RTI International, Research Triangle Park, North Carolina.
Ann N Y Acad Sci. 2014 Apr;1313(1):17-34. doi: 10.1111/nyas.12417. Epub 2014 Mar 27.
The development of disease-modifying treatments for Alzheimer's disease (AD) faces a number of barriers. Among these are the lack of surrogate biomarkers, the exceptional size and duration of clinical trials, difficulties in identifying appropriate populations for clinical trials, and the limitations of monotherapies in addressing such a complex multifactorial disease. This study sets out to first estimate the consequent impact on the expected cost of developing disease-modifying treatments for AD and then to estimate the potential benefits of bringing together industry, academic, and government stakeholders to co-invest in, for example, developing better biomarkers and cognitive assessment tools, building out advanced registries and clinical trial-readiness cohorts, and establishing clinical trial platforms to investigate combinations of candidate drugs and biomarkers from the portfolios of multiple companies. Estimates based on interviews with experts on AD research and development suggest that the cost of one new drug is now $5.7 billion (95% confidence interval (CI) $3.7-9.5 billion) and could be reduced to $2.0 billion (95% CI $1.5-2.9 billion). The associated acceleration in the arrival of disease-modifying treatments could reduce the number of case years of dementia by 7.0 million (95% CI 4.4-9.4 million) in the United States from 2025 through 2040.
阿尔茨海默病（AD）的疾病修饰治疗的发展面临着许多障碍。其中包括缺乏替代生物标志物、临床试验规模和持续时间异常长、难以确定临床试验的合适人群以及单一疗法在解决如此复杂的多因素疾病方面的局限性。本研究旨在首先估计这对开发 AD 疾病修饰治疗的预期成本的影响，然后估计将行业、学术和政府利益相关者聚集在一起共同投资的潜在好处，例如开发更好的生物标志物和认知评估工具、建立先进的登记处和临床试验准备队列，以及建立临床试验平台，以研究多家公司投资组合中的候选药物和生物标志物的组合。基于对 AD 研发专家的访谈进行的估计表明，现在开发一种新药的成本为 57 亿美元（95%置信区间（CI）为 370 亿至 950 亿美元），并且可以降低到 20 亿美元（95%CI 为 150 亿至 290 亿美元）。疾病修饰治疗的加速到来可能会减少美国从 2025 年到 2040 年因痴呆症而导致的病例年数 700 万例（95%CI 为 440 万至 940 万例）。
