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The "Efficacy-Effectiveness Gap": Historical Background and Current Conceptualization

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  • Clementine Nordon
  • Helene Karcher
  • Rolf H H Groenwold
  • Mikkel Zöllner Ankarfeldt
  • Franz Pichler
  • Helene Chevrou-Severac
  • Michel Rossignol
  • Adeline Abbe
  • Lucien Abenhaim
  • GetReal consortium
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BACKGROUND: The concept of the "efficacy-effectiveness gap" (EEG) has started to challenge confidence in decisions made for drugs when based on randomized controlled trials alone. Launched by the Innovative Medicines Initiative, the GetReal project aims to improve understanding of how to reconcile evidence to support efficacy and effectiveness and at proposing operational solutions.

OBJECTIVES: The objectives of the present narrative review were 1) to understand the historical background in which the concept of the EEG has emerged and 2) to describe the conceptualization of EEG.

METHODS: A focused literature review was conducted across the gray literature and articles published in English reporting insights on the EEG concept. The identification of different "paradigms" was performed by simple inductive analysis of the documents' content.

RESULTS: The literature on the EEG falls into three major paradigms, in which EEG is related to 1) real-life characteristics of the health care system; 2) the method used to measure the drug's effect; and 3) a complex interaction between the drug's biological effect and contextual factors.

CONCLUSIONS: The third paradigm provides an opportunity to look beyond any dichotomy between "standardized" versus "real-life" characteristics of the health care system and study designs. Namely, future research will determine whether the identification of these contextual factors can help to best design randomized controlled trials that provide better estimates of drugs' effectiveness.

Original languageEnglish
JournalValue in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research
Volume19
Issue number1
Pages (from-to)75-81
Number of pages7
ISSN1098-3015
DOIs
Publication statusPublished - Jan 2016
Externally publishedYes

    Research areas

  • Clinical Trials as Topic, Drug Therapy, Health Knowledge, Attitudes, Practice, Humans, Product Surveillance, Postmarketing, Treatment Outcome, Journal Article, Research Support, Non-U.S. Gov't

ID: 49778694