Theranostics by testing CFTR modulators in patient-derived materials: The current status and a proposal for subjects with rare CFTR mutations
Theranostics by testing CFTR modulators in patient-derived materials: The current status and a proposal for subjects with rare CFTR mutations
复制标题
DOI：
10.1016/j.jcf.2019.06.010
复制
发表时间：
2019-09-01
影响因子：
5.2
通讯作者：
Lee, Tim
中科院分区：
文献类型：
--
作者：
Amaral, Margarida;Davies, Jane C.;Lee, Tim
过去十年见证了CF药物管道的发展，这些发展既令人兴奋又史无前例，带来了以前从未考虑过的挑战。工作队小组齐聚一堂，审议这些挑战和应对这些挑战的可能战略。在过去的18个月里，我们进行了内部讨论，并从代表患者组织、临床和研究团队、制药业和监管机构的广泛个人那里收集了意见。在这篇文章和随附的文章中，我们讨论了两个主要的重点领域：i)优化试验设计和交付，以实现速度/效率；ii)针对罕见CFTR突变患者的药物开发。我们提出了一些应对未来挑战的战略，并强调了需要进一步思考的领域。我们认为这是一个过程的开始，而不是结束，并希望在此让更广泛的社区参与到为所有CF患者寻求改善治疗的解决方案中。(C)2019年，由Elsevier B.V.代表欧洲囊性纤维化学会出版。
The last decade has witnessed developments in the CF drug pipeline which are both exciting and unprecedented, bringing with them previously unconsidered challenges. The Task Force group came together to consider these challenges and possible strategies to address them. Over the last 18 months, we have discussed internally and gathered views from a broad range of individuals representing patient organizations, clinical and research teams, the pharmaceutical industry and regulatory agencies. In this and the accompanying article, we discuss two main areas of focus: i) optimising trial design and delivery for speed/efficiency; ii) drug development for patients with rare CFTR mutations. We propose some strategies to tackle the challenges ahead and highlight areas where further thought is needed. We see this as the start of a process rather than the end and hope herewith to engage the wider community in seeking solutions to improved treatments for all patients with CF. (C) 2019 Published by Elsevier B.V. on behalf of European Cystic Fibrosis Society.