The mixed randomized trial: combining randomized, pragmatic and observational clinical trial designs.

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.

journal_name

J Comp Eff Res

authors

Alsop J,Scott M,Archey W

doi

10.2217/cer-2016-0034

subject

Has Abstract

pub_date

2016-11-01 00:00:00

pages

569-579

issue

6

eissn

2042-6305

issn

2042-6313

journal_volume

5

pub_type

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