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

Research Involvement and Engagement

Robert A Holt¹  Harold Atkins²  Joshua Montroy³  Madison Foster³  Sarah Asad³  Grace Fox³  Gisell Castillo³  Natasha Kekre⁴  Manoj M Lalu⁵  Zarah Monfaredi⁶  Dean A Fergusson⁷  Kednapa Thavorn⁸  Justin Presseau⁹  Terry Hawrysh^1,10  Stuart Schwartz^1,10 

[1] BC Cancer Genome Sciences Centre, Canada’s Michael Smith Genome Sciences Centre, 570 W 7th Ave, V5Z 4S6, Vancouver, BC, Canada;Blood and Marrow Transplant Program, The Ottawa Hospital, 501 Smyth Road, K1H 8L6, Ottawa, ON, Canada;Clinical Epidemiology Program, Blueprint Translational Research Group, Ottawa Hospital Research Institute, 501 Smyth Road, K1H 8L6, Ottawa, ON, Canada;Clinical Epidemiology Program, Blueprint Translational Research Group, Ottawa Hospital Research Institute, 501 Smyth Road, K1H 8L6, Ottawa, ON, Canada;Blood and Marrow Transplant Program, The Ottawa Hospital, 501 Smyth Road, K1H 8L6, Ottawa, ON, Canada;Clinical Epidemiology Program, Blueprint Translational Research Group, Ottawa Hospital Research Institute, 501 Smyth Road, K1H 8L6, Ottawa, ON, Canada;Department of Anesthesiology, The Ottawa Hospital, 501 Smyth Road, K1H 8L6, Ottawa, ON, Canada;Department of Anesthesiology and Pain Medicine, University of Ottawa, 501 Smyth Road, K1H 8L6, Ottawa, ON, Canada;Clinical Epidemiology Program, Blueprint Translational Research Group, Ottawa Hospital Research Institute, 501 Smyth Road, K1H 8L6, Ottawa, ON, Canada;SPOR Program Facilitator, Ottawa Methods Centre, Ottawa Hospital Research Institute, 501 Smyth Road, K1H 8L6, Ottawa, ON, Canada;Clinical Epidemiology Program, Blueprint Translational Research Group, Ottawa Hospital Research Institute, 501 Smyth Road, K1H 8L6, Ottawa, ON, Canada;School of Epidemiology and Public Health, University of Ottawa, 451 Smyth Road, K1H 8M5, Ottawa, ON, Canada;Clinical Epidemiology Program, Blueprint Translational Research Group, Ottawa Hospital Research Institute, 501 Smyth Road, K1H 8L6, Ottawa, ON, Canada;School of Epidemiology and Public Health, University of Ottawa, 451 Smyth Road, K1H 8M5, Ottawa, ON, Canada;Institute for Clinical and Evaluative Sciences, ICES uOttawa, 1053 Carling Ave, K1Y 4E9, Ottawa, ON, Canada;Clinical Epidemiology Program, Blueprint Translational Research Group, Ottawa Hospital Research Institute, 501 Smyth Road, K1H 8L6, Ottawa, ON, Canada;School of Epidemiology and Public Health, University of Ottawa, 451 Smyth Road, K1H 8M5, Ottawa, ON, Canada;School of Psychology, University of Ottawa, 136 Jean-Jacques Lussier, Vanier Hall, K1N 6N5, Ottawa, ON, Canada;Patient Partner, Ottawa, ON, Canada;

关键词: Patient partners;    Patient engagement;    Patient and public involvement;    PPI;    Clinical trial;   

DOI  :  10.1186/s40900-020-00230-5

来源: Springer

【 摘 要 】

AimThough 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).MethodsOur 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.ResultsEngaging 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.ConclusionsContributions 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 registrationAffiliated with the development of NCT03765177.

【 授权许可】

CC BY