INTRODUCTION
Parkinson Disease, Parkinson’s disease, PD
PARKINSON DISEASE
There is currently no proven intervention to delay the progression of Parkinson’s disease (PD). A number of novel and promising treatment approaches are being developed to address this and need to be tested in clinical trials. Multi-arm, multi-stage (MAMS) platform trials may help accelerate the identification of potentially successful treatments by improving efficiency of the clinical trial process. MAMS trials evaluate multiple agents simultaneously against a shared placebo arm and allow the addition of new arms as well as cessation of ineffective treatments at interim stages. However, there is no current consensus on the most appropriate outcome measures (OM) for disease-modifying trials in PD to be included in such an approach.The Edmond J Safra Accelerating Clinical Trials in Parkinson Disease (EJS ACT-PD) initiative aims to accelerate the identification of disease-modifying treatments for PD through a MAMS platform trial approach. An important component of this novel approach is the identification and selection of appropriate outcome measures, suitable for inclusion across several different study arms as well as meeting the overarching aim. Here, we present an inventory of outcome measures based on current evidence and make initial recommendations for their potential inclusion as core, supplementary (depending on study arm) or exploratory outcome measures in such trials.This inventory of potential outcome measures for use in disease-modifying trials is based on a consensus effort by an expert group with strong patient and public engagement input. The group used information from literature reviews, other existing and ongoing efforts, and discussion with regulatory bodies and group discussions. Particular consideration for inclusion in the inventory was given to clinically relevant outcome measures that are meaningful to patients, align with regulatory expectations and provide data to support adoption in larger healthcare systems. For future adaptation according to emerging new evidence, a framework was also created for evaluation and inclusion of outcome measures of potential relevance in the future, including clinical outcome measures, biomarkers, and novel measurement technologies.