A Proposal for Integrated Efficacy-to-Effectiveness (E2E) Clinical Trials
Author(s)
Selker, H. P.; Eichler, H.-G.; Stockbridge, N. L.; Mehta, C. R.; Kaitin, K. I.; McElwee, N. E.; Honig, P. K.; Erban, J. K.; D’Agostino, R. B.; Oye, Kenneth A.; ... Show more Show less
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We propose an “efficacy-to-effectiveness” (E2E) clinical trial design, in which an effectiveness trial would commence seamlessly upon completion of the efficacy trial. Efficacy trials use inclusion/exclusion criteria to produce relatively homogeneous samples of participants with the target condition, conducted in settings that foster adherence to rigorous clinical protocols. Effectiveness trials use inclusion/exclusion criteria that generate heterogeneous samples that are more similar to the general patient spectrum, conducted in more varied settings, with protocols that approximate typical clinical care. In E2E trials, results from the efficacy trial component would be used to design the effectiveness trial component, to confirm and/or discern associations between clinical characteristics and treatment effects in typical care, and potentially to test new hypotheses. An E2E approach may improve the evidentiary basis for selecting treatments, expand understanding of the effectiveness of treatments in subgroups with particular clinical features, and foster incorporation of effectiveness information into regulatory processes.
Date issued
2013-09Department
Massachusetts Institute of Technology. Department of Political Science; Massachusetts Institute of Technology. Engineering Systems DivisionJournal
Clinical Pharmacology & Therapeutics
Publisher
Wiley Blackwell
Citation
Selker, H P, K A Oye, H-G Eichler, N L Stockbridge, C R Mehta, K I Kaitin, N E McElwee, P K Honig, J K Erban, and R B D’Agostino. “A Proposal for Integrated Efficacy-to-Effectiveness (E2E) Clinical Trials.” Clinical Pharmacology & Therapeutics 95, no. 2 (September 23, 2013): 147–153.
Version: Final published version
ISSN
0009-9236
1532-6535