Researchers of uncommon diseases are often interested in assessing potential risk factors. Given the low incidence of disease, these studies are frequently case-control in design. Such a design allows a sufficient number of cases to be obtained without extensive sampling and can increase efficiency; however, these case-control samples are then biased since the proportion of cases in the sample is not the same as the population of interest. Methods for analyzing case-control studies have focused on utilizing logistic regression models that provide conditional and not causal estimates of the odds ratio. This article will demonstrate the use of the prevalence probability and case-control weighted targeted maximum likelihood estimation (MLE), as described by van der Laan (2008), in order to obtain causal estimates of the parameters of interest (risk difference, relative risk, and odds ratio). It is meant to be used as a guide for researchers, with step-by-step directions to implement this methodology. We will also present simulation studies that show the improved efficiency of the case-control weighted targeted MLE compared to other techniques.

Ed. by Hubbard, Alan E. / van der Laan, Mark J.
1 Issue per year
IMPACT FACTOR 2011: 1.284
Issues
Volume 7 (2011)
Volume 6 (2010)
Volume 5 (2009)
Volume 4 (2008)
Volume 3 (2007)
Volume 2 (2006)
Volume 1 (2005)
Most Downloaded Articles
- An Introduction to Causal Inference by Pearl, Judea
- Meta-Analysis of Observational Studies with Unmeasured Confounders by McCandless, Lawrence C.
- Accuracy of Conventional and Marginal Structural Cox Model Estimators: A Simulation Study by Xiao, Yongling/ Abrahamowicz, Michal and Moodie, Erica E. M.
- Evaluating treatment effectiveness in patient subgroups: a comparison of propensity score methods with an automated matching approach by Radice, Rosalba/ Ramsahai, Roland/ Grieve, Richard/ Kreif, Noemi/ Sadique, Zia and Sekhon, Jasjeet S.
- Special Issue on Causal Inference in Health Research by Moodie, Erica E. M./ Kaufman, Jay S. and Platt, Robert W.
Simple Optimal Weighting of Cases and Controls in Case-Control Studies
Sherri Rose / Mark J. van der Laan
1University of California, Berkeley
1University of California, Berkeley
Citation Information: The International Journal of Biostatistics. Volume 4, Issue 1, Pages 1–24, ISSN (Online) 1557-4679, DOI: 10.2202/1557-4679.1115, September 2008
Publication History:
- Published Online:
- 2008-09-29
Keywords: case control sampling; causal effect; counterfactual; double robust estimation; estimating function; inverse probability of treatment weighting; locally efficient estimation; marginal structural models; targeted maximum likelihood estimation


















Comments (0)