Adaptive Designs for Randomized Trials in Public Health

Overview

Journal Annu Rev Public Health

Specialty Public Health

Date 2009 Mar 20

PMID 19296774

Citations 72

Authors

C Hendricks Brown

Thomas R Ten Have

Booil Jo

Getachew Dagne

Peter A Wyman

Bengt Muthen

Robert D Gibbons

Affiliations

Soon will be listed here.

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.

Citing Articles

Adaptive designs in critical care trials: a simulation study.

Li W, Cornelius V, Finfer S, Venkatesh B, Billot L BMC Med Res Methodol. 2023; 23(1):236.

PMID: 37853343 PMC: 10585789. DOI: 10.1186/s12874-023-02049-6.

Rationale and design for Healthy Hearts for Michigan (HH4M): A pragmatic single-arm hybrid effectiveness-implementation study.

Krefman A, Ciolino J, Kan A, Maki B, McHugh M, Smith J Contemp Clin Trials Commun. 2023; 35:101199.

PMID: 37671245 PMC: 10475469. DOI: 10.1016/j.conctc.2023.101199.

Impact of Sources of Strength on adolescent suicide deaths across three randomized trials.

Wyman P, Cero I, Brown C, Espelage D, Pisani A, Kuehl T Inj Prev. 2023; 29(5):442-445.

PMID: 37507212 PMC: 10579464. DOI: 10.1136/ip-2023-044944.

Whose knowledge counts? Involving communities in intervention and trial design using community conversations.

Burgess R, Shittu F, Iuliano A, Haruna I, Valentine P, Bakare A Trials. 2023; 24(1):385.

PMID: 37287035 PMC: 10249250. DOI: 10.1186/s13063-023-07320-1.

Expanding population-level interventions to help more low-income smokers quit: Study protocol for a randomized controlled trial.

Wolff J, McQueen A, Garg R, Thompson T, Fu Q, Brown D Contemp Clin Trials. 2023; 129:107202.

PMID: 37080354 PMC: 11440634. DOI: 10.1016/j.cct.2023.107202.

References

Gallo P, Chuang-Stein C, Dragalin V, Gaydos B, Krams M, Pinheiro J . Adaptive designs in clinical drug development--an Executive Summary of the PhRMA Working Group. J Biopharm Stat. 2006; 16(3):275-83. DOI: 10.1080/10543400600614742. View

McDonald C, Hui S, Tierney W . Effects of computer reminders for influenza vaccination on morbidity during influenza epidemics. MD Comput. 1992; 9(5):304-12. View

Zhang X, Cutter G . Bayesian interim analysis in clinical trials. Contemp Clin Trials. 2008; 29(5):751-5. DOI: 10.1016/j.cct.2008.05.007. View

Bhaumik D, Roy A, Aryal S, Hur K, Duan N, Normand S . Sample Size Determination for Studies with Repeated Continuous Outcomes. Psychiatr Ann. 2009; 38(12):765-771. PMC: 2743342. DOI: 10.3928/00485713-20081201-01. View

Thall P, Russell K . A strategy for dose-finding and safety monitoring based on efficacy and adverse outcomes in phase I/II clinical trials. Biometrics. 1998; 54(1):251-64. View

Zelen M . A new design for randomized clinical trials. N Engl J Med. 1979; 300(22):1242-5. DOI: 10.1056/NEJM197905313002203. View

Muthen B, Brown C, Masyn K, Jo B, Khoo S, Yang C . General growth mixture modeling for randomized preventive interventions. Biostatistics. 2003; 3(4):459-75. DOI: 10.1093/biostatistics/3.4.459. View

Collins L, Murphy S, Bierman K . A conceptual framework for adaptive preventive interventions. Prev Sci. 2004; 5(3):185-96. PMC: 3544191. DOI: 10.1023/b:prev.0000037641.26017.00. View

Brown C, Wang W, Kellam S, Muthen B, Petras H, Toyinbo P . Methods for testing theory and evaluating impact in randomized field trials: intent-to-treat analyses for integrating the perspectives of person, place, and time. Drug Alcohol Depend. 2008; 95 Suppl 1:S74-S104. PMC: 2560173. DOI: 10.1016/j.drugalcdep.2007.11.013. View

10.

Lake S, Kammann E, Klar N, Betensky R . Sample size re-estimation in cluster randomization trials. Stat Med. 2002; 21(10):1337-50. DOI: 10.1002/sim.1121. View

11.

Perry C, Bishop D, Taylor G, Davis M, Story M, Gray C . A randomized school trial of environmental strategies to encourage fruit and vegetable consumption among children. Health Educ Behav. 2004; 31(1):65-76. DOI: 10.1177/1090198103255530. View

12.

Lossius M, Hessen E, Mowinckel P, Stavem K, Erikssen J, Gulbrandsen P . Consequences of antiepileptic drug withdrawal: a randomized, double-blind study (Akershus Study). Epilepsia. 2007; 49(3):455-63. DOI: 10.1111/j.1528-1167.2007.01323.x. View

13.

Pillow D, Sandler I, Braver S, Wolchik S, Gersten J . Theory-based screening for prevention: focusing on mediating processes in children of divorce. Am J Community Psychol. 1991; 19(6):809-36. DOI: 10.1007/BF00937883. View

14.

Lang J . The use of a run-in to enhance compliance. Stat Med. 1990; 9(1-2):87-93; discussion 93-5. DOI: 10.1002/sim.4780090115. View

15.

Frangakis C, Brookmeyer R, Varadhan R, Safaeian M, Vlahov D, Strathdee S . Methodology for Evaluating a Partially Controlled Longitudinal Treatment Using Principal Stratification, With Application to a Needle Exchange Program. J Am Stat Assoc. 2015; 99(465):239-249. PMC: 4332568. DOI: 10.1198/016214504000000232. View

16.

Freedman B . Equipoise and the ethics of clinical research. N Engl J Med. 1987; 317(3):141-5. DOI: 10.1056/NEJM198707163170304. View

17.

Gould M, Petrie K, Kleinman M, Wallenstein S . Clustering of attempted suicide: New Zealand national data. Int J Epidemiol. 1994; 23(6):1185-9. DOI: 10.1093/ije/23.6.1185. View

18.

Zohar S, Chevret S . Recent developments in adaptive designs for Phase I/II dose-finding studies. J Biopharm Stat. 2007; 17(6):1071-83. DOI: 10.1080/10543400701645116. View

19.

Bekele B, Shen Y . A Bayesian approach to jointly modeling toxicity and biomarker expression in a phase I/II dose-finding trial. Biometrics. 2005; 61(2):343-54. DOI: 10.1111/j.1541-0420.2005.00314.x. View

20.

Inoue L, Thall P, Berry D . Seamlessly expanding a randomized phase II trial to phase III. Biometrics. 2002; 58(4):823-31. DOI: 10.1111/j.0006-341x.2002.00823.x. View