怎样才能使疾病取得进展?

Michael S Ringel, Julie Dethier, Michelle J. Davitt, Maria Denslow, R. Andrew Fowler, Sebastian C. Hasenfuss, Ulrik Schulze
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摘要

在本文中,我们采用病例对照设计来研究在防治疾病方面取得进展需要具备哪些条件:我们将病例(有成功疗法的疾病)与对照组(没有成功疗法的疾病)进行比较。我们发现,要想取得成功,通常必须具备五个条件("障碍"):(A) 了解生物驱动因素;(B) 调节生物学的能力;(C) 转化模型的可用性;(D1) 识别患者的能力;(D2) 测量临床反应的能力。该框架与评估单个候选药物的框架类似,但在此用于对整个疾病进行推断。它可用于确定哪些疾病最有可能取得进展,哪些疾病应集中力量在目前难以解决的疾病上取得进展,以及哪些疾病可从开发工具中获益,以解决各种疾病中最常见的最后一个障碍--即(C)转化模型。
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What does it take to make progress in a disease?
In this paper, we investigate what conditions need to be in place to make progress in combating a disease using a case-control design: we compare cases (diseases with a successful therapy) to controls (diseases without a successful therapy). We find five conditions ('hurdles') must typically be cleared for success: (A) understanding of biological drivers, (B) ability to modulate biology, (C) availability of translational models, (D1) ability to identify patients, and (D2) ability to measure clinical response. This framework is similar to ones deployed to evaluate individual drug candidates but is employed here to make inferences about entire diseases. It can be used to identify diseases most ready for progress, where efforts should be focused to make progress in diseases that are currently intractable, and where the industry could benefit from development of tools to address the hurdle that is most commonly the last to be cleared across diseases-namely, (C) translational models.
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