Abstract Details

Title Outcome Assessment in Early-Stage Parkinson’s Disease (PD) Clinical Trials: Are Legacy Patient-Reported Outcome (PRO) Instruments Fit for Purpose

Topic Movement Disorders

Presentation(s) P1 - Poster Session 1 (9:00 AM-5:00 PM)

Poster/Presentation
Number 258

Objective

To evaluate the extent to which legacy patient-reported outcome (PRO) instruments are fit for purpose for use in trials involving people with early-stage (Stages 1 and 2) Parkinson’s disease (PD).

Background Regulatory agencies increasingly recommend the use of PRO instruments in clinical trials investigating treatments aimed at slowing or stopping progression of PD, notably to assess improvements in patient daily functioning. Legacy PRO instruments typically used in PD research were not developed specifically for early-stage PD.

Design/Methods Cognitive debriefing interviews were conducted with 50 people living with early-stage PD, recruited through Parkinson’s UK and Parkinson’s Foundation. Participants completed the Parkinson’s Disease Questionnaire (PDQ-39) and Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Parts Ib (non-motor) and II (motor) and provided their feedback in parallel. A small-scale Rasch Measurement Theory (RMT) psychometric analysis was conducted.

Results

Qualitative feedback indicated concerns with item relevance/appropriateness and item clarity/ambiguity for the majority of the items of the PDQ-39 and MDS-UPDRS instruments, respectively, as well as some issues with the PDQ-39 response scale. Items related to walking problems and cutting up food for the PDQ-39, and constipation, swallowing pills or eating food, handling food, and freezing of gait for the MDS-UPDRS, were most frequently identified as not relevant to participants.

RMT analysis indicated sub-optimal targeting, particularly in scales related to motor and functional concepts, with the highest ceiling effects reported in the mobility scale for PDQ-39 and the motor domain of the MDS-UPDRS, where item content was found not to be relevant or was too easy for 41% and 20% of the participants, respectively.

Conclusions PDQ-39 and MDS-UPDRS Parts Ib and II are of limited use for evaluating
motor-related outcomes in clinical trials involving people living with early-stage PD.

Authors/Disclosures
Thomas Morel (UCB Pharma) PRESENTER	Dr. Morel has stock in UCB Pharma.
	No disclosure on file
	No disclosure on file
Roger Barker, PhD (University of Cambridge)	Roger Barker, PhD has nothing to disclose.
	No disclosure on file
Babak Boroojerdi, MD (UCB Biosciences GmbH)	Dr. Boroojerdi has received personal compensation for serving as an employee of UCB Biosciences. Dr. Boroojerdi has stock in UCB Biosciences.
	No disclosure on file
	No disclosure on file
Stefan Cano, MD (Modus Outcomes)	Stefan Cano, MD has received personal compensation in the range of $100,000-$499,999 for serving as an officer or member of the Board of Directors for Modus Outcomes. Stefan Cano, MD has received stock or an ownership interest from Modus Outcomes. Stefan Cano, MD has received intellectual property interests from a discovery or technology relating to health care.
Casey Gallagher (Parkinson's Foundation)	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file

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