Clinically meaningful outcomes in Alzheimer's disease and Alzheimer's disease related dementias trials

IF 4.9 Q1 CLINICAL NEUROLOGY

Alzheimer''s and Dementia: Translational Research and Clinical Interventions Pub Date : 2025-02-19 DOI:10.1002/trc2.70058

Luke E. Stoeckel, Elena M. Fazio, Kristina K. Hardy, Nicole Kidwiler, Kristina A. McLinden, Benfeard Williams

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引用次数: 0

Abstract

On March 12–14, 2024, the National Institute on Aging (NIA) together with the National Institute of Neurological Disorders and Stroke (NINDS) led a workshop exploring clinically meaningful changes in the context of Alzheimer's Disease and Alzheimer's Disease Related Dementias (AD/ADRD) clinical trials (https://www.nia.nih.gov/research/dbsr/workshops/clinically-meaningful-outcomes-ad-adrd-trials). The goals were to identify research gaps, opportunities, and tools to advance patient-centered, equitable assessment of clinically meaningful change focused on biomarker status, cognition, and everyday function. The workshop fostered robust, multidisciplinary discussion between lived experience experts, advocates, researchers, clinicians, funders, payers, and regulators. The workshop addressed the criteria used to assess whether an intervention has had a clinically meaningful impact, including consideration of both benefit and harm. Here, we report on (1) criteria to consider for development, testing, and selection of clinically meaningful outcomes in AD/ADRD clinical trials; (2) methods to validate and customize clinically meaningful outcomes that are fit-for-purpose; and (3) practices that will ensure that clinically meaningful outcomes are applicable to diverse populations.

Highlights

Patient-Centered Outcomes: Inclusive AD/ADRD clinical trials incorporate assessments that reflect what matters most to those impacted by these diseases, including patient- and caregiver-reported outcome measures.
Culturally Relevant Assessments: There is a need for culturally sensitive and equitable assessments to better serve diverse populations in AD/ADRD research.
Framework for Clinically Meaningful Change: We present a framework for defining and evaluating clinically meaningful outcomes in AD/ADRD trials, tailored to diverse stages of disease progression.
Interdisciplinary Approach: We draw on insights from a multidisciplinary workshop, fostering collaboration among researchers, clinicians, and lived experience experts to advance the field.

查看原文本刊更多论文

阿尔茨海默病和阿尔茨海默病相关痴呆试验的临床有意义的结果

2024年3月12日至14日，美国国家衰老研究所（NIA）与美国国家神经疾病和中风研究所（NINDS）共同举办了一场研讨会，探讨阿尔茨海默病和阿尔茨海默病相关痴呆（AD/ADRD）临床试验背景下的临床意义变化（https://www.nia.nih.gov/research/dbsr/workshops/clinically-meaningful-outcomes-ad-adrd-trials）。目标是确定研究差距、机会和工具，以推进以患者为中心、公平评估临床有意义的变化，重点是生物标志物状态、认知和日常功能。研讨会促进了生活经验专家、倡导者、研究人员、临床医生、资助者、支付方和监管机构之间强有力的多学科讨论。研讨会讨论了用于评估干预措施是否具有临床意义影响的标准，包括对益处和危害的考虑。在这里，我们报告了(1)在AD/ADRD临床试验中考虑开发、测试和选择临床有意义结果的标准；(2)验证和定制符合目的的有临床意义的结果的方法；(3)确保临床有意义的结果适用于不同人群的实践。强调以患者为中心的结果：包容性AD/ADRD临床试验纳入了对受这些疾病影响的患者最重要的评估，包括患者和护理人员报告的结果测量。文化相关评估：需要文化敏感和公平的评估，以便在AD/ADRD研究中更好地为不同人群服务。临床意义改变框架：我们提出了一个定义和评估AD/ADRD试验中临床意义结果的框架，该框架针对疾病进展的不同阶段量身定制。跨学科方法：我们借鉴多学科研讨会的见解，促进研究人员、临床医生和生活经验专家之间的合作，以推进该领域的发展。

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来源期刊

Alzheimer''s and Dementia: Translational Research and Clinical Interventions Medicine-Psychiatry and Mental Health

CiteScore

10.10

自引率

2.10%

发文量

134

审稿时长

10 weeks

期刊介绍： Alzheimer''s & Dementia: Translational Research & Clinical Interventions (TRCI) is a peer-reviewed, open access,journal from the Alzheimer''s Association®. The journal seeks to bridge the full scope of explorations between basic research on drug discovery and clinical studies, validating putative therapies for aging-related chronic brain conditions that affect cognition, motor functions, and other behavioral or clinical symptoms associated with all forms dementia and Alzheimer''s disease. The journal will publish findings from diverse domains of research and disciplines to accelerate the conversion of abstract facts into practical knowledge: specifically, to translate what is learned at the bench into bedside applications. The journal seeks to publish articles that go beyond a singular emphasis on either basic drug discovery research or clinical research. Rather, an important theme of articles will be the linkages between and among the various discrete steps in the complex continuum of therapy development. For rapid communication among a multidisciplinary research audience involving the range of therapeutic interventions, TRCI will consider only original contributions that include feature length research articles, systematic reviews, meta-analyses, brief reports, narrative reviews, commentaries, letters, perspectives, and research news that would advance wide range of interventions to ameliorate symptoms or alter the progression of chronic neurocognitive disorders such as dementia and Alzheimer''s disease. The journal will publish on topics related to medicine, geriatrics, neuroscience, neurophysiology, neurology, psychiatry, clinical psychology, bioinformatics, pharmaco-genetics, regulatory issues, health economics, pharmacoeconomics, and public health policy as these apply to preclinical and clinical research on therapeutics.