Parkinson’s Patients’ Tolerance for Risk and Willingness to Wait for Potential Benefits of Novel Neurostimulation Devices: A Patient-Centered Threshold Technique Study (PCOR Project Aim 2 paper)

Feb 19, 2021Clinical, Patient Centered Outcomes Research (PCOR), Regulatory Decision, SPI Guiding Principles

Patient Introduction

A growing literature has developed identifying outcomes that matter to patients. The study that these published articles (Aims 1, 2 & 3 of MDIC’s PCOR project) are based on demonstrates an approach to identifying outcomes of medical devices for Parkinson’s disease (PD) that are meaningful to patients and regulators. This approach is of interest to patients who seek to provide input to the design of medical device clinical trials to ensure that what matters to patients is incorporated into study endpoints. Although PD was the disease focused on for this study, the approach can be used to study patient perspectives about other disease or treatment areas. This paper summarizes Aim 2 of the project, describing the results of a survey of Parkinson’s patients focused on their willingness to accept certain trade-offs in the development of a potential new treatment device.

Summary

Parkinson’s disease (PD) is a neurodegenerative condition causing motor, cognitive, psychological, somatic, and autonomic symptoms. This publication reviews Aim 2 of the project, presenting the process by which PD patients were surveyed to understand the maximum level of risk that patients would accept to achieve the potential benefits of a neurostimulation device. The study’s objective was to understand patient preferences and willingness-to-wait for novel neurostimulation devices among PD patients to inform a model of optimal trial design. The study concluded that patients were willing to wait an average of 4 to 13 years for devices that provide different levels of benefit.