Selecting Optimal Subgroups for Treatment Using Many Covariates

Epidemiology
Tyler J VanderWeeleRonald C Kessler

Abstract

We consider the problem of selecting the optimal subgroup to treat when data on covariates are available from a randomized trial or observational study. We distinguish between four different settings including: (1) treatment selection when resources are constrained; (2) treatment selection when resources are not constrained; (3) treatment selection in the presence of side effects and costs; and (4) treatment selection to maximize effect heterogeneity. We show that, in each of these cases, the optimal treatment selection rule involves treating those for whom the predicted mean difference in outcomes comparing those with versus without treatment, conditional on covariates, exceeds a certain threshold. The threshold varies across these four scenarios, but the form of the optimal treatment selection rule does not. The results suggest a move away from the traditional subgroup analysis for personalized medicine. New randomized trial designs are proposed so as to implement and make use of optimal treatment selection rules in healthcare practice.

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Citations

Jun 18, 2019·Epidemiology·Tyler J VanderWeele
Nov 12, 2019·Annals of Internal Medicine·David M KentEwout W Steyerberg
Dec 27, 2019·American Journal of Health Promotion : AJHP·Jeffrey Lienert, Mitesh Patel
Apr 14, 2020·Epidemiology·Bas B L Penning de VriesAlex Luedtke
Dec 28, 2019·Epidemiology·David M Kent, David van Klaveren
Dec 28, 2019·Epidemiology·Tyler J VanderWeeleRonald C Kessler
Jun 3, 2020·Epidemiology·Tyler J VanderWeeleRonald C Kessler
Feb 18, 2021·European Urology·Susan Halabi, Andrew J Armstrong
May 5, 2021·Health Economics·Carl Bonander, Mikael Svensson
May 14, 2021·Statistics in Medicine·Siyun YangLaine E Thomas
May 30, 2021·Behaviour Research and Therapy·Shannon Wiltsey StirmanPaula P Schnurr
May 29, 2021·The Lancet. Digital Health·Chrianna BharatLouisa Degenhardt
Sep 23, 2021·JAMA Psychiatry·Ronald C Kessler, Alex Luedtke
Nov 6, 2021·International Journal of Methods in Psychiatric Research·Jose R ZubizarretaTeodor T Postolache

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