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dc.contributor.authorSelker, Harry P.
dc.contributor.authorEichler, Hans‐Georg
dc.contributor.authorStockbridge, Norman L.
dc.contributor.authorMcElwee, Newell E.
dc.contributor.authorDere, Willard H.
dc.contributor.authorCohen, Theodora
dc.contributor.authorErban, John K.
dc.contributor.authorSeyfert‐Margolis, Vicki L.
dc.contributor.authorHonig, Peter K.
dc.contributor.authorKaitin, Kenneth I.
dc.contributor.authorOye, Kenneth A
dc.contributor.authorD'Agostino, Ralph B.
dc.date.accessioned2020-11-20T22:55:48Z
dc.date.available2020-11-20T22:55:48Z
dc.date.issued2019-02
dc.date.submitted2018-10
dc.identifier.issn0009-9236
dc.identifier.issn1532-6535
dc.identifier.urihttps://hdl.handle.net/1721.1/128557
dc.description.abstractEfficacy 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.en_US
dc.description.sponsorshipNational Center for Advancing Translational Sciences (Grant U24TR001609)en_US
dc.language.isoen
dc.publisherWileyen_US
dc.relation.isversionofhttp://dx.doi.org/10.1002/cpt.1347en_US
dc.rightsCreative Commons Attribution NonCommercial License 4.0en_US
dc.rights.urihttps://creativecommons.org/licenses/by-nc/4.0/en_US
dc.sourceWileyen_US
dc.titleEfficacy and Effectiveness Too Trials: Clinical Trial Designs to Generate Evidence on Efficacy and on Effectiveness in Wide Practiceen_US
dc.typeArticleen_US
dc.identifier.citationSelker, 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 Authorsen_US
dc.contributor.departmentMassachusetts Institute of Technology. Department of Political Scienceen_US
dc.contributor.departmentMassachusetts Institute of Technology. Center for Biomedical Innovationen_US
dc.relation.journalClinical Pharmacology and Therapeuticsen_US
dc.eprint.versionFinal published versionen_US
dc.type.urihttp://purl.org/eprint/type/JournalArticleen_US
eprint.statushttp://purl.org/eprint/status/PeerRevieweden_US
dc.date.updated2020-06-12T15:47:55Z
dspace.date.submission2020-06-12T15:47:57Z
mit.journal.volume105en_US
mit.journal.issue4en_US
mit.licensePUBLISHER_CC


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