@article{c7e00d0d66dd493fbc05a50fc341217c,
title = "Listening session with the US Food and Drug Administration, Lewy Body Dementia Association, and an expert panel",
abstract = "The regulatory path for drug approval is increasingly well defined. Drugs for the treatment of Alzheimer disease (AD) need to show statistically significant benefit over placebo with respect to cognitive and functional measures, with the Clinical Dementia Rating scale and Alzheimer's Disease Assessment Scale–Cognitive Subscale being among the most often used instruments in AD clinical trials. In contrast, there are no validated instruments for use in clinical trials of drugs for the treatment of dementia with Lewy bodies. This poses challenges for drug development because the regulatory pathway to drug approval requires demonstrable efficacy measures. In December 2021, the Lewy Body Dementia Association advisory group met with representatives from the US Food and Drug Administration to discuss the lack of approved drugs and treatments, discernment of efficacy measures, and identification of biomarkers. Highlights: The Lewy Body with Dementia Association convened a listening session with the US Food and Drug Administration on dementia with Lewy bodies (DLB) and clinical trial design. Gaps include DLB-specific measures, alpha synuclein biomarkers, and coexisting pathologies. DLB clinical trial design should focus on clinical value and disease specificity.",
keywords = "clinical trials, dementia with Lewy bodies, drug development, outcome measures, regulatory pathways",
author = "Sabbagh, {Marwan N.} and Angela Taylor and Douglas Galasko and Galvin, {James E.} and Goldman, {Jennifer G.} and Leverenz, {James B.} and Poston, {Kathleen L.} and Boeve, {Bradley F.} and Irwin, {David J.} and Quinn, {Joseph F.}",
note = "Funding Information: We thank the representatives from the US Food and Drug Administration who attended this meeting: Teresa Buracchio, MD; Michelle Campbell, PhD; Billy Dunn, MD; and Chris Melton; we also thank Debra Babcock, PhD, from the National Institutes of Neurological Disorders and Stroke and Akanni Clark, PhD, from the National Institute on Aging. We thank the staff of Neuroscience Publications at Barrow Neurological Institute for assistance with manuscript preparation. Supported by the Lewy Body Dementia Association. J.B.L. is funded by Aging Mind Foundation, GE Healthcare, Lewy Body Dementia Association, P30 AG072959, P20 AG068053, R01AG022304, R01 AG057552, R01 AG070736, R56 AG074001, RF1 AG071566, U01 AG073323, and U01 NS100610. J.E.G. is funded by R01 AG071514, R01 NS101483, R01 NS101483‐S1, P01 AG066584, R01 AG071643, R01 AG069765, R01 AG057681, and P30 AG059295. J.G.G. is funded by grants and support from the American Parkinson's Disease Association, Acadia, Lewy Body Dementia Association Research Center of Excellence, Michael J. Fox Foundation, Parkinson's Foundation, and Shirley Ryan AbilityLab. D.J.I. is funded by U19‐AG062418 and the Penn Institute on Aging. M.N.S. is supported by grants from the National Institutes of Health (NIH R01AG059008, NIH P30 AG072980, and NIH R01AG073212). Funding Information: We thank the representatives from the US Food and Drug Administration who attended this meeting: Teresa Buracchio, MD; Michelle Campbell, PhD; Billy Dunn, MD; and Chris Melton; we also thank Debra Babcock, PhD, from the National Institutes of Neurological Disorders and Stroke and Akanni Clark, PhD, from the National Institute on Aging. We thank the staff of Neuroscience Publications at Barrow Neurological Institute for assistance with manuscript preparation. Supported by the Lewy Body Dementia Association. J.B.L. is funded by Aging Mind Foundation, GE Healthcare, Lewy Body Dementia Association, P30 AG072959, P20 AG068053, R01AG022304, R01 AG057552, R01 AG070736, R56 AG074001, RF1 AG071566, U01 AG073323, and U01 NS100610. J.E.G. is funded by R01 AG071514, R01 NS101483, R01 NS101483-S1, P01 AG066584, R01 AG071643, R01 AG069765, R01 AG057681, and P30 AG059295. J.G.G. is funded by grants and support from the American Parkinson's Disease Association, Acadia, Lewy Body Dementia Association Research Center of Excellence, Michael J. Fox Foundation, Parkinson's Foundation, and Shirley Ryan AbilityLab. D.J.I. is funded by U19-AG062418 and the Penn Institute on Aging. M.N.S. is supported by grants from the National Institutes of Health (NIH R01AG059008, NIH P30 AG072980, and NIH R01AG073212). Publisher Copyright: {\textcopyright} 2023 The Authors. Alzheimer's & Dementia: Translational Research & Clinical Interventions published by Wiley Periodicals LLC on behalf of Alzheimer's Association.",
year = "2023",
month = jan,
day = "1",
doi = "10.1002/trc2.12375",
language = "English (US)",
volume = "9",
journal = "Alzheimer's and Dementia: Translational Research and Clinical Interventions",
issn = "2352-8737",
publisher = "Elsevier Inc.",
number = "1",
}