Embedding Patient Input in Outcome Measures for Long‐Term Disease‐Modifying Parkinson Disease Trials
Embedding Patient Input in Outcome Measures for Long‐Term Disease‐Modifying Parkinson Disease Trials
Where did the research take place?
The study site has not been established. Author addresses may differ from where the research occurred.
London, GB · Author affiliation
Department of Clinical and Movement Neurosciences, UCL Queen Square Institute of Neurology University College London London United KingdomLocation evidence
Guildford, GB · Author affiliation
Expert by experience Guildford United KingdomLocation evidence
Oxford, GB · Author affiliation
Nuffield Department of Clinical Neurosciences University of Oxford Oxford United KingdomLocation evidence
Bristol, GB · Author affiliation
Population Health Sciences University of Bristol Bristol United KingdomLocation evidence
GB · Author affiliation · country only
Translational and Clinical Research Institute Clinical Ageing Research Unit Newcastle University Newcastle United KingdomLocation evidence
Canterbury, GB · Author affiliation
Expert by experience Canterbury United KingdomLocation evidence
Cambridge, GB · Author affiliation
Department of Clinical Neurosciences University of Cambridge Cambridge United KingdomLocation evidence
Plymouth, GB · Author affiliation
Faculty of Health University of Plymouth Plymouth United KingdomLocation evidence
Gothenburg, SE · Author affiliation
Department of Psychiatry and Neurochemistry, Institute of Neuroscience and Physiology, The Sahlgrenska Academy University of Gothenburg Mölndal SwedenLocation evidence
Mölndal, SE · Author affiliation
Department of Psychiatry and Neurochemistry, Institute of Neuroscience and Physiology, The Sahlgrenska Academy University of Gothenburg Mölndal SwedenLocation evidence
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Original abstract
AbstractBackgroundClinical trials of disease‐modifying therapies in PD require valid and responsive primary outcome measures that are relevant to patients.ObjectivesThe objective is to select a patient‐centered primary outcome measure for disease‐modification trials over three or more years.MethodsExperts 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.ResultsAlthough 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.ConclusionsThe 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.