Below we have described patient participation in each component study of the Accelerator approach.
Patient partners met with the research team members to provide input on outcome measures to be extracted from included studies. Our patient partners identified quality of life and health utility measures as important outcomes to patients. We found that of the 60 clinical trials identified by our systematic review, none reported these outcome measures (12). Though we were unable to extract data for these outcomes, our finding points to the need for CAR-T cell therapy clinical trials to consult with patients during protocol development so that such outcomes can be measured and reported.
Patient partners also worked alongside the research team to develop a non-technical (‘plain language’) summary of the final results; this summary has proven to be a valuable resource as it describes the current evidence available on CAR-T cell therapy in language accessible to a wide audience. We have also found this document to be useful in describing the rationale for our trial in other component studies (e.g. patient survey).
Barriers/Enablers to Trial Delivery Interviews
Patient partners provided a letter of support to the Research Ethics Board (REB), reviewed patient-facing materials such as the recruitment information sheet, as well as the interview guide to provide feedback. Our patient partners helped to modify the language of these documents to ensure they were approachable and accessible. Patient partners also suggested additional questions that could help to identify potential barriers and enablers to patient participation in the trial and prompts to help with the flow of the interview.
Upon difficulty in recruiting our minimum number of participants for this interview study, our patient partner (TH) aided in identifying additional organizations to approach for help in recruitment. TH helped to liaise recruitment through the LLSC, as well as identify additional avenues through which to distribute our recruitment information, such as the Chronic Lymphocytic Leukemia Patient Advocacy Group (CLLPAG). Our connection with the CLLPAG led to a last-minute offer to advertise our study at a conference where over 200 patients would be in attendance; we were unable to capitalize on this opportunity however, as we were not able to obtain REB approval in time.
After completion of the interviews, coding and anonymization of the data, our patient partner TH was involved in providing his outlook on key themes identified from the patient and hematologist interviews, confirming which were most relevant from a patient perspective. This was done through both a meeting with the research team where preliminary results were presented and discussed, as well as through TH’s review of a document outlining key themes and illustrative quotes. This process helped to shape the manuscript around the themes felt to be of most importance to patients.
Barriers/Enablers to Trial Delivery Survey
One of our patient partners (TH) participated in a think-aloud session to pilot the survey, by providing comments on their interpretation of the questions, feasibility of the survey, and any technical issues. This allowed us to assess whether questions were interpreted appropriately, ensure that the survey was feasible, and make technical and aesthetic modifications prior to sending out the survey.
Early Economic Analysis
Our patient partner (TH) worked with the team’s health economists to provide input on various expenses that may be incurred by patients throughout their illness and which may impact trial participation. This was done so that the economic model captures patient care trajectory and related costs. Importantly, TH also pointed out costs that may be incurred by patients’ supporting family members and caregivers. Identified costs included travel, parking fees, lodging expenses, and loss of income. This collaboration allowed us to identify expenses that we would not have otherwise recognized. TH also provided his perspective on the expectation of CAR-T therapy efficacy, and as well confirmed that the overall structure of the economic model appropriately captured the care pathway of a patient with a hematological malignancy. (20)
Retrospective Cohort Study
Though we were not able to identify a patient partner role throughout conduct of the retrospective cohort study, our patient partner attended a team meeting where the results from this project were discussed. In the future, we look forward to exploring how patient partners may be more actively involved in this type of study.
Production & Testing of Cell Product
Our patient partners participated in an information session where the head of production provided an in-depth description of production methods to share knowledge on how CAR-T cell therapy is developed and tested, as well as to answer questions. As engagement throughout this component primarily focused on informing and information sharing, we hope to have continued discussions with our patient partners as to how we may more actively engage patients in future, similar studies.
Patient Partner Diagnosis and Treatment Experience
One of our patient partners (TH) shared their diagnosis and treatment story with the team. All members greatly appreciated TH sharing his diagnosis and care experience as this helped to enlighten the team on the typical care pathway faced by blood cancer patients. Although the ultimate goal to improve patient care is always our main motivation, as researchers, we can sometimes get caught up in details, documentation and deadlines. Through sharing his experience, TH further strengthened the team’s motivation and brought our ultimate goal to the forefront. Hearing the patient perspective also highlighted the importance of ‘humanizing’ trial development at the earliest stages to ensure all downstream development of a therapy is patient centered.
Expansion to a Patient Panel & Preparation for the Trial
As we prepared for the initiation of the clinical trial, we expanded our program to include a patient peer support panel. Recruitment for our interview study and networking at the BioCanRx Summit allowed for patients in the provincial and national communities respectively, to learn more about our program. This resulted in our team being approached by two patients who expressed interest in joining the team.
Individual components of the Accelerator studies were used to inform the clinical trial protocol and ensure that its development was evidence informed. In addition, our patient partners contributed to trial development by reviewing the protocol and providing input on multiple aspects (e.g. frequency of follow-up appointments, what should be communicated to enrolled participants). Our patient partners also provided input into the patient informed consent approach created by NK and the clinical team, including the informed consent form, visual consent aids and a non-technical summary. In order to aid these activities, our team submitted a successful grant application with our patient partners, which has enabled us to continue to expand, as well as host a face-to-face meeting with our patient partners.