A Proposal for Integrated Efficacy-to-Effectiveness (E2E) Clinical Trials
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...
| Main Authors: | , , , , , , , , , |
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| Format: | Article |
| Language: | en_US |
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Wiley Blackwell
2015
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| Online Access: | http://hdl.handle.net/1721.1/96173 https://orcid.org/0000-0002-9811-8415 |
| _version_ | 1826197381851381760 |
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| author | 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. |
| author2 | Massachusetts Institute of Technology. Department of Political Science |
| author_facet | Massachusetts Institute of Technology. Department of Political Science 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. |
| author_sort | Selker, H. P. |
| collection | MIT |
| description | 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. |
| first_indexed | 2024-09-23T10:47:10Z |
| format | Article |
| id | mit-1721.1/96173 |
| institution | Massachusetts Institute of Technology |
| language | en_US |
| last_indexed | 2024-09-23T10:47:10Z |
| publishDate | 2015 |
| publisher | Wiley Blackwell |
| record_format | dspace |
| spelling | mit-1721.1/961732022-09-27T14:57:53Z A Proposal for Integrated Efficacy-to-Effectiveness (E2E) Clinical Trials 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. Massachusetts Institute of Technology. Department of Political Science Massachusetts Institute of Technology. Engineering Systems Division Oye, Kenneth A. 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. National Center for Research Resources (U.S.) (Grant UL1 RR025752) National Center for Advancing Translational Sciences (U.S.) (Grant UL1 TR000073) 2015-03-25T15:42:41Z 2015-03-25T15:42:41Z 2013-09 2013-08 Article http://purl.org/eprint/type/JournalArticle 0009-9236 1532-6535 http://hdl.handle.net/1721.1/96173 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. https://orcid.org/0000-0002-9811-8415 en_US http://dx.doi.org/10.1038/clpt.2013.177 Clinical Pharmacology & Therapeutics Creative Commons Attribution-NonCommercial-NoDerivs 3.0 License http://creativecommons.org/licenses/by-nc-nd/3.0/ application/pdf Wiley Blackwell Nature |
| spellingShingle | 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. A Proposal for Integrated Efficacy-to-Effectiveness (E2E) Clinical Trials |
| title | A Proposal for Integrated Efficacy-to-Effectiveness (E2E) Clinical Trials |
| title_full | A Proposal for Integrated Efficacy-to-Effectiveness (E2E) Clinical Trials |
| title_fullStr | A Proposal for Integrated Efficacy-to-Effectiveness (E2E) Clinical Trials |
| title_full_unstemmed | A Proposal for Integrated Efficacy-to-Effectiveness (E2E) Clinical Trials |
| title_short | A Proposal for Integrated Efficacy-to-Effectiveness (E2E) Clinical Trials |
| title_sort | proposal for integrated efficacy to effectiveness e2e clinical trials |
| url | http://hdl.handle.net/1721.1/96173 https://orcid.org/0000-0002-9811-8415 |
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