Partnering with patients to get better outcomes with chimeric antigen receptor T-cell therapy: towards engagement of patients in early phase trials

Abstract

Aim Though patient engagement in clinical research is growing, recent reports suggest few clinical trials report on such activities. To address this gap, we describe our approach to patient engagement in the development of a clinical trial protocol to assess a new immunotherapy for blood cancer (chimeric antigen receptor T-cell therapy, CAR-T cell therapy). Methods Our team developed a clinical trial protocol by working with patient partners from inception. Two patient partners with lived blood cancer experience were identified through referrals from our team’s professional network and patient organization contacts. Our patient partners were onboarded to the team and engaged in several studies conducted to develop the clinical trial protocol, including a systematic review of the existing literature on the therapy, patient interviews and a survey to obtain perspectives on barriers and enablers to participating in the trial, an early economic analysis, and a retrospective cohort study. Results Engaging patient partners enhanced our research in ways that would not have otherwise occurred. By selecting patient important outcomes for data collection, our partners helped flag that quality of life and health utility measures have not been reported in previous CAR-T cell therapy trials for blood cancer. Our partners also co-developed a non-technical summary of the systematic review that summarized results in an accessible manner. Our patient partners reviewed interview and survey questions, to improve the language and appropriateness; provided recruitment suggestions; and provided a patient perspective on the results, thereby confirming the importance of findings. Input was also obtained on costs for the early economic analysis. Our patient partners identified costs that may be a burden to both patients and caregivers during a trial and helped to confirm that the overall structure of the economic model reflected the patient care pathway. Our patient partners also shared their diagnosis and treatment stories, which helped to provide the research team with insight into this experience. Conclusions Contributions by our patient partners were invaluable to each component study, as well as the overall development of the trial protocol. We plan to use this approach in the future in order to meaningfully engage patients in the development of other clinical trials; we also hope that by reporting our methods this will help other research teams to do the same. Trial registration Affiliated with the development of NCT03765177.

Navigating choice in the face of uncertainty: using a theory informed qualitative approach to identifying potential patient barriers and enablers to participating in an early phase chimeric antigen receptor T (CAR-T) cell therapy trial

“…For example, patient partners have provided feedback on the trial protocol, consent forms, visual aides, and lay summaries, and in developing plans to organise a patient peer support panel. 58 …”

Patient and Patient Group Engagement in Cancer Clinical Trials: A Stakeholder Charter

“…Establishing partnerships with patients and patient groups (PGs) to engage them in all levels of clinical trials (CTs), including in real-world evidence research, is increasingly explored and applied globally [ 1 , 2 , 3 , 4 , 5 , 6 , 7 , 8 , 9 , 10 , 11 , 12 , 13 , 14 , 15 , 16 , 17 , 18 , 19 , 20 , 21 , 22 , 23 ]. Keys to such engagement—also referred to as co-design research, participatory research, patient-oriented research (POR), patient-involved research, patient public involvement, patient participation, co-creative research, citizen science, patient-centric initiative, open science—are the understanding and the manifestation of the concept of patient centricity or patient centeredness [ 1 , 3 , 4 , 24 , 25 , 26 , 27 ].…”

“…Advantages of involving the end-user groups in the broad research agenda include increased research relevance to both clinicians and patients [ 1 ], with positive impacts reported in setting research priorities, developing proposals, recruiting and retaining patients, as well as disseminating results, including societal and ethical benefits [ 2 , 4 , 7 , 9 , 10 , 14 , 28 , 29 , 30 , 31 , 32 , 33 ]. Guidelines and recommendations are emerging on research partnerships with patients, which support the principles of trust, respect, and co-learning [ 2 , 7 , 13 , 18 , 19 , 22 , 34 ].…”

“…Specifically in cancer CTs in Canada, initiatives to engage patients include the Getting better Outcomes with Chimeric Antigen Receptor T-cell therapy (GO-CART) program, which showed that engaging patients in early phase trials helped in each component of the study, including the development of the study protocol, non-technical summary, interview and survey questions, as well as estimating costs for the early economic analysis [ 4 ].…”

“…Despite developments on patient and PG engagement in health research, including the well-known frameworks of Patient-Centered Outcomes Research Institute (PCORI) in the US, INVOLVE in the UK, and Strategy for Patient-Oriented Research (SPOR) in Canada [ 3 , 4 , 6 , 23 , 38 , 39 , 40 ], in practice the prevalence of patient engagement across CTs remains extremely low; less than 1% as reported by Fergusson et al [ 15 ] in a systematic review from May 2011 to June 2016. An unmet need resides in providing tangible tools for CT stakeholders to commit to the principle of patient centricity and patient engagement [ 5 , 41 , 42 ] as well as empowering patients to fulfil their role specifically in cancer CTs.…”