Table 1 Challenges and solutions for patient engagement in COA research

Capacity of patients to engage

Each research team member must balance their role with their own daily life and work priorities, and patients must also live with and manage their disease daily. Researchers should allow for this and be mindful of where agreed tasks exceed capacity or time constraints of patients via regular review

Mediation and communication

An orchestrator with good interpersonal skills to keep the team united and committed over time is vital, especially where patient experts disagreed with each other

Managing expectations and communication

Where patient advice is not taken forward, decisions must be explained transparently to retain trust and a sense of ownership/feeling valued; for example, not all patient suggestions for improvements or outcomes to measure may be feasible in a trial (either due to trial design, such as trial duration, or due to hypothesized treatment benefit resulting from treatment mode of action), may be easily translated into a study endpoint, or may be appropriate for approval and labeling decisions by regulators

Maintenance of good communication on timelines and when patients may expect documents for comment may be challenging as the project progresses due to external factors including completion of the patient interviews or the volume of data to be analyzed

Pharmaceutical companies should be prepared for the unique needs of the patient community partners. For in-person meetings patient partners experts may need to fly in a day early to avoid fatigue at the time of a meeting or stay at a hotel that is in a central location; this may lead to higher costs for meetings but is essential to address respect and accessibility

Managing sources of irritation

The long R&D timeframe may be frustrating to patients, especially if their disease is degenerative and disabling, and it is often the case that research to which they are contributing may not directly benefit them or the course of their personal disease experience; however, the patient experts in this research had regular experience of contributing to research that would not directly benefit them

The duration of web-based meetings was challenging and required occasional in-depth analysis of data in a short time period, necessitating long calls. The maximum duration of calls was reduced from an initial 3 h and

mid-way breaks were introduced, based on feedback from patient experts

Time was an important project resource; all stakeholders had to respect meeting deadlines and the overall timescale for project plans. Unavoidable lengthy gaps between meetings and deadlines occasionally led to wavering motivation

Time and resource challenges

The imbalance in legal resources available to POs and the pharmaceutical company created delays in reaching mutually acceptable legal and compliance agreements

Addressing training needs and preparing specific documents in lay terms (with probes, accessible visuals summarizing analysis outputs, etc.) to elicit patient expert feedback was also time and resource intensive

Additional meetings to engage with patient experts required preparation of materials and post-meeting summary notes; some meetings had to be repeated to gather contributions from patient experts in different time zones

Ensuring that technical aspects of the work were presented in a way that could be easily comprehensible by patient experts also required time to explain processes throughout

The time required for proper publication development, with iterative rounds of review and shaping of content was not anticipated by patient experts and was a source of frustration, particularly where it was perceived that the patient-centered focus of the research was secondary to the tone of scientific reporting. With hindsight, better upfront communication of the publication process and timelines involved would have been beneficial. The inclusion of all 6 patient experts as co-authors also impacted progress, given the unpredictable nature of progressing Parkinson’s; future projects may benefit from appointing specific patients as co-authors to represent the group. However, having patient experts as co-authors, motivated the decision to publish all research outputs from project inception with plain language summaries