Data Interpretation, Statistical; D003924:Diabetes Mellitus, Type 2 / Q000139:chemically induced; D015340:Epidemiologic Research Design; D005260:Female; D006801:Humans; D057194:Intention to Treat Analysis; D008137:Longitudinal Studies; D008297:Male; D016555:Matched-Pair Analysis; D015233:Models, Statistical; D064887:Observational Studies as Topic; D057216:Propensity Score; D016032:Randomized Controlled Trials as Topic
chemical_list: D014150:Antipsychotic Agents
keywords: confounding; discrete-time failure analysis; inverse probability of treatment weighting; marginal effects; observational study; propensity score matching; randomized experiments