Adaptive Clinical Trials: Overview of Phase III Designs and Challenges. (January 2014)

• Record Type: Journal Article
• Authors: Maca, Jeff; Dragalin, Vladimir; Gallo, Paul
• Is Part Of: Therapeutic innovation & regulatory science. Volume 48:Number 1(2014:Jan.)
• Page Start: 31
• Page End: 40
• Publication Date: 2014-01
• Subjects: adaptive trial design -- enrichment design -- confirmatory studies -- sample size re-estimation -- seamless phase II/III; Drugs -- Periodicals; Pharmacology -- Periodicals; Drugs -- Research -- Periodicals; Drugs -- Testing -- Periodicals; 615.1
• DOI: 10.1177/2168479013507436
• Languages: English
• ISSNs: 2168-4790

Abstract

Adaptive designs use accruing data to make changes in an ongoing trial according to a prespecified plan and potentially offer great efficiencies for clinical development. There are many types of adaptive designs and many trial aspects that could in theory be adapted. However, the scope of adaptive designs with relevance in confirmatory trials is narrower, and in addition, extensive pre-planning is needed and various types of challenges need to be addressed in order to use these designs in this stage of development. Nevertheless, with careful planning, there are opportunities for these designs to offer important benefits even in the confirmatory stage of development. We provide an overview of adaptive designs that have relevance for confirmatory trials and discuss considerations that may affect whether they should or should not be used in particular trials or programs as well as the challenges that need to be addressed.