Pro104 Determining the Value of Emicizumab for the Prophylaxis of Severe Haemophilia a Patients Without Inhibitors in Spain by Multi-Criteria Decision Analysis (Mcda)

Abstract

Maverex limited, Manchester, UK Objectives: There is growing recognition that the patient perspective is fundamental in assessing the value of new treatments in health technology assessment (HTA). This is particularly relevant for orphan drugs (ODs) as there is often limited evidence describing disease burden and unmet need, and the impact of treatment on patients' day-today lives may not be fully captured in clinical trials. This study reviewed how patient input was provided and how it influenced reimbursement decisions in recent HTAs of ODs from countries that commonly include patient input as part of their HTA process. Methods: We searched HTA agency websites