Cummings J, Aisen P, Barton R, Bork J, Doody R, Dwyer J, Egan J C, Feldman H, Lappin D, Truyen L, Salloway S, Sperling R, Vradenburg G
Cleveland Clinic Lou Ruvo Center for Brain Health, Las Vegas, NV, USA.
University of Southern California, Los Angeles, CA, USA.
J Prev Alzheimers Dis. 2016 Jun;3(2):114-120. doi: 10.14283/jpad.2016.93. Epub 2016 Mar 4.
Alzheimer's disease (AD) drug development is costly, time-consuming, and inefficient. Trial site functions, trial design, and patient recruitment for trials all require improvement. The Global Alzheimer Platform (GAP) was initiated in response to these challenges. Four GAP work streams evolved in the US to address different trial challenges: 1) registry-to-cohort web-based recruitment; 2) clinical trial site activation and site network construction (GAP-NET); 3) adaptive proof-of-concept clinical trial design; and 4) finance and fund raising. GAP-NET proposes to establish a standardized network of continuously funded trial sites that are highly qualified to perform trials (with established clinical, biomarker, imaging capability; certified raters; sophisticated management system. GAP-NET will conduct trials for academic and biopharma industry partners using standardized instrument versions and administration. Collaboration with the Innovative Medicines Initiative (IMI) European Prevention of Alzheimer's Disease (EPAD) program, the Canadian Consortium on Neurodegeneration in Aging (CCNA) and other similar international initiatives will allow conduct of global trials. GAP-NET aims to increase trial efficiency and quality, decrease trial redundancy, accelerate cohort development and trial recruitment, and decrease trial costs. The value proposition for sites includes stable funding and uniform training and trial execution; the value to trial sponsors is decreased trial costs, reduced time to execute trials, and enhanced data quality. The value for patients and society is the more rapid availability of new treatments for AD.
阿尔茨海默病（AD）药物研发成本高昂、耗时且效率低下。试验场地功能、试验设计以及试验的患者招募都有待改进。全球阿尔茨海默病平台（GAP）正是为应对这些挑战而发起的。在美国，GAP形成了四个工作流程以应对不同的试验挑战：1）基于网络的从登记库到队列的招募；2）临床试验场地激活和场地网络建设（GAP-NET）；3）适应性概念验证临床试验设计；4）财务与筹资。GAP-NET提议建立一个由持续获得资金支持的试验场地组成的标准化网络，这些场地具备进行试验的高度资质（拥有成熟的临床、生物标志物、成像能力；有资质认证的评估人员；完善的管理系统）。GAP-NET将使用标准化的仪器版本和管理方式为学术和生物制药行业合作伙伴开展试验。与创新药物倡议组织（IMI）的欧洲预防阿尔茨海默病（EPAD）项目、加拿大衰老神经退行性变联盟（CCNA）以及其他类似的国际倡议开展合作，将能够进行全球试验。GAP-NET旨在提高试验效率和质量，减少试验冗余，加速队列发展和试验招募，并降低试验成本。对试验场地而言，价值主张包括稳定的资金支持以及统一的培训和试验执行；对试验申办者而言，价值在于降低试验成本、缩短试验执行时间以及提高数据质量。对患者和社会而言，价值在于能更快获得治疗AD的新疗法。
