Research Center and Memory Clinic, Fundació ACE, Institut Català de Neurociències Aplicades, Universitat Internacional de Catalunya, Barcelona, Spain.
Cognition and Brain Plasticity Group, Bellvitge Biomedical Research Institute, IDIBELL, Spain.
J Alzheimers Dis. 2018;62(3):1079-1090. doi: 10.3233/JAD-170866.
Alzheimer's disease (AD) research is at a critical time. The global society is increasingly aware of the frightening rate of growth of the human and financial burden caused by this condition and of the urgent need to halt its progression. Consequently, the scientific community holds great responsibility to quickly put in place and optimize the machinery necessary for testing new treatments or interventions. In this context demand for participants for AD research is at an all-time high. In this review, we will focus on a methodological factor that is increasingly recognized as a key factor that shapes trial populations and affects validity of results in clinical trials: patient engagement, recruitment, and retention. We outline specific problems relevant to patient engagement in AD including recruiting enough participants, difficulties in participant retention, ensuring the recruited sample is representative of the general AD population, the burden of screening failures, and new challenges related to recruiting in preclinical disease. To address the urgent need for more research studying the applicability and cost-effectiveness of different recruitment strategies across different settings and nationalities, we describe the Models of Patient Engagement for Alzheimer's Disease (MOPEAD) project, a public-private partnership promoted by the Innovative Medicine Initiative (IMI), which will provide a large multinational quantitative analysis comparing different innovative recruitment models. We also discuss strategies that address each problem and draw on the experience of Fundació ACE to argue that focusing resources on comprehensive AD centers that offer coordinated clinical and social care and participate in basic and clinical research, is an effective and efficient way of implementing many of the discussed strategies.
阿尔茨海默病（AD）研究正处于关键时刻。全球社会越来越意识到这种疾病导致的人力和财力负担的增长速度令人担忧，急需阻止其发展。因此，科学界有责任迅速建立和优化测试新治疗方法或干预措施所需的机制。在这种情况下，对 AD 研究参与者的需求达到了历史最高水平。在这篇综述中，我们将重点关注一个方法学因素，该因素越来越被认为是塑造试验人群并影响临床试验结果有效性的关键因素：患者参与、招募和保留。我们概述了与 AD 患者参与相关的具体问题，包括招募足够的参与者、参与者保留的困难、确保招募的样本代表一般 AD 人群、筛选失败的负担，以及与临床前疾病招募相关的新挑战。为了解决在不同环境和国籍下研究不同招募策略的适用性和成本效益的迫切需求，我们描述了阿尔茨海默病患者参与模型（MOPEAD）项目，这是一个由创新药物倡议（IMI）推动的公私合作伙伴关系，该项目将提供一个大型跨国定量分析，比较不同的创新招募模型。我们还讨论了解决每个问题的策略，并借鉴 ACE 基金会的经验，认为将资源集中在提供协调的临床和社会护理并参与基础和临床研究的综合性 AD 中心，是实施所讨论的许多策略的有效和高效方式。
