DEVELOPMENT OF IMPROVED EFFICACY ASSESSMENT MEASURES

制定改进的疗效评估措施

• 批准号: 6315218
• 负责人: STEVEN H FERRIS
• 金额: $ 22.84万
• 依托单位: UNIVERSITY OF CALIFORNIA, SAN DIEGO
• 依托单位国家: 美国
• 财政年份: 2000
• 资助国家: 美国
• 起止时间: 2000-07-01 至 2001-09-14
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6315218
• 关键词: Alzheimer's disease; behavior test; brain disorder chemotherapy; brain disorder diagnosis; clinical research; cognition; cooperative study; diagnosis design /evaluation; functional ability; health care cost /financing; human old age (65+); human subject; human therapy evaluation; neuropsychological tests; psychopharmacology; quality of life

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.

本提案的目标是合作开发新的或 改进临床评估方法和工具 AD 临床试验的改进。 评估的两个核心问题 治疗效果是指药物是否有治疗作用，如果 因此，观察到的效果是否具有临床意义且足够 规模超过可能的风险。 获得准确答案取决于 使用精心设计的协议，采用适当的、灵敏的、可靠的 和临床相关的结果测量。 合理充分的措施是 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）、全球范围、日常生活活动（ADL）范围，以及 非认知行为症状量表。 然而，目前各州采取的措施 这些域有很大的局限性。 两项附加评估 对于扩大患者获得临床治疗的机会而言，这些领域变得越来越重要 试验：针对严重受损患者的测量，以及非英式量表。 之前在这两个领域所做的工作相对较少。 这 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 各单位（ADSU）在这些方面制定改进的或新的成果衡量标准 六个评估领域。 为了实现这一目标，我们建议（1）组建 每个评估领域的专家小组委员会将 构思、制定和规划每个领域新措施的评估 领域。 联合体评估委员会，由主席和成员组成 每个评估小组委员会的主席将规划和协调 制定新措施的总体方案； (2) 使用人员和 30 个参与 ADSU 的患者群体来评估效用， 开发的新量表和测试的可靠性和有效性， (3) 现场测试新措施的实用性和药物敏感性 通过将它们作为多中心的次要结果指标来制定 ADSU 进行的临床试验。