Our GO-CART patient engagement program helped to improve the development of our trial protocol substantially. In addition to identifying important outcomes for measure in our upcoming trial, our patient partners helped to describe our systematic review findings in an accessible manner. Our patient partners provided guidance on how to improve our study materials, including the interview study recruitment document, interview guide, and survey, in addition to providing their perspective on the key findings. One of our patient partners also provided insight on important patient and caregiver expenses and helped to ensure that the early economic analysis model appropriately captures a blood cancer patient’s care pathway. Furthermore, development of a terms of reference helped to document how our team worked together. To our knowledge, this is one of relatively few blood cancer treatment programs wherein patient partners worked alongside the research team from inception to develop a trial protocol for an early phase clinical trial.
Benefits of Patient Engagement in Early Phase Clinical Trials
Implementation of the GO-CART patient engagement program resulted in numerous benefits. Partnering with patients from inception and obtaining the patient perspective throughout our research program helped to improve not only each individual component study, but as well the final clinical trial protocol. Most importantly, we believe working with our patient partners from inception of trial development will help improve the relevance of the clinical trial findings to patients. For instance, based on the outcomes identified by our patient partners we plan to include a quality of life measurement within our trial. The impact of a treatment on the quality of life of a patient is a key piece of information for both patients and oncologists, and yet its use remains relatively low in cancer trials (24). Despite the early-phase nature of our trial, collecting quality of life information from participants may provide us with critical data moving forward.
We believe a key component to the success of our engagement program was engaging with patients early (at the inception of the project) to ensure our relationships were built on a strong foundation. This is consistent with research teams engaging stakeholders in other health areas such as depression and asthma. McConnell et al. reported that early engagement was beneficial in that it “promoted equal ownership” (25), while Supple et al. noted that engagement “is often most impactful in the project formation phase” (26). Certainly, our own experience would support these previous observations, as we believe our early engagement of patient partners improved feasibility of incorporating their feedback, strengthened relationships, and thereby maximized impact of patient engagement.
We also found engaging our patient partners all the way through the research continuum (e.g. discussion of study results and co-production of manuscripts) to be a rewarding experience. While a review of clinical trials found that most trials involving patients do try to continue their initiatives across the research continuum, overall very few report engaging patients at all (9). By working with our patient partner to identify key themes from the interview study we were able to ensure that the results we focused on were also felt to be of importance from a patient’s perspective. Additionally, by working with our patient partners to develop a non-technical summary of the systematic review findings we hope to have improved the accessibility of our findings to a wider audience. A recent study similarly reported that working with stakeholder partners throughout the analysis and dissemination phases of a project not only improved the format of the article but as well allowed for incorporation of “real-world interpretations” (25).
Our patient partners also found participating in this research project to be a rewarding experience. TH noted that being involved allowed him to learn more about “how things work,” which he felt enabled him to provide more meaningful feedback. He also felt that being involved throughout the various stages of the project provided him with a sense of contribution to improving patient outcomes.
Challenges & Areas for Improvement
A key challenge of this project was the identification of our patient partners, and in turn, difficulty in obtaining multiple and diverse perspectives. Though various recruitment methods were used, we were only able to recruit two patient partners. As we primarily recruited through a third party, it is unclear why we faced this challenge, however we speculate that a few factors may have contributed, such as patients needing to focus on their health care, lack of compensation for time, and timing of meetings. We aim to address the issue of compensation for time in future initiatives as described in detail below. We also speculate that patients may have been intimidated by the technical nature of some of the activities involved in developing an early phase clinical trial protocol (review of the existing evidence, early economic analysis). Though we aimed to address this issue by highlighting that no prior experience with clinical trials was needed in our advertisement, we recognize that further effort, such as working with patient partners to develop recruitment materials (e.g. testimonial video), will be required in future initiatives.
We recognize that a limitation of involving only two patients is the potential for bias towards personal views and experiences of a limited number of patient voices. As we start to expand to a larger patient panel, we aim to work towards involving more partners with diverse backgrounds and viewpoints. We also note that we did not engage with patient partners who themselves were eligible for CAR-T cell therapy. While this was done to ensure that engagement was feasible, we recognize that engagement of this population may have yielded different perspectives. To address this limitation, we attempted to gain input from patients who would be eligible for CAR-T cell therapy through our barriers/enablers to trial delivery interviews and survey, which allowed for short-term participation (one-hour phone or in-person interview). Furthermore, we were unable to recruit any caregiver partners, a group who our patient partners pointed out would additionally provide a meaningful and unique perspective. Challenges in recruiting have been reported by others in the literature (9, 14, 27). A previous review aimed to characterize best methods to identify and recruit patient partners, however, limited reporting and lack of comparison studies resulted in few proposed solutions (14). Our experience would suggest that targeted recruitment through direct referrals may be more successful than open calls for patient partners. Clearly, further work remains to determine best methods of practice for identification and recruitment of patient partners.
Another key challenge stemmed from the technical nature of early-phase clinical trials. Because the primary outcome of most early phase clinical trials is safety and feasibility, much of the focus is shifted towards the therapy and away from the clinical aspects. In our experience, it is clear that cell therapy development processes are complicated concepts to understand for individuals without a basic science background. Not only does this challenge patient partner engagement, but also that of all team members without basic science training. Although this is a clear limitation throughout this program, it has highlighted the need to explore avenues for patient engagement in basic science research.
When providing feedback on the program, our patient partner (TH) stressed the importance of training and provision of background information. Though TH felt the education and briefing sessions were well done, he suggested several potential areas for improvement including ensuring topics are covered at a manageable pace (e.g. parsed into several sessions), sending presentations, materials and a list of acronyms ahead of time to allow for partners to do their own reading and research, as well as some additional topics of interest. Another potential option may be to co-develop and produce educational sessions with a patient partner, as done by Bell et al. for a patient-oriented research curriculum (28). TH also noted that reflection and iterative revision of the terms of reference, and consistently communicating how patient partner contributions were incorporated into the project (and any associated outcomes) are essential to providing a sense of accomplishment. These activities also help to highlight how contributions are valued. Although many of these goals were met, an increase in personnel dedicated to patient engagement in our program would have helped ensure these issues were consistently addressed.
We were unable to provide monetary compensation for our patient partners’ time; however, we were able to reimburse for all travel and accommodation fees incurred for meetings. These terms were discussed with our patient partners prior to beginning the project, as well as outlined in our terms of reference. For future projects, we are incorporating patient partner compensation into our grant application budgets, in accordance with recent guidance published by the Strategy for Patient Oriented Research (SPOR) Networks in Chronic Diseases and Primary and Integrated Health Care Innovations (PICHI) Network (29) and the SPOR Evidence Alliance (30). Upon asking for feedback from our patient partner, it was also noted that re-payment of expenses associated with travel tended to be slow from our academic institution. We aim to better address this concern by communicating reimbursement timelines to our patient partners, setting firm targets and connecting our patient partners with a research assistant who will help to expedite the process.
At various times throughout the program, our team (both researchers and patient partners) faced technical difficulties when using the teleconference software. This led to some frustration, however with time and practice our team was able to overcome this issue. Another challenge was that team meetings typically took place during usual business hours. As our patient partners both work full-time, our team greatly appreciated that they were willing to take the time to attend and understood that other priorities would sometimes prevent them from attending. We aimed to address this challenge by documenting discussions through meeting minutes and encouraging our patient partners to schedule a phone discussion with the lead investigator if they had any remaining questions or were interested in a recap of the discussion. Finally, while we had aimed to provide consistent updates through a newsletter, given the small number of patient partners engaged we ended up providing regular updates through email and felt that a more frequent newsletter would have been redundant. However, as the program expands to a larger patient panel we intend to send more consistent updates though a newsletter, which will also help to ensure patient partner contributions are summarized and shared with the team and a broader network of stakeholders.