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 D...
| Main Authors: | , , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Wiley
2023-01-01
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| Series: | Alzheimer’s & Dementia: Translational Research & Clinical Interventions |
| Subjects: | |
| Online Access: | https://doi.org/10.1002/trc2.12375 |
| _version_ | 1797857615700557824 |
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| author | Marwan N. Sabbagh Angela Taylor Douglas Galasko James E. Galvin Jennifer G. Goldman James B. Leverenz Kathleen L. Poston Bradley F. Boeve David J. Irwin Joseph F. Quinn |
| author_facet | Marwan N. Sabbagh Angela Taylor Douglas Galasko James E. Galvin Jennifer G. Goldman James B. Leverenz Kathleen L. Poston Bradley F. Boeve David J. Irwin Joseph F. Quinn |
| author_sort | Marwan N. Sabbagh |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-04-09T20:59:35Z |
| format | Article |
| id | doaj.art-4e846c116fef4bab9db52b268fd87230 |
| institution | Directory Open Access Journal |
| issn | 2352-8737 |
| language | English |
| last_indexed | 2024-04-09T20:59:35Z |
| publishDate | 2023-01-01 |
| publisher | Wiley |
| record_format | Article |
| series | Alzheimer’s & Dementia: Translational Research & Clinical Interventions |
| spelling | doaj.art-4e846c116fef4bab9db52b268fd872302023-03-29T10:44:28ZengWileyAlzheimer’s & Dementia: Translational Research & Clinical Interventions2352-87372023-01-0191n/an/a10.1002/trc2.12375Listening session with the US Food and Drug Administration, Lewy Body Dementia Association, and an expert panelMarwan N. Sabbagh0Angela Taylor1Douglas Galasko2James E. Galvin3Jennifer G. Goldman4James B. Leverenz5Kathleen L. Poston6Bradley F. Boeve7David J. Irwin8Joseph F. Quinn9Department of Neurology Alzheimer's and Memory Disorders Division Barrow Neurological Institute St. Joseph's Hospital and Medical Center Phoenix Arizona USALewy Body Dementia Association Lilburn Georgia USAUniversity of California San Diego Shiley‐Marcos Alzheimer's Disease Research Center and Department of Neurosciences University of California San Diego La Jolla California USAComprehensive Center for Brain Health Department of Neurology University of Miami Miller School of Medicine Miami Florida USAParkinson's and Movement Disorders Shirley Ryan Ability Lab and Departments of Physical Medicine and Rehabilitation and Neurology Northwestern University Feinberg School of Medicine Chicago Illinois USACleveland Alzheimer's Disease Research Center and Cleveland Clinic Center for Brain Health Cleveland Ohio USADepartment of Neurology Stanford University Palo Alto California USAMayo Alzheimer's Disease Research Center and Department of Neurology Mayo Clinic Rochester Minnesota USADepartment of Neurology University of Pennsylvania Perelman School of Medicine Philadelphia Pennsylvania USADepartment of Neurology Oregon Health Sciences University Portland Oregon USAAbstract 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.https://doi.org/10.1002/trc2.12375clinical trialsdementia with Lewy bodiesdrug developmentoutcome measuresregulatory pathways |
| spellingShingle | Marwan N. Sabbagh Angela Taylor Douglas Galasko James E. Galvin Jennifer G. Goldman James B. Leverenz Kathleen L. Poston Bradley F. Boeve David J. Irwin Joseph F. Quinn Listening session with the US Food and Drug Administration, Lewy Body Dementia Association, and an expert panel Alzheimer’s & Dementia: Translational Research & Clinical Interventions clinical trials dementia with Lewy bodies drug development outcome measures regulatory pathways |
| title | Listening session with the US Food and Drug Administration, Lewy Body Dementia Association, and an expert panel |
| title_full | Listening session with the US Food and Drug Administration, Lewy Body Dementia Association, and an expert panel |
| title_fullStr | Listening session with the US Food and Drug Administration, Lewy Body Dementia Association, and an expert panel |
| title_full_unstemmed | Listening session with the US Food and Drug Administration, Lewy Body Dementia Association, and an expert panel |
| title_short | Listening session with the US Food and Drug Administration, Lewy Body Dementia Association, and an expert panel |
| title_sort | listening session with the us food and drug administration lewy body dementia association and an expert panel |
| topic | clinical trials dementia with Lewy bodies drug development outcome measures regulatory pathways |
| url | https://doi.org/10.1002/trc2.12375 |
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