What Parkinson's patients want from treatment


Another successful public-private-patient collaboration provides new insight into what patients want from treatment.  Listen to the PREFER webinar where patients in this Parkinson’s disease Project identified a new benefit they would trading off against a treatment’s burden.

On 10 April 2018, PREFER arranged a webinar on using the two threshold technique formats to elicit benefit-risk preferences for Parkinson’s Disease device attributes. The webinar is now available for viewing.

Speakers: Brett Hauber, PhD, Senior Economist, Vice President of Health Preference Assessment, RTI Health Solutions & Stephanie Christopher, Program Director, Medical Device Innovation Consortium.

Moderator: Bennett Levitan, Senior Director, Epidemiology, at Janssen R&D and one of the leaders of PREFER's methodology work.

About the project

The Medical Device Innovation Consortium (MDIC), the Michael J Fox Foundation for Parkinson’s Research, the Center for Devices and Radiological Health of the US Food and Drug Administration, RTI Health Solutions, and the Massachusetts Institute of Technology are working together on a project to include patient preferences into clinical trials for devices to treat Parkinson’s disease. The 3-phase study is collecting data to understand how and when to include both a patients’ urgency for new treatment options and their level of acceptance for uncertainty regarding a treatment’s effect. The urgency means the severity of the disease and how many people the disease affects. The uncertainty means a bigger possibility that the treatment will not work for a patient.

Phase 1 used patient advocate, patient scientist, and regulatory consultants’ opinions to create a list of important effects of a new hypothetical device. The list included both the good and the bad effects.  Researchers then asked participants to rank how much they valued the good and bad effects. 

The top five good effects were increase in daily “on time”, decrease in motor symptoms, decrease in PD pain, decrease in cognitive impairment, and decrease in medication and side-effect burden. Notably, the good effect of treating Parkinson’s disease pain was only identified with patient input!  This alone is a success of the project. The top four bad effects were worsening depression or anxiety, serious brain bleed, increase in 1-year mortality risk, increase in wait time for getting the new device. 

In Phase 2, researchers created a survey to measure how much of each top effect a patient would accept from the new treatment. To measure this, participants answered multiple questions. Each question required choosing between the good and bad effects of a standard treatment versus the good and bad effects of the new treatment. 

Initial survey results showed that the age of the participant affected how much of a good or bad effect they would accept.  But complete results were not available for this PREFER webinar.  Complete Phase 2 results were presented during a webinar held by MDIC on May 18th.

Phase 3 of the study will be a model developed to understand the acceptable level of evidence and uncertainty when developing clinical studies of Parkinson’s devices. Keep an eye out on the PREFER website and at MDIC.org for upcoming results from the final phase of this very successful public-private-patient collaboration in Parkinson’s disease.

By Kristin Bullok

Last modified: 2021-11-10