Abstract
Many of the secondary outcomes in observational studies and randomized trials are rare. Methods for estimating causal effects and associations with rare outcomes, however, are limited, and this represents a missed opportunity for investigation. In this article, we construct a new targeted minimum loss-based estimator (TMLE) for the effect or association of an exposure on a rare outcome. We focus on the causal risk difference and statistical models incorporating bounds on the conditional mean of the outcome, given the exposure and measured confounders. By construction, the proposed estimator constrains the predicted outcomes to respect this model knowledge. Theoretically, this bounding provides stability and power to estimate the exposure effect. In finite sample simulations, the proposed estimator performed as well, if not better, than alternative estimators, including a propensity score matching estimator, inverse probability of treatment weighted (IPTW) estimator, augmented-IPTW and the standard TMLE algorithm. The new estimator yielded consistent estimates if either the conditional mean outcome or the propensity score was consistently estimated. As a substitution estimator, TMLE guaranteed the point estimates were within the...Continue Reading
References
Feb 1, 1993·Annals of Internal Medicine·J ConcatoT R Holford
Feb 28, 1996·Statistics in Medicine·F E HarrellD B Mark
Dec 1, 1996·Journal of Clinical Epidemiology·P PeduzziA R Feinstein
Aug 24, 1999·American Journal of Epidemiology·M M Joffe, P R Rosenbaum
Aug 24, 2000·Epidemiology·M A HernánJ M Robins
Oct 16, 2002·Annals of Internal Medicine·Leonard E Braitman, Paul R Rosenbaum
Jul 29, 2003·American Journal of Epidemiology·M Soledad CepedaBrian L Strom
Dec 22, 2006·American Journal of Epidemiology·Eric Vittinghoff, Charles E McCulloch
May 17, 2007·American Journal of Epidemiology·Roger MånssonSean Hennessy
Jul 4, 2007·Archives of General Psychiatry·Deborah S HasinBridget F Grant
Oct 4, 2007·Statistical Applications in Genetics and Molecular Biology·Mark J van der LaanAlan E Hubbard
Mar 4, 2008·American Journal of Epidemiology·Jennifer AhernS Leonard Syme
Oct 30, 2010·Statistical Methods in Medical Research·Maya L PetersenMark J van der Laan
Feb 6, 2013·Biometrics·Susan Gruber, Mark J van der Laan
Jul 17, 2013·Journal of Clinical Epidemiology·Samuel D LendleMark J van der Laan
Citations
Oct 11, 2017·Epidemiology·Richard WyssJessica M Franklin
May 27, 2020·Epidemiology·Laura B BalzerMaya L Petersen
Apr 25, 2018·Statistics in Medicine·Miguel Angel Luque-FernandezMireille E Schnitzer
Jul 4, 2017·Statistics in Medicine·David BenkeserPeter B Gilbert
Apr 4, 2021·Journal of Clinical Medicine·Jae-Woo JuTae Kyong Kim