A new era in idiopathic pulmonary fibrosis: considerations for future clinical trials
A new era in idiopathic pulmonary fibrosis: considerations for future clinical trials
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DOI：
10.1183/09031936.00200614
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发表时间：
2015-07-01
影响因子：
24.3
通讯作者：
Brown, Kevin K.
中科院分区：
文献类型：
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作者：
Collard, Harold R.;Bradford, Williamson Z.;Brown, Kevin K.
过去十年，人们对特发性肺纤维化（IPF）的病理学、自然史和临床意义的理解取得了实质性进展，最终建立了两种有效的药物疗法。现在似乎是重新考虑未来 IPF 临床试验的设计和实施的重要时机。根据过去十年的经验教训，我们从这个角度提出了四个关键考虑因素，以有效和高效地推进下一代 IPF 临床试验。这些是： 开发协调的 IPF 临床试验网络；建立对早期阶段概念验证研究的期望；调整后期疗效试验设计以适应已批准疗法的出现；以及；就后期临床试验的主要终点达成一致。 IPF 领域的持续进步需要所有利益相关者的创造力和协作。我们相信，解决这四个问题将鼓励并促进对 IPF 药物开发新时代的投资，并将导致有效疗法的更快开发。
The past decade has seen substantial progress in understanding the pathobiology, natural history, and clinical significance of idiopathic pulmonary fibrosis (IPF), culminating in the establishment of two effective medical therapies. Now seems an important time to reconsider the design and conduct of future IPF clinical trials. Building on lessons learned over the past decade, we use this perspective to lay out four key considerations for moving forward effectively and efficiently with the next generation of clinical trials in IPF. These are: development of a coordinated IPF clinical trials network; establishment of expectations for early phase proof of concept studies; adaptation of late-phase efficacy trial designs to the emergence of approved therapies, and; agreement on primary end-points for late phase clinical trials. Continued progress in the field of IPF will require creativity and collaboration on the part of all stakeholders. We believe that addressing these four considerations will encourage and enable investment in this new era of drug development in IPF, and will lead to more rapid development of effective therapies.