World Parkinson’s Day 2022 - A patient centred model for Parkinson's research

Last November UCB, Parkinson’s UK, and the Parkinson's Foundation in the U.S., announced that they had joined forces to establish a Patient Engagement Council for Parkinson’s Research (PECPR), building upon existing work they had been doing together for many years.

The aim of the council is to ensure that patient insights are central to the overall strategy and activities across UCB’s Parkinson’s disease research and early clinical development program. Through this collaborative partnership, the council aims to embed patient involvement in the earliest stages and throughout each step of UCB’s Parkinson’s drug development program to improve outcomes for people living with Parkinson’s.

In the last few months, the group has made great strides in focusing in on what areas they consider will have the greatest impact and value for people living with Parkinson’s. Topics discussed included decentralised research, the optimization of clinical trial design and experience, an understanding disease modification therapy, and patient informed Target Patient Value Profiles (TPVPs).

The PECPR adopted the Clinical Trials Transformation Initiative (CTTI) prioritisation tool, to help identify which of the focus areas presented high-value opportunities. These topics were then ranked further leading to a consensus on the top priorities. The group overwhelmingly agreed to concentrate initially on TPVPs, to embed early patient involvement and co-creation in the drug development process.

The purpose of a TPVP is to ensure that the drug development process is efficient and provides all the required relevant medical, technical, and scientific information for evaluating the viable outcome of a potential new medicine. Historically the early design of TPVP’s was largely driven by joint insights from clinical scientists, desk research and conversations with expert healthcare professionals, with little or no participation of people living with the condition.

To help address these issues the PECPR will look to build a transparent and robust process clearly setting out where and how patients should be involved to inform and co-create the development of TPVP’s in the future. 

In order to bolster representation from the Parkinson’s community the PECPR has welcomed three people who live with Parkinson’s. Each of them has a very personal Parkinson’s story, and they described why they wanted to be involved with the group:

• Carol Schulte, a patient advocate from the United States, living with Parkinson’s for over 20 years: “My primary motivator for joining the PECPR is to ensure that the development of TPVP’s are inclusive of all groups, taking into account the concerns and considerations of all people living with Parkinson’s. When I was diagnosed in 1998, so little was understood about Parkinson's, even by the medical profession, and I have been fascinated by the development of the Movement Disorders specialty.”

• Harold de Wit, a patient advocate from the Netherlands, diagnosed with Parkinson’s in 2018: “When I first learned I had Parkinson’s my world collapsed, but along the way I managed to accept this fact. But acceptance is not resignation. I will fight this disease until the better end. By joining PECPR I can extend this fight and make it a collective battle of many against this terrible disease. Finding new therapies or even a cure against Parkinson’s is all about co-creation of the Parkinson’s community. Patients, healthcare professionals, pharmaceutical companies and foundations have to come together to make a stand. PECPR is a beautiful example of co-creation to join forces!”

• Marc van Grieken, a patient advocate from the United Kingdom, diagnosed with Parkinson’s in 2006: “I firmly believe that research will in the end lead to finding a cure.  Without research: nothing will happen. Research with proactive, continuous and equitable involvement of people with Parkinson’s will get to the solution much sooner than research without such involvement. Patient Engagement Councils can play a meaningful role in this but how do we know what is meaningful? Only through participating in initiatives such as the Patient Engagement Council of UCB, the Parkinson’s Foundation and Parkinson’s UK, may I ultimately be able to express an opinion about success, limitation or even failures of ‘patient engagement.’”  

We look forward to providing more updates on the work of the group as we progress our ambitious agenda.