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. 2024 May 14;10(1):101.
doi: 10.1038/s41531-024-00716-z.

Patient-centered development of clinical outcome assessments in early Parkinson disease: key priorities and advances

Tiago A Mestre  1 , Glenn T Stebbins  2 , Diane Stephenson  3 , David Dexter  4 , Karen K Lee  5 , Yuge Xiao  6 , Tien Dam  7 , Catherine M Kopil  6 , Tanya Simuni  8
Affiliations

Patient-centered development of clinical outcome assessments in early Parkinson disease: key priorities and advances

Tiago A Mestre et al. NPJ Parkinsons Dis. .

Abstract

Novel therapies with the ability to delay disease progression are a gap in the care of people living with Parkinson disease (PD) today. Clinical outcomes assessments (COAs) that are sensitive to the earliest clinical changes in PD are deemed essential for a successful therapeutic development. To understand the current landscape of COAs use in clinical trials in PD and define priorities for future research in the field, a stakeholder roundtable meeting was held in November 2022. The current paper 1) proposes the collaborative development of patient-centric COAs that can adequately document the effectiveness of disease modification therapies in PD based on key priorities identified during this initial meeting, 2) summarizes the progress made in the subsequent 12 months, and 3) presents the deliverables expected in the near future. Key priorities include 1) the development of a consensus conceptual model of early PD experiences, 2) the adaptation of existing patient-reported outcomes (PROs), 3) the investigation of the role of observer-reported outcomes in addition to 4) enabling diversity in PD research and advocacy, 5) fostering data sharing, and 6) reaching consensus on a biological staging system for PD to drive the development of appropriate PROs for biologically defined populations.

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Conflict of interest statement

Tiago A. Mestre: consulting and Advisory Board Membership with honoraria: Abbvie, International Parkinson and Movement Disorder Society, AAN, Abbvie, CHDI Foundation/Management, Sunovion, Valeo Pharma, Roche, nQ Medical, Medtronic. Grants and Research: EU Joint Program - Neurodegenerative Disease Research, uOBMRI, Roche, Ontario Research Fund, CIHR, Michael J. Fox Foundation for Parkinson’s Research, Parkinson Canada, PDF/PSG, LesLois Foundation, Parkinson Research Consortium and Brain Canada. Salary: UOMA. Glenn Stebbins: consulting and Advisory Board Membership with honoraria: Adamas Pharmaceuticals, CHDI Management, Inc., Cleveland Clinic Foundation, Huntington Study Group, Neurocrine Biosciences, Inc., Pfizer, Inc., Tools-4-Patients. Grants and Research: Critical Path Institute, Department of Defense, Dystonia Coalition, CHDI, International Parkinson and Movement Disorder Society, Michael J. Fox Foundation for Parkinson’s Research, Ottawa Hospital Research Institute. Honoraria: Alzheimer’s Association, Critical Path Institute, International Parkinson and Movement Disorder Society, Michael J. Fox Foundation for Parkinson’s Research. Diane Stephenson, David Dexter, Karen K Lee: no conflicts to disclose. Yuge Xiao: employee of The Michael J. Fox Foundation for Parkinson’s Research. No other disclosures. Tien Dam: employee of Neumora Therapeutics, Past employee of Biogen during preparation of this manuscript. Catherine M. Kopil: employee of The Michael J. Fox Foundation for Parkinson’s Research. No other disclosures. Tanya Simuni: consulting and Advisory Board Membership with honoraria: 4D Pharma, Acadia, AcureX, AskBio, Amneal, Blue Rock Therapeutics, Caraway Therapeutics, Critical Path for Parkinson’s Consortium (CPP), Denali, Michael J Fox Foundation, Neuroderm, Sanofi, Sinopia, Sunovion, Roche, Takeda, UCB, VanquaBio. Grants and research: Amneal, Biogen, Roche, Neuroderm, Sanofi, Prevail and UCB and an investigator for NINDS, MJFF, Parkinson’s Foundation.

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References

    1. Dorsey ER, Sherer T, Okun MS, Bloem BR. The emerging evidence of the Parkinson pandemic. J. Parkinson’s Dis. 2018;8:S3–S8. doi: 10.3233/JPD-181474. - DOI - PMC - PubMed
    1. Postuma RB, et al. MDS clinical diagnostic criteria for Parkinson’s disease. Mov. Disord. 2015;30:1591–1601. doi: 10.1002/mds.26424. - DOI - PubMed
    1. Kalia LV, Lang AE. Parkinson’s disease. Lancet. 2015;386:896–912. doi: 10.1016/S0140-6736(14)61393-3. - DOI - PubMed
    1. Chahine LM, et al. Proposal for a biologic staging system of Parkinson’s disease. J. Parkinson’s Dis. 2023;13:297–309. doi: 10.3233/JPD-225111. - DOI - PMC - PubMed
    1. Höglinger GU, et al. A biological classification of Parkinson’s disease: the SynNeurGe research diagnostic criteria. Lancet Neurology. 2024;23:191–204. doi: 10.1016/S1474-4422(23)00404-0. - DOI - PubMed
-