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好问题?针对无症状晚发性阿尔茨海默病的疾病修饰疗法的政策和社会影响。

Good problems to have? Policy and societal implications of a disease-modifying therapy for presymptomatic late-onset Alzheimer's disease.

机构信息

Initiative for Science and Society and Social Science Research Institute, Duke University, Durham, North Carolina, 27708-0222, USA.

Duke University, Durham, USA.

出版信息

Life Sci Soc Policy. 2020 Oct 12;16(1):11. doi: 10.1186/s40504-020-00106-2.

Abstract

In the United States alone, the prevalence of AD is expected to more than double from six million people in 2019 to nearly 14 million people in 2050. Meanwhile, the track record for developing treatments for AD has been marked by decades of failure. But recent progress in genetics, neuroscience and gene editing suggest that effective treatments could be on the horizon. The arrival of such treatments would have profound implications for the way we diagnose, triage, study, and allocate resources to Alzheimer's patients. Because the disease is not rare and because it strikes late in life, the development of therapies that are expensive and efficacious but less than cures, will pose particular challenges to healthcare infrastructure. We have a window of time during which we can begin to anticipate just, equitable and salutary ways to accommodate a disease-modifying therapy Alzheimer's disease. Here we consider the implications for caregivers, clinicians, researchers, and the US healthcare system of the availability of an expensive, presymptomatic treatment for a common late-onset neurodegenerative disease for which diagnosis can be difficult.

摘要

仅在美国,预计到 2050 年,AD 的患病率将从 2019 年的 600 万人增加一倍以上,达到近 1400 万人。与此同时,开发 AD 治疗方法的记录却充满了几十年的失败。但遗传学、神经科学和基因编辑方面的最新进展表明,有效的治疗方法可能即将出现。这些治疗方法的出现将对我们诊断、分类、研究和分配资源给阿尔茨海默病患者的方式产生深远影响。由于这种疾病并不罕见,而且发生在生命晚期,因此开发昂贵、有效但不能治愈的疗法将对医疗保健基础设施构成特别挑战。我们有一段可以开始预测的时间窗口,以便为一种能够改变疾病进程的阿尔茨海默病疗法找到公正、公平和有益的方法。在这里,我们考虑了对于护理人员、临床医生、研究人员和美国医疗保健系统来说,一种昂贵的、有症状前的治疗方法对于一种常见的、发病较晚的神经退行性疾病的影响,这种疾病的诊断可能很困难。

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