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Adding flexibility to clinical trial designs: an example-based guide to the practical use of adaptive designs

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Adding flexibility to clinical trial designs : an example-based guide to the practical use of adaptive designs. / Burnett, T.; Mozgunov, P.; Pallmann, P.; Villar, S.S.; Wheeler, G.M.; Jaki, T.

In: BMC Medicine, Vol. 18, No. 1, 352, 19.11.2020.

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@article{fe3f341b69f14ed9b2d39c9a46b34bca,
title = "Adding flexibility to clinical trial designs: an example-based guide to the practical use of adaptive designs",
abstract = "Adaptive designs for clinical trials permit alterations to a study in response to accumulating data in order to make trials more flexible, ethical, and efficient. These benefits are achieved while preserving the integrity and validity of the trial, through the pre-specification and proper adjustment for the possible alterations during the course of the trial. Despite much research in the statistical literature highlighting the potential advantages of adaptive designs over traditional fixed designs, the uptake of such methods in clinical research has been slow. One major reason for this is that different adaptations to trial designs, as well as their advantages and limitations, remain unfamiliar to large parts of the clinical community. The aim of this paper is to clarify where adaptive designs can be used to address specific questions of scientific interest; we introduce the main features of adaptive designs and commonly used terminology, highlighting their utility and pitfalls, and illustrate their use through case studies of adaptive trials ranging from early-phase dose escalation to confirmatory phase III studies. {\textcopyright} 2020, The Author(s).",
keywords = "Efficient methods, Enrichment designs, Innovative trials, Multi-arm multi-stage platform trials, Novel designs, adaptive clinical trial, article, controlled study, human, nomenclature, phase 3 clinical trial",
author = "T. Burnett and P. Mozgunov and P. Pallmann and S.S. Villar and G.M. Wheeler and T. Jaki",
year = "2020",
month = nov,
day = "19",
doi = "10.1186/s12916-020-01808-2",
language = "English",
volume = "18",
journal = "BMC Medicine",
issn = "1741-7015",
publisher = "BIOMED CENTRAL LTD",
number = "1",

}

RIS

TY - JOUR

T1 - Adding flexibility to clinical trial designs

T2 - an example-based guide to the practical use of adaptive designs

AU - Burnett, T.

AU - Mozgunov, P.

AU - Pallmann, P.

AU - Villar, S.S.

AU - Wheeler, G.M.

AU - Jaki, T.

PY - 2020/11/19

Y1 - 2020/11/19

N2 - Adaptive designs for clinical trials permit alterations to a study in response to accumulating data in order to make trials more flexible, ethical, and efficient. These benefits are achieved while preserving the integrity and validity of the trial, through the pre-specification and proper adjustment for the possible alterations during the course of the trial. Despite much research in the statistical literature highlighting the potential advantages of adaptive designs over traditional fixed designs, the uptake of such methods in clinical research has been slow. One major reason for this is that different adaptations to trial designs, as well as their advantages and limitations, remain unfamiliar to large parts of the clinical community. The aim of this paper is to clarify where adaptive designs can be used to address specific questions of scientific interest; we introduce the main features of adaptive designs and commonly used terminology, highlighting their utility and pitfalls, and illustrate their use through case studies of adaptive trials ranging from early-phase dose escalation to confirmatory phase III studies. © 2020, The Author(s).

AB - Adaptive designs for clinical trials permit alterations to a study in response to accumulating data in order to make trials more flexible, ethical, and efficient. These benefits are achieved while preserving the integrity and validity of the trial, through the pre-specification and proper adjustment for the possible alterations during the course of the trial. Despite much research in the statistical literature highlighting the potential advantages of adaptive designs over traditional fixed designs, the uptake of such methods in clinical research has been slow. One major reason for this is that different adaptations to trial designs, as well as their advantages and limitations, remain unfamiliar to large parts of the clinical community. The aim of this paper is to clarify where adaptive designs can be used to address specific questions of scientific interest; we introduce the main features of adaptive designs and commonly used terminology, highlighting their utility and pitfalls, and illustrate their use through case studies of adaptive trials ranging from early-phase dose escalation to confirmatory phase III studies. © 2020, The Author(s).

KW - Efficient methods

KW - Enrichment designs

KW - Innovative trials

KW - Multi-arm multi-stage platform trials

KW - Novel designs

KW - adaptive clinical trial

KW - article

KW - controlled study

KW - human

KW - nomenclature

KW - phase 3 clinical trial

U2 - 10.1186/s12916-020-01808-2

DO - 10.1186/s12916-020-01808-2

M3 - Journal article

VL - 18

JO - BMC Medicine

JF - BMC Medicine

SN - 1741-7015

IS - 1

M1 - 352

ER -