Randomized Discontinuation Trials

Valerii V. Fedorov ()
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Valerii V. Fedorov: ICON

Chapter 75 in Principles and Practice of Clinical Trials, 2022, pp 1439-1453 from Springer

Abstract: Abstract Randomized discontinuation trials are usually considered as a special case of population enrichment trials. Most often they consist of a few stages with the early ones targeting the selection of a subpopulation that responds or may respond to an experimental treatment and allowing an early escape of patients doing poorly. The succeeding stages validate the treatment’s superiority to placebo or more generally to a comparator for the selected (enriched with potential responders) subpopulation. The approach increases the trial feasibility and the chances of correct response-to-treatment detection, often at the expense of decreased ability to extrapolate results over the initially targeted population. The first randomized discontinuation trials were used exclusively to test long-term, non-curative therapies for chronic or slow evolving diseases. The development of stochastic longitudinal models combined with Bayesian ideas and the increase of computing power led to their acceptance in various therapeutic areas, most notably in oncology. If the statistically valid partitioning of the source population is proven, then it might be viewed as evidence of the existence of some classifiers and pave the road to precision medicine.

Keywords: Enrichment trials; Population enrichment; Randomized discontinuation trials; Screening; Subpopulation selection (search for similar items in EconPapers)
Date: 2022
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DOI: 10.1007/978-3-319-52636-2_104

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