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. 2021 Jun 26;7(1):e12181.
doi: 10.1002/trc2.12181. eCollection 2021.

Building clinically relevant outcomes across the Alzheimer's disease spectrum

Dorene M Rentz  1   2 , Alette M Wessels  3 , Ananth V Annapragada  4 , Anna-Karin Berger  5 , Chris J Edgar  6 , Michael Gold  7 , David S Miller  8 , Christopher Randolph  9   10 , J Michael Ryan  11 , Glen Wunderlich  12 , Megan Canniere Zoschg  13 , Dominic Trépel  14   15 , David S Knopman  16 , Adam M Staffaroni  17 , Lisa J Bain  18 , Maria C Carrillo  19 , Christopher J Weber  19
Affiliations

Building clinically relevant outcomes across the Alzheimer's disease spectrum

Dorene M Rentz et al. Alzheimers Dement (N Y). .

Abstract

Demonstrating that treatments are clinically meaningful across the Alzheimer's disease (AD) continuum is critical for meeting our goals of accelerating a cure by 2025. While this topic has been a focus of several Alzheimer's Association Research Roundtable (AARR) meetings, there remains no consensus as to what constitutes a "clinically meaningful outcome" in the eyes of patients, clinicians, care partners, policymakers, payers, and regulatory bodies. Furthermore, the field has not come to agreement as to what constitutes a clinically meaningful treatment effect at each stage of disease severity. The AARR meeting on November 19-20, 2019, reviewed current approaches to defining clinical meaningfulness from various perspectives including those of patients and care partners, clinicians, regulators, health economists, and public policymakers. Participants discussed approaches that may confer clinical relevance at each stage of the disease continuum and fostered discussion about what should guide us in the future.

Keywords: Alzheimer's disease; Research Roundtable; clinical meaningfulness; cognitive decline; dementia; treatment.

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Conflict of interest statement

AW is a full‐time employee and shareholder of Eli Lilly and Company. AA declares stock ownership and consulting fees from Alzeca Inc, stock ownership in Sensulin LLC. CJE is a full‐time employee of Cogstate Ltd. MG is a full‐time employee of Abbvie, Inc. DSM is a full‐time employee of Signant Health. CR is a full‐time employee of MedAvante‐ProPhase. JMR, previously with Rodin Therapeutics, at time of publication is a full‐time employee of Carnot Pharma, LLC dba RA Ventures. GW is a full‐time employee of Boehringer Ingelheim. DSK served on a Data Safety Monitoring Board for the DIAN study. He serves on a Data Safety monitoring Board for a tau therapeutic for Biogen, but receives no personal compensation. He is an investigator in clinical trials sponsored by Biogen, Lilly Pharmaceuticals, and the University of Southern California. He serves as a consultant for Samus Therapeutics, Third Rock, Roche, and Alzeca Biosciences but receives no personal compensation. He receives research support from the NIH. AMS receives support from the NIH/NIA, the Larry L. Hillblom Foundation, and the Bluefield Project to Cure FTD. MCC, CW are full‐time employees of the Alzheimer's Association.

References

    1. Jack CR Jr, Bennett DA, Blennow K, Research Framework NIA‐AA. Toward a biological definition of Alzheimer's disease. Alzheimers Dement. 2018;14:535‐562. - PMC - PubMed
    1. U.S. Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation Early Alzheimer's Disease: Developing Drugs for Treatment, Guidelines for Industry. 2018.
    1. Rabinovici GD, Gatsonis C, Apgar C, et al. Association of amyloid positron emission tomography with subsequent change in clinical management among medicare beneficiaries with mild cognitive impairment or dementia. JAMA. 2019;321(13):1286‐1294. - PMC - PubMed
    1. FDA‐NIH Biomarker Working Group . BEST (Biomarkers, EndpointS, and other Tools) Resource [Internet]. Silver Spring (MD): Food and Drug Administration (US); 2016. - PubMed
    1. Food and Drug Administration . Patient‐Focused Drug Development: Collecting comprehensive and representative input. Guidance for industry, Food and Drug Administration staff, and other stakeholders [Internet]. 2018 [cited 2019 Dec 17]. https://www.fda.gov/regulatory‐information/search‐fda‐guidance‐documents...

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