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. 2024 Feb;39(2):433-438.
doi: 10.1002/mds.29691. Epub 2023 Dec 22.

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

Cristina Gonzalez-Robles  1 , Michèle Bartlett  2 , Matthew Burnell  3 , Caroline S Clarke  1 , Shlomi Haar  4 , Michele T Hu  5 , Brook Huxford  6 , Ashwani Jha  1 , Michael Lawton  7 , Alastair Noyce  6 , Paola Piccini  4 , Kuhan Pushparatnam  8 , Lynn Rochester  9 , Carroll Siu  10 , Daniel van Wamelen  11 , Caroline H Williams-Gray  12 , Marie-Louise Zeissler  13 , Henrik Zetterberg  14 , Camille B Carroll  9   13 , Thomas Foltynie  1 , Rimona S Weil  1   15 , Anette Schrag  1 ; EJS ACT-PD Consortium
Affiliations

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

Cristina Gonzalez-Robles et al. Mov Disord. 2024 Feb.

Abstract

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.

Keywords: Parkinson disease; Patient and Public Involvement and Engagament (PPIE); clinical trials; disease modification; endpoints; outcome measures.

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