TY - JOUR
T1 - Outcome Measures for Disease-Modifying Trials in Parkinson's Disease
T2 - Consensus Paper by the EJS ACT-PD Multi-Arm Multi-Stage Trial Initiative
AU - Gonzalez-Robles, Cristina
AU - Weil, Rimona S
AU - van Wamelen, Daniel
AU - Bartlett, Michèle
AU - Burnell, Matthew
AU - Clarke, Caroline S
AU - Hu, Michele T
AU - Huxford, Brook
AU - Jha, Ashwani
AU - Lambert, Christian
AU - Lawton, Michael
AU - Mills, Georgia
AU - Noyce, Alastair
AU - Piccini, Paola
AU - Pushparatnam, Kuhan
AU - Rochester, Lynn
AU - Siu, Carroll
AU - Williams-Gray, Caroline H
AU - Zeissler, Marie-Louise
AU - Zetterberg, Henrik
AU - Carroll, Camille B
AU - Foltynie, Thomas
AU - Schrag, Anette
AU - EJS ACT-PD Consortium
AU - Sammler, Esther
PY - 2023/9/8
Y1 - 2023/9/8
N2 - BACKGROUND: Multi-arm, multi-stage (MAMS) platform trials can accelerate the identification of disease-modifying treatments for Parkinson's disease (PD) but there is no current consensus on the optimal outcome measures (OM) for this approach.OBJECTIVE: To provide an up-to-date inventory of OM for disease-modifying PD trials, and a framework for future selection of OM for such trials.METHODS: As part of the Edmond J Safra Accelerating Clinical Trials in Parkinson Disease (EJS ACT-PD) initiative, an expert group with Patient and Public Involvement and Engagement (PPIE) representatives' input reviewed and evaluated available evidence on OM for potential use in trials to delay progression of PD. Each OM was ranked based on aspects such as validity, sensitivity to change, participant burden and practicality for a multi-site trial. Review of evidence and expert opinion led to the present inventory.RESULTS: An extensive inventory of OM was created, divided into: general, motor and non-motor scales, diaries and fluctuation questionnaires, cognitive, disability and health-related quality of life, capability, quantitative motor, wearable and digital, combined, resource use, imaging and wet biomarkers, and milestone-based. A framework for evaluation of OM is presented to update the inventory in the future. PPIE input highlighted the need for OM which reflect their experience of disease progression and are applicable to diverse populations and disease stages.CONCLUSION: We present a range of OM, classified according to a transparent framework, to aid selection of OM for disease-modifying PD trials, whilst allowing for inclusion or re-classification of relevant OM as new evidence emerges.
AB - BACKGROUND: Multi-arm, multi-stage (MAMS) platform trials can accelerate the identification of disease-modifying treatments for Parkinson's disease (PD) but there is no current consensus on the optimal outcome measures (OM) for this approach.OBJECTIVE: To provide an up-to-date inventory of OM for disease-modifying PD trials, and a framework for future selection of OM for such trials.METHODS: As part of the Edmond J Safra Accelerating Clinical Trials in Parkinson Disease (EJS ACT-PD) initiative, an expert group with Patient and Public Involvement and Engagement (PPIE) representatives' input reviewed and evaluated available evidence on OM for potential use in trials to delay progression of PD. Each OM was ranked based on aspects such as validity, sensitivity to change, participant burden and practicality for a multi-site trial. Review of evidence and expert opinion led to the present inventory.RESULTS: An extensive inventory of OM was created, divided into: general, motor and non-motor scales, diaries and fluctuation questionnaires, cognitive, disability and health-related quality of life, capability, quantitative motor, wearable and digital, combined, resource use, imaging and wet biomarkers, and milestone-based. A framework for evaluation of OM is presented to update the inventory in the future. PPIE input highlighted the need for OM which reflect their experience of disease progression and are applicable to diverse populations and disease stages.CONCLUSION: We present a range of OM, classified according to a transparent framework, to aid selection of OM for disease-modifying PD trials, whilst allowing for inclusion or re-classification of relevant OM as new evidence emerges.
KW - Humans
KW - Consensus
KW - Disease Progression
KW - Outcome Assessment, Health Care
KW - Parkinson Disease/drug therapy
KW - Quality of Life
U2 - 10.3233/JPD-230051
DO - 10.3233/JPD-230051
M3 - Article
C2 - 37545260
SN - 1877-7171
VL - 13
SP - 1011
EP - 1033
JO - Journal of Parkinson's Disease
JF - Journal of Parkinson's Disease
IS - 6
ER -