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2016 ; 35
(8
): 1245-56
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Using pilot data to size a two-arm randomized trial to find a nearly optimal
personalized treatment strategy
#MMPMID26506890
Laber EB
; Zhao YQ
; Regh T
; Davidian M
; Tsiatis A
; Stanford JB
; Zeng D
; Song R
; Kosorok MR
Stat Med
2016[Apr]; 35
(8
): 1245-56
PMID26506890
show ga
A personalized treatment strategy formalizes evidence-based treatment selection
by mapping patient information to a recommended treatment. Personalized treatment
strategies can produce better patient outcomes while reducing cost and treatment
burden. Thus, among clinical and intervention scientists, there is a growing
interest in conducting randomized clinical trials when one of the primary aims is
estimation of a personalized treatment strategy. However, at present, there are
no appropriate sample size formulae to assist in the design of such a trial.
Furthermore, because the sampling distribution of the estimated outcome under an
estimated optimal treatment strategy can be highly sensitive to small
perturbations in the underlying generative model, sample size calculations based
on standard (uncorrected) asymptotic approximations or computer simulations may
not be reliable. We offer a simple and robust method for powering a single stage,
two-armed randomized clinical trial when the primary aim is estimating the
optimal single stage personalized treatment strategy. The proposed method is
based on inverting a plugin projection confidence interval and is thereby regular
and robust to small perturbations of the underlying generative model. The
proposed method requires elicitation of two clinically meaningful parameters from
clinical scientists and uses data from a small pilot study to estimate nuisance
parameters, which are not easily elicited. The method performs well in simulated
experiments and is illustrated using data from a pilot study of time to
conception and fertility awareness.
|Biostatistics
[MESH]
|Computer Simulation
[MESH]
|Confidence Intervals
[MESH]
|Data Interpretation, Statistical
[MESH]
|Evidence-Based Practice/statistics & numerical data
[MESH]
|Female
[MESH]
|Fertility
[MESH]
|Humans
[MESH]
|Male
[MESH]
|Models, Statistical
[MESH]
|Pilot Projects
[MESH]
|Precision Medicine/statistics & numerical data
[MESH]
|Pregnancy
[MESH]
|Randomized Controlled Trials as Topic/*statistics & numerical data
[MESH]