Presentation (CTI)

OBJECTIVES: Qualitative research can inform healthcare decision making by assessing treatment acceptability and value, understanding lived experiences, collecting data from hard-to-reach groups, and supplementing quantitative research. Interest in how extensively qualitative research is used in healthcare decision making, specifically in health technology assessment (HTA) is expanding. This review aimed to characterize the contemporary use of patient- or caregiver-centered qualitative research by sponsors and patient groups in Canada’s Drug Agency (CDA) reimbursement reviews.
METHODS: Sponsored reimbursement reviews of non-oncology pharmaceuticals for rare disease (conditions affecting <1/2,000 as listed on orpha.net), with recommendations issued in 2024 were accessed. Qualitative research use in the clinical and pharmacoeconomic submissions, and in patient group input(s), were extracted and synthesized.
RESULTS: Ten reimbursement reviews were identified. Two sponsors included de novo patient- or caregiver-centered qualitative research in their submissions (20%). These qualitative studies were used to understand perspectives and provide context (n=2), assess treatment acceptability and subjective value (n=1), reach groups other methods cannot reach (n=1), inform subsequent quantitative exercises (n=1), and contribute to economic model development (n=1). In all reviews, patient groups provided input, eight of which included qualitative data from patients/caregivers. Data presented included quotes from interviews, social media posts, and free-text responses in surveys. However, there was little methodological information provided to assess the quality of these data collection methods and no indication that qualitative analysis methods were applied.
CONCLUSIONS: Few sponsors submitted patient- or caregiver-centred qualitative evidence; however, CDA received qualitative data from patient groups to consider for most submissions. Limited inclusion of qualitative research by sponsors may be explained by the absence of clear guidance from decisionmakers on its use and benefits to HTA. While qualitative data submitted by patient groups provide important insight into patient priorities and values, these data could be strengthened with the inclusion of greater methodological detail.