Advancing outcome measures for the new era of drug development in cystic fibrosis.
Advancing outcome measures for the new era of drug development in cystic fibrosis.
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DOI：
10.1513/pats.200703-040br
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发表时间：
2007-08-01
期刊：
影响因子：
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通讯作者：
Kronmal, Richard A
中科院分区：
文献类型：
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作者：
Mayer-Hamblett, Nicole;Ramsey, Bonnie W;Kronmal, Richard A
越来越多的囊性纤维化（CF）候选药物正在挑战临床试验研究。从评估旨在治疗CF继发表现的药物到评估针对慢性肺病一级预防的药物已经发生了转变。由于CF是一种孤儿病，因此根本需要通过最佳利用可用患者数量的机制有效和准确地评估新疗法。这一需求可以通过不断改进和完善CF结果措施来解决。我们开始概述CF临床研究中目前使用的结局指标，根据三个主要终点类别之一进行定义和分类：临床疗效指标、替代终点和生物标志物。为了有效地向前推进，CF的临床研究取决于能够捕获新治疗方法的生物学和临床反应的新结果的开发。最后，我们讨论的标准，所有新的结果的措施应进行评估。一个系统的，严格的方法来衡量结果的发展，需要提供必要的工具，评估新的疗法和移动药物的管道，并进入CF诊所。
The growing pipeline of candidate drugs for cystic fibrosis (CF) is challenging clinical trial research. There has been a shift from evaluating drugs aimed at treating the secondary manifestations of CF to evaluating drugs targeted toward the primary prevention of chronic lung disease. As CF is an orphan disease, there is a fundamental need to assess new therapies efficiently and accurately by mechanisms that best use the number of available patients. This need can be addressed with the continued advancement and refinement of CF outcome measures. We begin by presenting an overview of the outcome measures currently used in CF clinical studies, defined and categorized in terms of one of the three main classes of endpoints: clinical efficacy measures, surrogate endpoints, and biomarkers. To move forward efficiently, clinical research in CF is dependent on the development of new outcomes able to capture biologic and clinical response to novel therapeutic approaches. We conclude with a discussion of the criteria by which all new outcome measures should be evaluated. A systematic, rigorous approach to outcome measure development is needed to provide the tools necessary for evaluating new therapies and moving drugs out of the pipeline and into the CF clinic.