OBJECTIVES: Determining the value of emicizumab (Hemlibra®) compared with Feiba® and Novoseven® in the prophylaxis of haemophilia A patients with inhibitors using MCDA.

METHODS: A Medline search of clinical trials completed with available reference documents. Orphan disease adapted EVIDEM framework (v. 4.0) weighted by 98 Spanish national and regional evaluators was used. Two Evidence Matrixes were scored by a panel of 8 experts: 2 regional and local health managers, 3 hospital pharmacists, 2 haematologists and 1 patients representative. Qualitative criteria were scored in a scale from 1 to 5 and comparative criteria in a scale from -5 to 5. The relative value contribution of emicizumab vs. comparators (Feiba® and Novoseven®) was obtained considering criteria scoring and weighting assigned by the expert panel.

RESULTS: The most valued criteria of emicizumab were relative efficacy and therapeutic benefit. Haemophilia A with inhibitors was perceived as a severe and life conditioning disease (mean and standard deviation: 4.1±0.6) with unmet needs, especially for high inhibitor titres patients with bleeding complications (4±1.3). When compared with Feiba® and Novoseven®, emicizumab had higher therapeutic benefit (4.3±0.7 emicizumab/Feiba®-Novoseven®) with greater relative efficacy (4.4±0.5 emicizumab/Feiba® and 4.3±0.9 emicizumab/Novoseven®) and patient reported outcomes (4±0.8 emicizumab/Feiba® and 3.6±0.7 emicizumab/Novoseven®). Comparative safety and tolerability were perceived as similar for all the alternatives (1.3±2.1 emicizumab/Feiba® and -0.3±0.7 emicizumab/Novoseven®). Quality of evidence (4±0.8) and expert consensus (3.9±0.6) were also positive. System capacity and appropriate use of emicizumab were not considered a problem and, contextual criteria were valued positively.

CONCLUSIONS: Emicizumab adds higher therapeutic value compared to the current treatments for hemofilia A with inhibitors and satisfies important unmet needs such as higher efficacy, better quality of life and a more convenient administration and possible savings derived from less use of resources due to treated bleedings avoided.