Objectives: Stakeholder engagement in health technology assessment is not new; however, little is known about specific processes of engagement during the conduct of the assessment, especially for economic evaluations. Patients and their caregivers have insight that can enhance the scope and validity of an economic model, though are often not engaged in such studies. We sought to address this within a study that involved patients and their caregivers in the conduct of an early economic evaluation of Chimeric Antigen Receptor T-cell therapy for adults with relapsed or refractory B-cell acute lymphoblastic leukemia to inform a more realistic estimate of potential treatment value.

Methods: Online, audio-visual (i.e., Zoom) group discussions involving patients with experience of hematological cancer and their caregivers were conducted using an adapted version of the Nominal Group Technique, a consensus-building discussion approach, to generate focused qualitative data in response to questions specific to the development of the economic model.

Results: The study included eight participants across two stakeholder group discussions (median age 59.5y; 50% female; seven patients and one caregiver). Participants highlighted several costs and benefits for consideration in the early economic evaluation. Patients and their caregivers acknowledged the direct costs to receive 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 highlighted by participants. Treatment benefits prioritized by participants included the efficacy of treatment, manageable and minimal side effects, improved quality of life, accessibility of treatment, and short treatment duration.

Conclusion: This research demonstrates how patient and caregiver engagement can be conducted to improve the usefulness and validity of an early economic evaluation, which could inform broader stakeholder engagement in future similar studies.