Recently, Cure Parkinson’s was pleased to see the results of one of our funded studies published in the Journal of Parkinson’s Disease. This study aimed to understand the perspectives among Parkinson’s research stakeholders on how trials of potentially disease-modifying drugs (that might slow or stop Parkinson’s progression) should be designed in the future.
Led by Professor Camille Carroll at the University of Plymouth, this study was initiated to help inform the design of the upcoming Edmond J. Safra Accelerating Clinical Trials for Parkinson’s Disease (EJS ACT-PD) project – a multi-arm, multi-stage clinical trial platform for testing multiple disease-modifying treatments for Parkinson’s simultaneously.
92 participants from 13 countries participated in this study, known as a ‘delphi survey’. The panelists represented a wide range of stakeholder groups; people with Parkinson’s and care partners, clinical scientists, industry and regulatory representatives, and funders. Over the course of four survey rounds, panellists were asked to evaluate factors within four domains relevant to Phase 3 trial design: overall goals and trial structure, inclusion criteria, assessment of trial outcomes, and trial delivery (for example if participants are required to regularly visit a hospital).
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What are delphi surveys?
A delphi survey is a multi-round surveying method designed to help a group reach a consensus. At the end of each survey round, participants can view all the results and compare their responses to the rest of the group. This helps reign-in outliers and move the group towards reaching an agreement in subsequent survey rounds.
Overall, the panel reached consensus on 14 of the 31 survey items. Many of these items were factors that would either help speed up the clinical trial process, such as having multiple treatments being tested simultaneously, or initiatives that would ease participant burden, including unanimous support for offering virtual or home-based visits when possible. The domain the panel were most divided on was inclusion criteria. Generally, people with Parkinson’s were more likely to support broader eligibility requirements compared to industry representatives. This may be in response to the often narrow inclusion criteria in current clinical trials, meaning the demographic involved in clinical trials may not be representative of the wider Parkinson’s population.
Ultimately, this paper provides insights into stakeholder priorities and highlights potential considerations when designing new clinical trials for Parkinson’s. Representing the voice of people with Parkinson’s in this study provides an opportunity for trial protocols to develop in alignment with patient priorities and improve participant experiences in research. This has been especially important in the design of the EJS ACT-PD project, where the Patient and Public Involvement and Engagement (PPIE) Group have driven decisions throughout the trial’s design. We look forward to seeing the impact of this as the platform moves closer to launch.