WHAT ARE THE PERCEPTIONS AND MOST RELEVANT TREATMENT OUTCOMES REGARDING GENE THERAPY ACCORDING TO PEOPLE WITH HEMOPHILIAS IN BRAZIL? STUDY PROTOCOL FOR A QUALITATIVE PATIENT PREFERENCE STUDY

Gene therapies for hemophilias present a new reality for patients and their families. Gaining a better understanding of patient perceptions regarding these therapies may provide insights regarding their willingness to accept them. Moreover, as gene therapies will likely be soon available for hemophilias, health technology assessment decision-making needs to be tailored towards patients'preferences and needs. To describe a study protocol aimed at understanding patient preferences among people with hemophilias, and their opinions and concerns regarding gene therapies. The protocol for this qualitative study was designed following the general steps described in the PAVING Study (Patient Preferences to Assess Value IN Gene Therapies in Hemophilia). The protocol consisted of (i) a target literature review and (ii) patient relevant information needs and (treatment) attributes identification and ranking. A target literature review of attributes and previous published preference studies will be performed. Participants will be recruited through purposive sampling. Relevance of attributes will be investigated in online one-to one interviews with up to 20 people with hemophilia A and B. An interview guide for semi-structured interviews was designed. Prior to any questions about gene therapy, participants will receive information regarding the disease, standard of care and gene therapy using an educational tool validated in previous studies to ensure comprehension of the attributes and the gene therapy context. We will translate the educational tool and pilot them with Brazilian participants. Interviews will also cover opinions and concerns of people with hemophilias regarding gene therapies. The selected attributes (from the literature review and interviews) will be included in an exercise, in which participants will rank their top six attributes. Data from answers to open questions will be organized using NVivo and analyzed following framework analysis. Data saturation will be assessed by a saturation table and a documented codebook. Details on the methods and results of the interviews will be reported according to the consolidated criteria for reporting qualitative research (COREQ) checklist. Results are expected to be obtained and presented in conferences and scientific peer-reviewed journals by the end of 2024. This protocol describes the methodological steps to qualitatively elicit and study patient preferences and perspectives in hemophilias. These findings will inform future quantitative patient preference studies (discrete choice and best-worst scaling experiments). Results will provide evidence on patient preferences regarding alternatives of care for hemophilias in Brazil. Patient preferences should be considered for decision making related to individual care and to reimbursement and coverage decisions in health systems, along with traditional cost-effectiveness and budget impact analysis. All authors contributed to the abstract conception and design. All authors read and approved the final manuscript.