Two-stage randomized trial design for testing treatment, preference, and self-selection effects for count outcomes.
count outcome
preference effect
two-stage clinical trials
Journal
Statistics in medicine
ISSN: 1097-0258
Titre abrégé: Stat Med
Pays: England
ID NLM: 8215016
Informations de publication
Date de publication:
10 11 2020
10 11 2020
Historique:
received:
01
04
2020
revised:
08
06
2020
accepted:
13
06
2020
pubmed:
3
9
2020
medline:
22
6
2021
entrez:
3
9
2020
Statut:
ppublish
Résumé
While the traditional clinical trial design lays emphasis on testing the treatment effect between randomly assigned groups, it ignores the role of patient preference for a particular treatment in the trial. Yet, for healthcare providers who seek to optimize the patient-centered treatment strategy, the evaluation of a patient's psychology toward each treatment could be a key consideration. The two-stage randomized trial design allows researchers to test patient's preference and selection effects, in addition to the treatment effect. The current methodology for the two-stage design is limited to continuous and binary outcomes; this article extends the model to include count outcomes. The test statistics for preference, selection, and treatment effects are derived. Closed-form sample size formulae are presented for each effect. Simulations are presented to demonstrate the properties of the unstratified and stratified designs. Finally, we apply methods to the use of antimicrobials at the end of life to demonstrate the applicability of the methods.
Types de publication
Journal Article
Randomized Controlled Trial
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
3653-3683Subventions
Organisme : NCATS NIH HHS
ID : UL1TR001863
Pays : United States
Informations de copyright
© 2020 John Wiley & Sons, Ltd.
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