Value Contribution of Etranacogene Dezaparvovec for the Treatment of Severe and Moderately Severe Haemophilia B in Spain Through Multicriteria Decision Analysis (MCDA)

Speaker(s)

Benítez O1, García-Diego DA2, Jiménez-Yuste V3, Juarez JC4, Núñez R5, Perez-Santamarina R6, Poveda JL7, Trillo JL8, Valles JA9, Badia X10, Blanco M11
1Hospital Universitario Vall d’Hebrón, Barcelona, Spain, 2FedHemo, Madrid, Spain, 3University Hospital La Paz, Madrid, Spain, 4Hospital Unviersitario Vall d'Hebrón, Barcelona, Spain, 5Hospital Universitario Virgen del Rocío, Sevilla, Spain, 6Hospital Universitario La Paz, Madrid, Spain, 7Hospital Universitario La Fe, Valencia, Spain, 8Department of Health of Valencia Clínico-Malvarrosa, Valencia, Valencia, Spain, 9Instituto Catalán de Salud, Barcelona, Spain, 10Omakase Consulting S.L., Barcelona, B, Spain, 11CSL Behring, Barcelona, Spain

Presentation Documents

OBJECTIVES: Determining value of gene therapies requires innovative and holistic approaches to capture full benefit. This study assessed the value contribution of Etranacogene dezaparvovec (ED) versus long-acting recombinant factor IX (LA) for the treatment of severe and moderately severe Haemophilia B (sHB) in Spain using MCDA.

METHODS: MCDA framework for orphan-drug evaluation included nine quantitative and four qualitative criteria. Evidence matrices were developed to compare ED vs the three LA based on a literature review. Multidisciplinary group of twenty-eight experts (haematologists, hospital pharmacists, decision-makers & patient) scored the evidence matrices using an ordinal scale from 0 to + 5 (highest value) for non-comparative criteria and from − 5 to + 5 for comparative criteria. Mean values of the evidence matrices and standard deviation were calculated. Global value contribution was calculated using a standardised scale from -1 to +1 (highest value).

RESULTS: Haemophilia B was considered a severe disease (4.3±0.7) associated with a low life expectancy, increased morbidity, and relevant unmet needs (3,3±0.9). ED was considered a more effective treatment (2.0±1.3), as the percentage of patients who do not return to prophylaxis and had zero bleeding was higher than comparators. Uncertainties regarding safety profile (-1.2±1.8) and long-term efficacy were highlighted. Quality of life was considered superior (1.9±1.5), since 96% remained free from prophylaxis with a single dose. ED was considered to provide savings over time in both direct medical (1.6±2.0) and indirect costs (2.0±1.5) due to potential reduction of hospitalisations and prophylaxis treatment. ED was perceived to have a high therapeutic impact (3.8±0.8) supported by high-quality evidence (3.8±0.9). The global value contribution was 0.45, consistent with other innovative orphan drugs.

CONCLUSIONS: Holistic higher benefit of Entranacogene dezaparvovec (ED) was demonstrated compared to LA for sHB using MCDA.

Code

HTA297

Topic

Health Technology Assessment

Topic Subcategory

Decision & Deliberative Processes, Value Frameworks & Dossier Format

Disease

Personalized & Precision Medicine, Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)