MIT Libraries logoDSpace@MIT

MIT
View Item 
  • DSpace@MIT Home
  • MIT Open Access Articles
  • MIT Open Access Articles
  • View Item
  • DSpace@MIT Home
  • MIT Open Access Articles
  • MIT Open Access Articles
  • View Item
JavaScript is disabled for your browser. Some features of this site may not work without it.

Efficacy and Effectiveness Too Trials: Clinical Trial Designs to Generate Evidence on Efficacy and on Effectiveness in Wide Practice

Author(s)
Selker, Harry P.; Eichler, Hans‐Georg; Stockbridge, Norman L.; McElwee, Newell E.; Dere, Willard H.; Cohen, Theodora; Erban, John K.; Seyfert‐Margolis, Vicki L.; Honig, Peter K.; Kaitin, Kenneth I.; Oye, Kenneth A; D'Agostino, Ralph B.; ... Show more Show less
Thumbnail
DownloadPublished version (437.3Kb)
Publisher with Creative Commons License

Publisher with Creative Commons License

Creative Commons Attribution

Terms of use
Creative Commons Attribution NonCommercial License 4.0 https://creativecommons.org/licenses/by-nc/4.0/
Metadata
Show full item record
Abstract
Efficacy trials, designed to gain regulatory marketing approval, evaluate drugs in optimally selected patients under advantageous conditions for relatively short time periods. Effectiveness trials, designed to evaluate use in usual practice, assess treatments among more typical patients in real-world conditions with longer follow-up periods. In “efficacy-to-effectiveness (E2E) trials,” if the initial efficacy trial component is positive, the trial seamlessly transitions to an effectiveness trial component to efficiently yield both types of evidence. Yet more time could be saved by simultaneously addressing efficacy and effectiveness in an “efficacy and effectiveness too (EE2) trial.” Additionally, hybrids of the E2E and EE2 approaches with differing degrees of overlap of the two components could allow flexibility for specific drug development needs. In planning EE2 trials, each stakeholder's current and future needs, incentives, and perspective must be considered. Although challenging, the ultimate benefits to stakeholders, the health system, and the public should justify this effort.
Date issued
2019-02
URI
https://hdl.handle.net/1721.1/128557
Department
Massachusetts Institute of Technology. Department of Political Science; Massachusetts Institute of Technology. Center for Biomedical Innovation
Journal
Clinical Pharmacology and Therapeutics
Publisher
Wiley
Citation
Selker, Harry P. et al. "Efficacy and Effectiveness Too Trials: Clinical Trial Designs to Generate Evidence on Efficacy and on Effectiveness in Wide Practice." Clinical Pharmacology and Therapeutics 105, 4 (February 2019): 857-866 © 2019 The Authors
Version: Final published version
ISSN
0009-9236
1532-6535

Collections
  • MIT Open Access Articles

Browse

All of DSpaceCommunities & CollectionsBy Issue DateAuthorsTitlesSubjectsThis CollectionBy Issue DateAuthorsTitlesSubjects

My Account

Login

Statistics

OA StatisticsStatistics by CountryStatistics by Department
MIT Libraries
PrivacyPermissionsAccessibilityContact us
MIT
Content created by the MIT Libraries, CC BY-NC unless otherwise noted. Notify us about copyright concerns.