Nordon Clementine, Karcher Helene, Groenwold Rolf H H, Ankarfeldt Mikkel Zöllner, Pichler Franz, Chevrou-Severac Helene, Rossignol Michel, Abbe Adeline, Abenhaim Lucien
LASER Research, Paris, France.
LASER Analytica, London, UK.
Value Health. 2016 Jan;19(1):75-81. doi: 10.1016/j.jval.2015.09.2938. Epub 2015 Nov 19.
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.
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.
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.
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.
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.
“疗效-效果差距”（EEG）的概念已开始挑战仅基于随机对照试验做出的药物决策的可信度。由创新药物倡议发起的“GetReal”项目旨在增进对如何协调证据以支持疗效和效果的理解，并提出实际解决方案。
本叙述性综述的目的是：1）了解EEG概念出现的历史背景；2）描述EEG的概念化。
对灰色文献以及以英文发表的报道EEG概念见解的文章进行了重点文献综述。通过对文献内容进行简单归纳分析来确定不同的“范式”。
关于EEG的文献可分为三大范式，其中EEG与以下方面相关：1）医疗保健系统的现实特征；2）用于衡量药物效果的方法；3）药物的生物学效应与背景因素之间的复杂相互作用。
第三种范式提供了一个机会，超越医疗保健系统的“标准化”与“现实生活”特征以及研究设计之间的任何二分法去看待问题。也就是说，未来的研究将确定这些背景因素的识别是否有助于最佳设计随机对照试验，从而更好地评估药物的效果。
