Changyu Shen, Robert W. Platt, Shibeshih Belachew, Hiroko H. Dodge
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引用次数: 0
Abstract
Recent accelerated and traditional approval of anti-amyloid therapies by the U.S. Food and Drug Administration for the treatment of patients with early Alzheimer's disease has stimulated heated debate on whether or not the benefits of these therapeutic agents achieve a minimum clinically important effect size, or minimum clinically important difference. We argue that these debates are rooted in the entanglement of two fundamentally different concepts, the minimum clinically important difference for an individual versus that of a population. At the core of the indiscrimination between the two concepts is the unrealistic requirement or expectation that a drug should provide the same clinically important effect for every patient in the target population to be considered achieving meaningful benefit for the population. We discuss the difference and connection between the two concepts to facilitate the communication about their difference and relatedness.
Highlights
Minimum clinically important difference (MCID) is defined for an individual and population-level mimimum clinically important difference (pMCID) is defined for a population.
MCID and pMCID are fundamentally different measures.
We established their connection and showed in general pMCID < MCID.
Discussion of effect size of Alzheimer's disease treatments should clearly distinguish the two measures.
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.
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