Abstract

Qualitative research with patients generates information on the clinical relevance of treatment effects and increases the validity of subsequent quantitative preference surveys. However, uncertainty exists on how to conduct qualitative research aiming to inform preference surveys. Understanding patient preferences may be extremely valuable in the case of multiple myeloma (MM), where the existence of treatments with different profiles raises uncertainty about the value of these treatments to patients. This study aimed to understand which treatment effects are most important for MM patients. Nominal group discussions with MM patients in Belgium, Romania, Finland and Spain asked patients to reach consensus on treatment effects they find most important when considering treatments. Here, findings from an interim thematic analysis of discussions in Belgium (n=6 patients) and Romania (n=5 patients) are presented. Four themes capturing prioritised treatment effects were identified. First, patients agreed on the importance of the treatment’s ability to increase survival. Second, patients prioritised severe side effects potentially endangering survival such as the risk of developing a new cancer. Third, side-effects limiting patients’ independence such as bone pain and destruction were prioritised. Fourth, patients worried about cognitive and psychological side-effects, negatively impacting their wellbeing and how they interact with others such as memory loss or confusion. Patients highlighted the influence of treatment experience and their societal role (family, job) on their ranking. This study indicates that survival, independence and psychological wellbeing are most important for MM patients in Belgium and Romania. Additionally, this study underlines the importance of considering patients’ personal characteristics when designing and analysing preference surveys. This methodology may guide stakeholders considering the use of qualitative methods and contributes to the debate regarding best practices for qualitative research to develop quantitative preference surveys. This work received support from the EU/EFPIA Innovative Medicines Initiative [2] Joint Undertaking PREFER grant no. 115966.

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