Wason, James M. S., Jaki, Thomas and Stallard, Nigel (2013) Planning multi-arm screening studies within the context of a drug development program. Statistics in Medicine, Volume 32 (Number 20). pp. 3424-3435. doi:10.1002/sim.5787

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Abstract

Screening trials are small trials used to decide whether an intervention is sufficiently promising to warrant a large confirmatory trial. Previous literature examined the situation where treatments are tested sequentially until one is considered sufficiently promising to take forward to a confirmatory trial. An important consideration for sponsors of clinical trials is how screening trials should be planned to maximize the efficiency of the drug development process. It has been found previously that small screening trials are generally the most efficient. In this paper we consider the design of screening trials in which multiple new treatments are tested simultaneously. We derive analytic formulae for the expected number of patients until a successful treatment is found, and propose methodology to search for the optimal number of treatments, and optimal sample size per treatment. We compare designs in which only the best treatment proceeds to a confirmatory trial and designs in which multiple treatments may proceed to a multi-arm confirmatory trial. We find that inclusion of a large number of treatments in the screening trial is optimal when only one treatment can proceed, and a smaller number of treatments is optimal when more than one can proceed. The designs we investigate are compared on a real-life set of screening designs.

Item Type:	Journal Article
Subjects:	Q Science > QA Mathematics R Medicine > RM Therapeutics. Pharmacology
Divisions:	Faculty of Medicine > Warwick Medical School > Health Sciences Faculty of Medicine > Warwick Medical School
Library of Congress Subject Headings (LCSH):	Medical statistics, Drugs -- Testing , Clinical pharmacology
Journal or Publication Title:	Statistics in Medicine
Publisher:	John Wiley & Sons Ltd.
ISSN:	0277-6715
Official Date:	2013
Dates:	Date Event 2013 Published
Date of first compliant deposit:	25 December 2015
Volume:	Volume 32
Number:	Number 20
Page Range:	pp. 3424-3435
DOI:	10.1002/sim.5787
Status:	Peer Reviewed
Publication Status:	Published
Access rights to Published version:	Restricted or Subscription Access
Funder:	Medical Research Council (Great Britain) (MRC), National Institute for Health Research (Great Britain) (NIHR)
Grant number:	G0800860, MR/J004979/1 (MRC) ; NIHR-CDF-2010-03-32 (NIHR)

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