Efficacy and Effectiveness Too Trials: Clinical Trial Designs to Generate Evidence on Efficacy and on Effectiveness in Wide Practice
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‐worl...
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| Veröffentlicht in: | Clinical pharmacology and therapeutics 2019-04, Vol.105 (4), p.857-866 |
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| container_title | Clinical pharmacology and therapeutics |
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| creator | 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. |
| description | 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. |
| doi_str_mv | 10.1002/cpt.1347 |
| format | Article |
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| source | MEDLINE; Wiley Online Library Journals Frontfile Complete |
| subjects | Clinical Trials as Topic - legislation & jurisprudence Cost-Benefit Analysis - legislation & jurisprudence Drug Approval - legislation & jurisprudence Drug Development - legislation & jurisprudence Humans Marketing - legislation & jurisprudence Patient Selection Research Design - legislation & jurisprudence Reviews State of the Art Treatment Outcome |
| title | Efficacy and Effectiveness Too Trials: Clinical Trial Designs to Generate Evidence on Efficacy and on Effectiveness in Wide Practice |
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