EFFICACY-TO-EFFECTIVENESS CLINICAL TRIALS

Efficacy trials, which assess treatments in optimally selected patients under advantageous conditions for relatively short time periods, are necessary to gain regulatory approval for marketing. In contrast, effectiveness trials, which test treatments across a spectrum of patients in real-world condi...

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Bibliographic Details
Published in:Transactions of the American Clinical and Climatological Association 2018, Vol.129, p.279-300
Main Authors: Selker, Harry P, Gorman, Sheeona, Kaitin, Kenneth I
Format: Article
Language:English
Subjects:
Acute Coronary Syndrome - blood
Acute Coronary Syndrome - diagnosis
Acute Coronary Syndrome - drug therapy
Administration, Intravenous
Clinical Trials as Topic - methods
Drug Administration Schedule
Drug Approval
Endpoint Determination
Glucose - administration & dosage
Humans
Insulin - administration & dosage
Potassium - administration & dosage
Research Design
Time Factors
Treatment Outcome
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Summary:Efficacy trials, which assess treatments in optimally selected patients under advantageous conditions for relatively short time periods, are necessary to gain regulatory approval for marketing. In contrast, effectiveness trials, which test treatments across a spectrum of patients in real-world conditions with follow-up periods that match typical treatment regimens, provide critical information on drug effects in those patients who may ultimately receive the treatment. We previously proposed a study design that integrates efficacy and effectiveness trials into a 2-component "efficacy-to-effectiveness (E2E) trial," in which if the initial efficacy trial component is positive, then the trial immediately and seamlessly transitions to the effectiveness trial component. However, we believe that total study duration could be even further shortened by simultaneously addressing efficacy and effectiveness too (EE2). An EE2 trial rigorously demonstrates efficacy, but uses broad inclusion characteristics of effectiveness trials. An example of a study using EE2 design, the IMMEDIATE (Immediate Myocardial Metabolic enhancement During Initial Assessment and Treatment in Emergency Care) trial, is provided.
ISSN:0065-7778