The mixed randomized trial: combining randomized, pragmatic and observational clinical trial designs.
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.
Numerus Ltd, Wokingham, UK.
Jonathan
J
Alsop
Numerus Ltd, Wokingham, UK.
Martin
M
Scott
Numerus Analytics AG, Basel, Switzerland.
William
W
Archey
D064887:Observational Studies as Topic; D016032:Randomized Controlled Trials as Topic; D012107:Research Design
clinical trials; comparative effectiveness research; pragmatic clinical trials