Bridging the efficacy-effectiveness gap: a regulator's perspective on addressing variability of drug response
Bridging the efficacy-effectiveness gap: a regulator's perspective on addressing variability of drug response
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DOI：
10.1038/nrd3348
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发表时间：
2011-07-01
影响因子：
120.1
通讯作者：
Bloechl-Daum, Brigitte
中科院分区：
文献类型：
--
作者：
Eichler, Hans-Georg;Abadie, Eric;Bloechl-Daum, Brigitte
药品监管机构应确保药物的益处大于其风险，但在日常临床实践中，获得许可的药物有时无法达到预期效果。失败可能与低于预期的疗效或高于预期的不良反应发生率或严重程度有关。在这里，我们表明，获益-风险的问题在相当大的程度上是一个药物反应的变异性问题。我们描述了变异性的生物学和行为学来源，以及这些来源如何导致长期以来已知的疗效-有效性差距。在这种情况下，疗效描述了药物在临床试验条件下的表现，而有效性描述了它在日常临床实践条件下的表现。我们认为，需要利用广泛的许可前和许可后技术来弥合功效-效果差距。成功的方法将不仅限于目前基于药物基因组学的个性化药物概念，还需要更广泛地使用电子保健工具，以改善药物处方和患者依从性。
Drug regulatory agencies should ensure that the benefits of drugs outweigh their risks, but licensed medicines sometimes do not perform as expected in everyday clinical practice. Failure may relate to lower than anticipated efficacy or a higher than anticipated incidence or severity of adverse effects. Here we show that the problem of benefit-risk is to a considerable degree a problem of variability in drug response. We describe biological and behavioural sources of variability and how these contribute to the long-known efficacy-effectiveness gap. In this context, efficacy describes how a drug performs under conditions of clinical trials, whereas effectiveness describes how it performs under conditions of everyday clinical practice. We argue that a broad range of pre- and post-licensing technologies will need to be harnessed to bridge the efficacy-effectiveness gap. Successful approaches will not be limited to the current notion of pharmaco-genomics-based personalized medicines, but will also entail the wider use of electronic health-care tools to improve drug prescribing and patient adherence.