Adaptive clinical trial design.

Journal Article (Journal Article;Review)

In recent years, the use of adaptive design methods in clinical trials based on accumulated data at interim has received much attention because of its flexibility and efficiency in pharmaceutical/clinical development. In practice, adaptive design may provide the investigators a second chance to modify or redesign the trial while the study is still ongoing. However, it is a concern that a shift in target patient population may occur after significant adaptations are made. In addition, the overall type I error rate may not be preserved. Moreover, the results may not be reliable and hence are difficult to interpret. As indicated by the US Food and Drug Administration draft guidance on adaptive design clinical trials, the adaptive design has to be a prospectively planned opportunity and should be based on information collected within the study, with or without formal statistical hypothesis testing. This article reviews the relative advantages, limitations, and feasibility of commonly considered adaptive designs in clinical trials. Statistical concerns when implementing adaptive designs are also discussed.

Full Text

Duke Authors

Cited Authors

  • Chow, S-C

Published Date

  • 2014

Published In

Volume / Issue

  • 65 /

Start / End Page

  • 405 - 415

PubMed ID

  • 24422576

Electronic International Standard Serial Number (EISSN)

  • 1545-326X

Digital Object Identifier (DOI)

  • 10.1146/annurev-med-092012-112310


  • eng

Conference Location

  • United States