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. 2024;14(4):809-821.
doi: 10.3233/JPD-230444.

Patient and Public Involvement and Engagement in the Development of a Platform Clinical Trial for Parkinson's Disease: An Evaluation Protocol

Marie-Louise Zeissler  1 , Nikul Bakshi  2 , Michèle Bartlett  3 , Amit Batla  4 , David Byrom  3 , Rebecca Chapman  1 , Sally Collins  1 , Elaine Cowd  3 , Eric Deeson  3 , Romy Ellis-Doyle  4 , Jodie Forbes  3 , Cristina Gonzalez-Robles  4 , Anna Jewell  3 , Emma L Lane  5 , Nancy R LaPelle  6 , Keith Martin  3 , Helen Matthews  7 , Laurel Miller  3 , Georgia Mills  4 , Antony Morgan  3 , Miriam Parry  8 , Kuhan Pushparatnam  3 , Natasha Ratcliffe  9 , Dorothy Salathiel  3 , Paula Scurfield  3 , Carroll Siu  3 , Sue Whipps  3 , Sheila Wonnacott  3 , Thomas Foltynie  4 , Camille B Carroll  1   10 , Kevin McFarthing  3 ; EJS ACT-PD consortium
Affiliations

Patient and Public Involvement and Engagement in the Development of a Platform Clinical Trial for Parkinson's Disease: An Evaluation Protocol

Marie-Louise Zeissler et al. J Parkinsons Dis. 2024.

Abstract

Background: Patient and public involvement and engagement (PPIE) in the design of trials is important, as participant experience critically impacts delivery. The Edmond J Safra Accelerating Clinical Trials in PD (EJS ACT-PD) initiative is a UK consortium designing a platform trial for disease modifying therapies in PD.

Objective: The integration of PPIE in all aspects of trial design and its evaluation throughout the project.

Methods: PwP and care partners were recruited to a PPIE working group (WG) via UK Parkinson's charities, investigator patient groups and participants of a Delphi study on trial design. They are supported by charity representatives, trial delivery experts, researchers and core project team members. PPIE is fully embedded within the consortium's five other WGs and steering group. The group's terms of reference, processes for effective working and PPIE evaluation were co-developed with PPIE contributors.

Results: 11 PwP and 4 care partners have supported the PPIE WG and contributed to the development of processes for effective working. A mixed methods research-in-action study is ongoing to evaluate PPIE within the consortium. This includes the Patient Engagement in Research Scale -a quantitative PPIE quality measure; semi-structured interviews -identifying areas for improvement and overall impressions of involvement; process fidelity- recording adherence; project documentation review - identifying impact of PPIE on project outputs.

Conclusions: We provide a practical example of PPIE in complex projects. Evaluating feasibility, experiences and impact of PPIE involvement in EJS ACT-PD will inform similar programs on effective strategies. This will help enable future patient-centered research.

Keywords: Evaluation study; Parkinson’s disease; clinical trial protocol; patient and public involvement.

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Conflict of interest statement

CBC, TF, ELL and KMF are Editorial Board members of this journal, but were not involved in the peer-review process nor had access to any information regarding its peer review.

Figures

Fig. 1
Fig. 1
The EJS ACT-PD consortium structure. The consortium consists of five working groups each charged with one aspect of the trial’s development: Trial design, treatment selection, outcome measures, infrastructure, funding, and sustainability. Two to three PwP/care partners together support each of the five working groups. Together with charity representatives as well as trial delivery experts they form a sixth patient and public engagement working group. The chairs of each working group and the project leads form the steering group. A core team consisting of a project funded administrator, project manager, and research staff overseen by the project leads support the working groups in delivering their objectives.
Fig. 2
Fig. 2
Study overview. a) The evaluation study integrates both quantitative and qualitative data capture elements allowing for an ongoing evaluation of PPIE within the EJS ACT-PD initiative. The rigorous, systematic and iterative evaluation framework will allow PPIE to be continually adapted to improve PPIE within EJS ACT-PD and to inform best practice of PPIE in relation to existing PPIE quality guidance. b) Timeline of evaluation processes throughout the EJS ACT-PD project.
See this image and copyright information in PMC

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