OBJECTIVES: With the recent (conditional) EU authorization of several gene therapies for hemophilia A and B, the treatment landscape for people with hemophilia (PWH) has expanded with potentially curative gene therapies. Some countries, such as Germany, the UK, and Austria, have agreed on pricing and reimbursement. However, health technology assessment remains challenging due to uncertainties about long-term efficacy, and many countries have yet to commercialize these therapies. Understanding patient preferences can aid in decision-making by demonstrating the value of these gene therapies despite uncertainties. The Patient Preferences to Assess Value IN Gene Therapies (PAVING) II study, in continuation of the original PAVING study, aims to determine if preferences between prophylactic factor replacement therapy (PFRT) and gene therapy have changed following the approval of several gene therapies and successful treatment of patients.

METHODS: Using a similar two-phase approach as PAVING, the first qualitative phase will involve semi-structured interviews with adult Belgian PWH types A and B to determine their willingness to receive gene therapies. Updated with real-world data, the interview will include open-ended questions, attribute ranking, and case scenarios. This phase will validate or modify previously patient-relevant attributes (i.e. annual bleeding rate, factor levels, uncertainty of long-term risks, daily life impact, possibility of stopping prophylaxis) and data will be analyzed using Nvivo and framework analysis. In the second quantitative phase, an online survey with an integrated educational tool will be distributed among EU PWH. This survey, informed by the first phase's attributes, will explore trade-offs between PFRT and gene therapy and quantify the minimum acceptable benefit (MAB) using the threshold technique. Data will be analyzed with Stata based on interval regression models.

RESULTS: Results will be presented at the conference.

CONCLUSIONS: These outcomes could refine clinical or payer-initiated trials, inform managed entry agreements and facilitate evaluations of gene therapies for hemophilia.