DEVELOPMENT OF IMPROVED EFFICACY ASSESSMENT MEASURES

本提案的目标是合作开发新的或 改进的评估方法和工具，用于评估临床 改善AD临床试验。 评估中的两个核心问题 治疗效果是药物是否具有治疗效果，如果 因此，观察到的效果是否具有临床意义， 风险大于可能的风险。 获得准确的答案取决于 使用设计良好的协议，这些协议采用适当的、敏感的、可靠的 和临床相关的结局指标。 合理的适当措施是 目前可用于四个主要AD评估类别： 认知功能测量（综合量表和心理测量 电池）、全球量表、日常生活活动（ADL）量表，以及 非认知行为症状量表。 然而，目前各国的措施 这些领域都有很大的局限性。 两项额外评估 这些领域对于拓宽患者获得临床治疗的途径越来越重要。 试验：严重受损患者的测量和非英语量表。 以前在这两个领域所做的工作相对较少。 的 该提案的总体目标是利用独特的专业知识， 阿尔茨海默病研究的经验、患者资源和工作人员 单位（ADSU），以制定改善或新的结果措施，在每一个这些 六个评估领域。 为了实现这一目标，我们建议（1）建立 每个评估领域的专家小组委员会， 概念化，发展，并计划在每一个新的措施的评价 域 联合体评估委员会，由主席和 每个评估小组委员会的主席，将计划和协调 制定新措施的总体方案;（2）利用员工， 30个参与ADSU的患者人群，以评估效用， 所开发的新量表和测试的可靠性和有效性， 及（3）实地测试新措施的效用及药物敏感性 通过将其作为多中心研究的次要结局指标进行开发， 由ADSU进行的临床试验。

The objective of this proposal is to develop, cooperatively, new or improved assessment methodologies and instruments for evaluating clinical improvement in AD clinical trials. The two central questions in evaluating treatment efficacy are whether the drug has a therapeutic effect, and if so, whether the effect observed is clinically meaningful and of sufficient magnitude to outweigh possible risks. Obtaining accurate answers hinges on using well-designed protocols which employ appropriate, sensitive, reliable and clinically relevant outcome measures. Reasonably adequate measures are currently available for the four primary AD assessment categories: Measures of cognitive function (comprehensive scales and psychometric batteries), global scales, activities of daily living (ADL) scales, and non-cognitive behavioral symptom scales. However, current measures in each of these domains have significant limitations. Two additional assessment domains are of growing importance for widening patient access to clinical trials: measures for severly impaired patients, and non-English scales. Relatively little previous work has been done in these two areas. The general goal of this proposal is to capitalize on the unique expertise, experience, patient resources and staff of the Alzheimer's Disease Study Units (ADSUs) to develop improved or new outcome measures in each of these six assessment domains. To achieve this goal, we propose (1) to form subcommittees of experts for each assessment domain which will conceptualize, develop, and plan the evaluation of new measures in each domain. A Consortium Assessment Committee, consisting of a chair and the chairs of each assessment subcommittee, will plan and coordinate the overall program to develop new measures; (2) to utilize the staff and patient populations of the 30 participating ADSUs to assess the utility, reliability and validity of the new scales and tests that are developed, and (3) to field-test the utility and drug sensitivity of the new measures developed by including them as secondary outcome measures in multicenter clinical trials conducted by the ADSUs.