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临床试验的苦与乐:囊性纤维化药物研究的起起落落。

Trials and tribulations: The highs and lows of running cystic fibrosis drug studies.

机构信息

Imperial College London and Royal Brompton & Harefield NHS Foundation Trust, London, UK.

出版信息

Paediatr Respir Rev. 2019 Aug;31:25-27. doi: 10.1016/j.prrv.2019.03.002. Epub 2019 Mar 14.

Abstract

So many new trials of drugs for CF are being conducted each year it can be difficult to keep up. Yet, almost as numerous are the review articles written about them. I have chosen therefore to use this paper to discuss some of the difficult ethical issues arising during trials, in particular how to fairly distribute opportunities to participate. Some of these issues may be specific to countries like my own with very limited access to genotype-specific CFTR modulators, others may apply more generally. We have made enormous progress as a community over the recent past, but there is still much more to do before all our patients are reaping health and quality of life benefits.

摘要

每年都有许多新的 CF 药物试验在进行,这使得人们很难跟上。然而,相关的综述文章也几乎同样多。因此,我选择用这篇论文来讨论在试验中出现的一些困难的伦理问题,特别是如何公平地分配参与机会。其中一些问题可能特定于像我所在的国家,那里获得特定基因型 CFTR 调节剂的机会非常有限,其他问题可能更具有普遍性。在最近的过去,我们作为一个社区取得了巨大的进步,但在我们所有的患者都能从中获得健康和生活质量的好处之前,还有很多工作要做。

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