Adaptive Design Practice at the Center for Devices and Radiological Health (CDRH),January 2007 to May 2013

Abstract

Adaptive designs have generated great interest in the clinical trial community as a result of their versatility and efficiency. Recently, the Center for Devices and Radiological Health (CDRH) at the US Food and Drug Administration (FDA) surveyed all adaptive design applications submitted between 2007 and May 2013 for regulatory review. In this paper, we discuss the overall results and findings that emerged from an in-depth examination of the submissions. We summarize the current status of adaptive designs used in medical device studies. We also identify some of the lessons learned and common pitfalls that we encountered in our review of the designs.

Keywords

adaptive design medical device survey FDA

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References

Food and Drug Administration. Draft guidance for industry and Food and Drug Administration staff: adaptive designs for medical device clinical studies. Rockville, MD: FDA, 2015.

Pocock

. Group sequential methods in the design and analysis of clinical trials. Biometrika. 1977;64:191–199.

O’Brien

Fleming

. A multiple testing procedure for clinical trials. Biometrics. 1979;35:549–556.

Lan

KKG

DeMets

. Group sequential procedures: calendar versus information time. Stat Med. 1987;8:1191–1198.

Proschan

Hunsberger

. Designed extension of studies based on conditional power. Biometrics. 1995;51:1315–1324.

Cui

Hung

Wang

. Modification of sample size in group sequential clinical trials. Biometrics. 1999;55:853–857.

Müller

Schäfer

. Adaptive group sequential designs for clinical trials: Combining the advantages of adaptive and of classical group sequential approaches. Biometrics. 2001;57:886–891.

Chen

YHJ

DeMets

Lan

KKG

. Increasing the sample size when the unblinded interim result is promising. Stat Med. 2004;23:1023–1038.

Gao

Ware

Mehta

. Sample size re-estimation for adaptive sequential design in clinical trials. J Biopharm Stat. 2008;18:1184–1196.

10.

Berry

Carlin

Lee

. Bayesian Adaptive Methods for Clinical Trials. Boca Raton, FL: CRC Press; 2011.

11.

Campbell

. Similarities and differences of Bayesian designs and adaptive designs for medical devices: a regulatory view. Stat Biopharm Res. 2013;5:356–368.

12.

Morgan

Huyck

Jenkins

. Adaptive Design: Results of 2012 Survey on Perception and Use. Therapeutic Innovation & Regulatory Science. 2014;48:473–81.

13.

Elsäßer

Regnstrom

Vetter

. Adaptive clinical trial designs for European marketing authorization: a survey of scientific advice letters from the European Medicines Agency. Trials. 2014;15:383.

14.

Lin

Lee

Zhen

. CBER’s experience with adaptive design clinical trials. Therapeutic Innovation & Regulatory Science. 2015; September 16 online.

15.

Shih

. Sample size re-estimation—journey for a decade. Stat Med. 2001;20:515–518.

16.

Food and Drug Administration. Guidance for the use of bayesian statistics in medical device clinical trials. Rockville, MD: FDA, 2010.

17.

Gelman

Carlin

Stern

Dunson

Vehtari

Rubin

. Bayesian Data Analysis. Boca Raton, FL: CRC Press; 2014.

18.

Pennello

Thompson

. Experience with reviewing Bayesian medical device trials. J Biopharm Stat. 2008;18:81–115.

19.

Campbell

. Bayesian statistics in medical devices: innovation sparked by the FDA. J Biopharm Stat. 2011;21:871–887.

20.

Schulz

Altman

Moher

. CONSORT 2010 Statement: updated guidelines for reporting parallel group randomised trials. BMJ 2010;340:c332.

21.

Moher

Hopewell

Schulz

. CONSORT 2010 Explanation and Elaboration: updated guidelines for reporting parallel group randomised trials. BMJ. 2010;340:c869.

22.

Detry

Lewis

Broglio

. Standards for the design, conduct, and evaluation of adaptive randomized clinical trials, http://www.pcori.org/assets/Standards-for-the-Design-Conduct-and-Evaluation-of-Adaptive-Randomized-Clinical-Trials.pdf. Published 2012. Accessed March 15, 2012.

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