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Estimation of Survival Distributions of Treatment Policies in Two‐Stage Randomization Designs in Clinical Trials
Author(s) -
Lunceford Jared K.,
Davidian Marie,
Tsiatis Anastasios A.
Publication year - 2002
Publication title -
biometrics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.298
H-Index - 130
eISSN - 1541-0420
pISSN - 0006-341X
DOI - 10.1111/j.0006-341x.2002.00048.x
Subject(s) - randomization , clinical trial , estimator , randomized controlled trial , sample size determination , stage (stratigraphy) , statistics , set (abstract data type) , medicine , survival analysis , econometrics , mathematics , computer science , paleontology , biology , programming language
Summary. Some clinical trials follow a design where patients are randomized to a primary therapy at entry followed by another randomization to maintenance therapy contingent upon disease remission. Ideally, analysis would allow different treatment policies, i.e., combinations of primary and maintenance therapy if specified up‐front, to be compared. Standard practice is to conduct separate analyses for the primary and follow‐up treatments, which does not address this issue directly. We propose consistent estimators for the survival distribution and mean restricted survival time for each treatment policy in such two‐stage studies and derive large‐sample properties. The methods are demonstrated on a leukemia clinical trial data set and through simulation.