The mixed randomized trial: combining randomized, pragmatic and observational clinical trial designs. (November 2016)

• Record Type: Journal Article
• Authors: Alsop, Jonathan; Scott, Martin; Archey, William
• Is Part Of: Journal of comparative effectiveness research. Volume 5:Number 6(2016)
• Page Start: 569
• Page End: 579
• Publication Date: 2016-11
• Subjects: clinical trials -- comparative effectiveness research -- pragmatic clinical trials; Medical care -- Research -- Periodicals; Medical care -- Research -- Evaluation -- Periodicals; Medical care -- Decision making -- Periodicals; Evidence-based medicine -- Periodicals; Medicine, Comparative -- Periodicals; 362.1072
• DOI: 10.2217/cer-2016-0034
• Languages: English
• ISSNs: 2042-6305

Abstract

Clinical trial designs often fail to deliver data that jointly satisfy evolving demands of both regulatory and reimbursement authorities. We propose a new multi-tiered trial design to integrate efficacy and effectiveness, and address the evolving needs of authorities. The mixed randomized trial allocates patients first to trial arm – randomized controlled, pragmatic (randomized) or observational – and then to treatment group – experimental, placebo, active comparator, best available therapy or standard of care. Trial arms may be staggered over time to reflect the current state of randomized and non-randomized data of the experimental drug, and thereby still prioritize safety. At the same time, the mixed randomized trial allows for the collection of real-world data in a randomized setting, and thereby reduces selection bias.