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Traditionally, economic evaluations have engaged clinicians and policymakers; however, patients and their caregivers have insight that can ensure that the economic evaluation process appropriately reflects disease consequences and adequately addresses their priorities related to treatment.We aimed to identify patient priorities to inform an early economic evaluation of chimeric antigen receptor T-cell therapy for adults with relapsed or refractory B-cell acute lymphoblastic leukemia.We conducted two online group discussions of four participants each, involving patients with experience of hematological cancer and a caregiver. We used an adapted version of the nominal group technique, a consensus-building discussion approach, to generate focused qualitative data.Patients and a caregiver acknowledged both the costs directly related to clinical care, such as the out-of-pocket cost of drugs, and the indirect treatment costs, such as the cost of transport, accommodation, and food. The emotional and physical toll of treatment and the influence of treatment on employment and education were additional costs emphasized by participants. Treatment benefits prioritized by participants included the efficacy of treatment, manageable side effects, improved quality of life, accessibility of treatment, and short treatment duration.Engaging patients and caregivers in an early economic evaluation could help identify additional costs and benefits of therapies that are not typically recognized in economic evaluations but have the potential to increase the commercial viability of novel therapies. This research also demonstrates how patients and caregivers can be engaged at different levels in the development of early economic evaluation models.

IntroductionChimeric antigen receptor T-cell (CAR-T) therapy is a class of immunotherapy. An economic evaluation conducted at an early stage of development of CAR-T therapy for treatment of adult relapsed or refractory acute lymphoblastic leukaemia could provide insight into factors contributing to the cost of treatment, the potential clinical benefits, and what the health system can afford. Traditionally, stakeholders are engaged in certain parts of health technology assessment processes, such as in the identification and selection of technologies, formulation of recommendations, and implementation of recommendations; however, little is known about processes for stakeholder engagement during the conduct of the assessment. This is especially the case for economic evaluations. Stakeholders, such as clinicians, policy-makers, patients, and their support networks, have insight into factors that can enhance the validity of an economic evaluation model. This research outlines a specific methodology for stakeholder engagement and represents an avenue to enhance health economic evaluations and support the use of these models to inform decision making for resource allocation. This protocol may inform a tailored framework for stakeholder engagement processes in future economic evaluation model development.Methods and analysisWe will involve clinicians, healthcare researchers, payers, and policy-makers, as well as patients and their support networks in the conduct and verification of an early economic evaluation of a novel health technology to incorporate stakeholder-generated knowledge. Three stakeholder-specific focus groups will be conducted using an online adaptation of the nominal group technique to elicit considerations from each. This study will use CAR-T therapy for adults with relapsed or refractory B-cell acute lymphoblastic leukaemia as a basis for investigating broader stakeholder engagement processes.Ethics and disseminationThis study received ethics approval from the Ottawa Hospital Research Institute Research Ethics Board (REB 20200320-01HT) and the results will be shared via conference presentations, peer-reviewed publications, and ongoing stakeholder engagement.

ObjectivesBench to bedside translation of groundbreaking treatments like chimeric antigen receptor T (CAR-T) cell therapy depends on patient participation in early phase trials. Unfortunately, many novel therapies fail to be adequately evaluated due to low recruitment rates, which slows patient access to emerging treatments. Using the Theoretical Domains Framework (TDF), we sought to identify potential patient barriers and enablers to participating in an early phase CAR-T cell therapy trial.DesignWe used qualitative semistructured interviews to identify potential barriers and enablers to patients’ hypothetical participation in an early phase CAR-T cell therapy trial. We used the TDF and directed content analysis to identify relevant domains based on frequency, relevance and the presence of conflicting beliefs.ParticipantsCanadian adult patients diagnosed with haematological malignancies.ResultsIn total, we interviewed 13 participants (8 women, 5 men). Participants ranged in age from 18 to 73 (median=56) and had been living with haematological cancer from a few months to several years. We found participants were unfamiliar with CAR-T cell therapy but wished to know more about treatment safety, efficacy and trial logistics (domains: knowledge, beliefs about consequences). They were motivated by altruistic considerations, though many prioritised personal health benefits despite recognising the goals (ie, establishing safety) of early phase clinical trials (domains: goals, intentions). Every participant valued receiving medical advice from their haematologists and oncologists, though some preferred impartial medical experts to inform their decision making (domain: social influences). Finally, participants indicated that improving access to financial and social supports would improve their trial participation experience (domain: environmental context and resources).ConclusionUsing the TDF allowed us to identify factors that might undermine participation to a CAR-T cell therapy trial and to optimise recruitment processes by considering patient perspectives to taking part in early phase trials.Trial regestration:NCT03765177; Pre-results.

Patients want personalized information before surgery; most do not receive personalized risk estimates. Inadequate information contributes to poor experience and medicolegal complaints. We hypothesized that exposure to the Personalized Risk Evaluation and Decision Making in Preoperative Clinical Assessment (PREDICT) app, a personalized risk communication tool, would improve patient knowledge and satisfaction after anesthesiology consultations compared with standard care.We conducted a prospective clinical study (before-after design) and used patient-reported data to calculate personalized risks of morbidity, mortality, and expected length of stay using a locally calibrated National Surgical Quality Improvement Program risk calculator embedded in the PREDICT app. In the standard care (before) phase, the application's materials and output were not available to participants; in the PREDICT app (after) phase, personalized risks were communicated. Our primary outcome was knowledge score after the anesthesiology consultation. Secondary outcomes included patient satisfaction, anxiety, feasibility, and acceptability.We included 183 participants (90 before; 93 after). Compared with standard care phase, the PREDICT app phase had higher post-consultation: knowledge of risks (14.3% higher; 95% confidence interval [CI], 6.5 to 22.0; P0.001) and satisfaction (0.8 points; 95% CI, 0.1 to 1.4; P = 0.03). Anxiety was unchanged (- 1.9%; 95% CI, - 4.2 to 0.5; P = 0.13). Acceptability was high for patients and anesthesiologists.Exposure to a patient-facing, personalized risk communication app improved knowledge of personalized risk and increased satisfaction for adults before elective inpatient surgery.www.clinicaltrials.gov (NCT03422133); registered 5 February 2018.RéSUMé: OBJECTIF: Les patients veulent disposer d’informations personnalisées avant leur chirurgie, mais la plupart d’entre eux ne reçoivent pas d’estimations de leur risque personnalisées. Des informations inadéquates contribuent à une mauvaise expérience et à des plaintes médicolégales. Nous avons émis l’hypothèse qu’une exposition à l’application PREDICT (Personalized Risk Evaluation and Decision Making in Preoperative Clinical Assessment), un outil de communication du risque personnalisé, améliorerait les connaissances et la satisfaction des patients après leurs consultations en anesthésiologie comparativement à des soins standard. MéTHODE: Nous avons réalisé une étude clinique prospective (de type avant-après) et utilisé les données rapportées par les patients afin de calculer leur risque personnalisé de morbidité et de mortalité, ainsi que la durée de séjour anticipée à l’aide d’un calculateur de risque tiré du Programme national d’amélioration de la qualité chirurgicale que nous avons calibré localement et intégré à l’application PREDICT. Dans la phase de soins standard (avant), le contenu et les résultats de l’application n’étaient pas divulgués aux participants; dans la phase comportant l’application PREDICT (après), les risques personnalisés étaient communiqués. Notre critère d’évaluation principal était le score des connaissances des patients après la consultation en anesthésiologie. Les critères d’évaluation secondaires comprenaient la satisfaction des patients et leur niveau d’anxiété ainsi que la faisabilité et l’acceptabilité d’une telle approche. RéSULTATS: Nous avons inclus 183 participants (90 avant; 93 après). Comparativement à la phase de soins standard, la phase avec l’application PREDICT a démontré un niveau plus élevé de connaissances des risques post consultation (14,3 % plus élevé; intervalle de confiance [IC] 95 %, 6,5 à 22,0; P0,001) et de satisfaction (0,8 point; IC 95 %, 0,1 à 1,4; P = 0,03). L’anxiété est demeurée inchangée (− 1,9 %; IC 95 %, − 4,2 à 0,5; P = 0,13). L’acceptabilité était élevée, tant chez les patients que chez les anesthésiologistes. CONCLUSION: L’exposition des patients à une application de communication du risque personnalisé a amélioré leurs connaissances de leur risque personnalisé et augmenté la satisfaction des adultes avant une chirurgie non urgente et non ambulatoire. ENREGISTREMENT DE L’éTUDE: www.clinicaltrials.gov (NCT03422133); enregistrée le 5 février 2018.