Embedding Patient Input in Outcome Measures for Long-Term Disease-Modifying Parkinson Disease Trials

Background: Clinical trials of disease-modifying therapies in PD require valid and responsive primary outcome measures that are relevant to patients. Objectives: The objective is to select a patient-centered primary outcome measure for disease-modification trials over three or more years. Methods: Experts in Parkinson's disease (PD), statistics, and health economics and patient and public involvement and engagement (PPIE) representatives reviewed and discussed potential outcome measures. A larger PPIE group provided input on their key considerations for such an endpoint. Feasibility, clinimetric properties, and relevance to patients were assessed and synthesized. Results: Although initial considerations favored the Movement Disorder Society-sponsored revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III in Off, feasibility, PPIE input, and clinimetric properties supported the MDS-UPDRS Part II. However, PPIE input also highlighted the importance of nonmotor symptoms, especially in the longer term, leading to the selection of the MDS-UPDRS Parts I + II sum score. Conclusions: The MDS-UPDRS Parts I + II sum score was chosen as the primary outcome for large 3-year disease-modification trials. © 2023 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

MEDICIN OCH HÄLSOVETENSKAP Medicinska och farmaceutiska grundvetenskaper Neurovetenskaper hsv//swe
MEDICAL AND HEALTH SCIENCES Basic Medicine Neurosciences hsv//eng
clinical trials
disease modification
endpoints
outcome measures
Parkinson disease
Patient and Public Involvement and Engagament (PPIE)

Bartlett, Michèle (author)
Burnell, Matthew (author)
Clarke, Caroline S. (author)
Haar, Shlomi (author)
Hu, Michele T. (author)
Huxford, Brook (author)
Jha, Ashwani (author)
Lawton, Michael (author)
Noyce, Alastair (author)
Piccini, Paola (author)
Pushparatnam, Kuhan (author)
Rochester, Lynn (author)
Siu, Carroll (author)
van Wamelen, Daniel (author)
Williams-Gray, Caroline H. (author)
Zeissler, Marie Louise (author)
Zetterberg, Henrik,1973Gothenburg University,Göteborgs universitet,Institutionen för neurovetenskap och fysiologi, sektionen för psykiatri och neurokemi,Institute of Neuroscience and Physiology, Department of Psychiatry and Neurochemistry(Swepub:gu)xzethe (author)
Carroll, Camille B. (author)
Foltynie, Thomas (author)
Weil, Rimona S. (author)
Schrag, Anette (author)
Göteborgs universitetInstitutionen för neurovetenskap och fysiologi, sektionen för psykiatri och neurokemi (creator_code:org_t)

About the subject

MEDICAL AND HEALTH SCIENCES: MEDICAL AND HEAL ...; and Basic Medicine; and Neurosciences

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