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

Determining the value of emicizumab compared to octocog alfa (OA) and efmoroctocog alfa (EA) in the prophylaxis of severe haemophilia A (HA) patients without inhibitors using Multi-Criteria Decision Analysis (MCDA). Literature review of available evidence completed with reference documents (national, regional and hospital evaluations and clinical guidelines). Rare diseases adapted EVIDEM framework weighted by 98 Spanish national and regional evaluators was used. Two evidence matrixes were scored by a panel of 9 multidisciplinary experts in HA: 4 hospital pharmacists, 2 haematologists, 1 local health manager, 1 patients’ representative and 1 nurse. Quantitative criteria were scored in a scale from 0 to 5, comparative criteria from -5 to 5 and qualitative criteria according to emicizumab’s “positive”, “negative” or “no” impact on these criteria. The relative value contribution of emicizumab vs. OA and EA was obtained considering criteria scoring assigned by the expert panel. Relative-quality of life and therapeutic benefit were the most valued criteria for emicizumab. HA without inhibitors was perceived as a serious disease (4.3±0.5) with unmet needs, especially for severe HA patients with higher probability of FVIII inhibitors development (3.3±0.9). When compared with OA and EA, emicizumab had higher therapeutic benefit (4.3±0.9 emicizumab/OA and 4.2±1.0 emicizumab/EA) with greater patient reported outcomes (4.0±1.2 emicizumab/OA and 4.3±0.9 emicizumab/EA) and relative efficacy (3.7±1.6 emicizumab/OA and 3.3±1.4 emicizumab/EA). Comparative safety and tolerability were perceived as similar for all the alternatives (2.1±2.1 emicizumab/OA and 1.6±1.5 emicizumab/EA). Quality of evidence (3.9±0.9 emicizumab/OA and 3.8±0.8 emicizumab/EA) and expert consensus (3.0±1.3) were also positive. All contextual criteria were valued positively for emicizumab. Emicizumab adds higher therapeutic value and improves patients’ quality of life compared to the current prophylaxis agents for severe HA without inhibitors and satisfies important unmet needs such as higher efficacy, improved posology and subcutaneous administration that might improve patients’ adherence.