Determining the value contribution of emicizumab (hemlibra®) for the prophylaxis of haemophilia a patients without inhibitors in Spain by multi-criteria decision analysis (MCDA)
Year: 2020
Type: article
Abstract: Objective: 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). Methodology: The EVIDEM framework adapted to orphan drugs and weighted by a sample of 98 national and regional Spanish evaluators was used. Two structured evidence matrices were developed: emicizumab against octocog alfa (OA) and emicizumab against efmoroctocog alfa (EA). A multidisciplinary team of haemophilia experts rated each one of the criteria. Mean and standard deviation was calculated by each criterion and discussed among all participants. Results: 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, 4.2±1.0 emicizumab/EA) with greater patient reported outcomes (4.0±1.2 emicizumab/OA, 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. Conclusions: 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. Keywords: haemophilia A; drug value; multicriteria decision analysis, emicizumab
Source: Economía de la Salud
DOI: 10.61679/1503035044
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: bronze
OpenAlex ID: W4389735376