Capturing meaningful patient experiences in Parkinson’s research

Parkinson’s disease (PD) is an incurable neurodegenerative disorder that causes a range of motor and non-motor symptoms that begin gradually and worsen over time. Motor symptoms include unintended or uncontrollable movements, such as shaking, stiffness, and difficulty with balance and coordination. People may also have mental and behavioral changes, sleep problems, depression, memory difficulties, and fatigue. 

Capturing the true problems associated with living with PD from the early stages of the disease is critical, as substantial research efforts are ongoing to develop disease modifying therapies, and associated important and meaningful outcome measures for those clinical trials. 

Research specifically focused on the experience of people living with early-stage PD is limited and, to date, there is no consensus regarding the definition of early-stage PD among the scientific and regulatory communities. In addition, the clinical outcome assessments, used in clinical trials investigating early-stage PD, do not fully capture the subtle concepts meaningful to people with this disease.

UCB partnered with Parkinson’s UK and the Parkinson’s Foundation to coinvestigate the patient experience in early stages of the disease. Six people living with PD were part of the research team and involved from developing the study design through to analyzing the data.

Interviews with people with the disease and their relatives, were used to identify, those primary symptoms and impacts, which might be more useful to measure in clinical trials.

“Involving patients in research is a symbiotic partnership; this is very much the case in my experience in working with UCB. Right at the onset, this partnership had been a two-way process,” said Carroll Siu, PD patient expert. “The nurturing and guidance from UCB in the last 5 years had allowed me to gain knowledge, skills and confidence, which I could now transpose to my engagement in future research projects. UCB gained from our patient involvement to such an extent that they have formed a Patient Engagement Council for Parkinson’s research by working closely with Parkinson’s charities to involve patients at the heart of future Parkinson’s research.”

We learned that the concepts that were most meaningful to people living with early-stage PD were:

• bradykinesia/slowness (notably in the form of ‘functional’ slowness, with being slower at completing tasks reportedly more important than the difficulty of completing tasks)
• tremor
• rigidity/stiffness
• effects on mobility (particularly fine motor/dexterity and subtle gait abnormalities)
• fatigue
• depression
• sleep/dreams
• pain

“Incorporating the lived expertise of six people living with Parkinson's added rich insights and key knowledge to this project,” said Karlin Schroeder, Associate Vice President of Community Engagement at the Parkinson's Foundation. “The Parkinson's Foundation is honored to be collaborators in this work, helping to guide patient engagement in research. We look forward to our ongoing work with UCB, Parkinson's UK and the community to advance science and improve the lives of people with Parkinson's.”

Our study adds to the low amount of qualitative research on early-stage PD. It showed that patient experiences in early-stage PD are complex and wide-ranging, and that currently available patient-reported outcome measures do not evaluate many early-stage PD concepts such as functional slowness, fine motor skills and subtle gait abnormalities. The inclusion of patients in the research team provided a unique perspective based on first-hand experience of how PD affects all aspects of patients’ lives, and how they feel about living with and managing their condition.

Going forward, we need new measures, created in conjunction with people with PD, that fully assesses symptoms and the experience of living with early-stage PD. For more information on the study, you can visit https://link.springer.com/article/10.1007/s40120-022-00375-3.

A plain language summary of the research is now available in Future Medicine. See below to find out more:

Plain language summary: what symptoms should be measured in clinical studies for early-stage Parkinson's? (futuremedicine.com)