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Review
. 2009:30:1-25.
doi: 10.1146/annurev.publhealth.031308.100223.

Adaptive designs for randomized trials in public health

C Hendricks Brown  1 , Thomas R Ten Have, Booil Jo, Getachew Dagne, Peter A Wyman, Bengt Muthén, Robert D Gibbons
Affiliations
Review

Adaptive designs for randomized trials in public health

C Hendricks Brown et al. Annu Rev Public Health. 2009.

Abstract

In this article, we present a discussion of two general ways in which the traditional randomized trial can be modified or adapted in response to the data being collected. We use the term adaptive design to refer to a trial in which characteristics of the study itself, such as the proportion assigned to active intervention versus control, change during the trial in response to data being collected. The term adaptive sequence of trials refers to a decision-making process that fundamentally informs the conceptualization and conduct of each new trial with the results of previous trials. Our discussion below investigates the utility of these two types of adaptations for public health evaluations. Examples are provided to illustrate how adaptation can be used in practice. From these case studies, we discuss whether such evaluations can or should be analyzed as if they were formal randomized trials, and we discuss practical as well as ethical issues arising in the conduct of these new-generation trials.

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Figures

Figure 1
Figure 1
Adaptive designs leading to early stopping or modifying allocation probabilities.
Figure 2
Figure 2
A typical sequencing of trials.
Figure 3
Figure 3
Cumulative adaptive trials with continuous quality improvement.
See this image and copyright information in PMC

References

    1. Angrist JD, Imbens GW, Rubin DB. Identification of causal effects using instrumental variables. J. Am. Stat. Assoc. 1996;91:444–55.
    1. Ayanlowo A, Redden D. A two-stage conditional power adaptive design adjusting for treatment by covariate interaction. Contemp. Clin. Trials. 2008;29:428–38. - PubMed
    1. Barnard J, Frangakis CE, Hill JL, Rubin DB. Principal stratification approach to broken randomized experiments: a case study of school choice vouchers in New York City/Comment/Rejoinder. J. Am. Stat. Assoc. 2003;98:299–323.
    1. Bartlett RH, Gazzaniga AB, Jefferies MR, Huxtable RF, Haiduc NJ, Fong SW. Extracorporeal membrane oxygenation (ECMO) cardiopulmonary support in infancy. Trans. Am. Soc. Artif. Intern. Organs. 1976;22:80–93. - PubMed
    1. Bartlett RH, Roloff DW, Cornell RG, Andrews AF, Dillon PW, Zwischenberger JB. Extracorporeal circulation in neonatal respiratory failure: a prospective randomized study. Pediatrics. 1985;76:479–87. - PubMed

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