LONG-TERM ECONOMIC IMPACT OF GENE THERAPY ETRANACOGENE DEZAPARVOVEC VERSUS EXTENDED HALF-LIFE FACTOR IX THERAPIES FOR SEVERE OR MODERATELY SEVERE HAEMOPHILIA B IN SPAIN
Author(s)
Manuel Rodriguez Lopez, MD1, Maria Teresa Alvarez Roman, MD PhD2, José Mateo, MD, PhD3, Juan Megías-Vericat, PhD PhamD4, Juan Carlos Juarez, PhD PhamD5, Belén Espinos, MSc (Econ)6, Miquel Sastre, BSc7, Monica Serra, MSc7.
1Department of Hematology and Hemotherapy, Hospital Universitario Álvaro Cunqueiro, Galicia Sur Health Research Institute, Vigo, Spain, 2Department of Hematology and Hemotherapy, Hospital Universitario La Paz, IdIPaz, Madrid, Madrid, Spain, 3Thrombosis and Hemostasis Unit, Hemathology Department, Hospital de La Santa Creu I Sant Pau, Barcelona, Spain, 4Pharmacy Department, Hospital Universitari i Politècnic La Fe, Valencia, Spain, 5Hospital Pharmacy Department, Hospital Universitari Vall d'Hebron, Barcelona, Spain, 6CSL Iberia, Barcelona, Spain, 7Market Access and Healthcare Consulting, Cencora Spain, Barcelona, Spain.
1Department of Hematology and Hemotherapy, Hospital Universitario Álvaro Cunqueiro, Galicia Sur Health Research Institute, Vigo, Spain, 2Department of Hematology and Hemotherapy, Hospital Universitario La Paz, IdIPaz, Madrid, Madrid, Spain, 3Thrombosis and Hemostasis Unit, Hemathology Department, Hospital de La Santa Creu I Sant Pau, Barcelona, Spain, 4Pharmacy Department, Hospital Universitari i Politècnic La Fe, Valencia, Spain, 5Hospital Pharmacy Department, Hospital Universitari Vall d'Hebron, Barcelona, Spain, 6CSL Iberia, Barcelona, Spain, 7Market Access and Healthcare Consulting, Cencora Spain, Barcelona, Spain.
OBJECTIVES: To compare the economic impact of etranacogene dezaparvovec (EDZ) with extended half-life (EHL) factor IX (FIX) prophylaxis for haemophilia B patients in Spain.
METHODS: A cost-offset model was developed comparing two hypothetical patient cohorts (n=20), receiving EDZ or EHL FIX prophylaxis over a 20-year time horizon. EHL FIX prophylaxis assumed a weighted average of Alprolix®, Refixia® and Idelvion® based on Spanish utilization data. Clinical inputs included long-term efficacy (FIX activity ≥5%) and annual bleeding rate (ABR). Maintenance of FIX≥5% over time following EDZ was derived from a Bayesian non-linear model based on HOPE-B 5-year data, the EHL FIX prophylaxis cohort was assumed to maintain FIX activity≥5% over the time horizon. ABRs for the first 5 years were obtained from HOPE-B 5-year data for EDZ and indirect treatment comparison for EHL FIX prophylaxis after which ABR was assumed equal. Breakthrough bleeds incurred on demand FIX therapy and EDZ patients FIX <5% returned to EHL FIX prophylaxis. Drug costs used local list prices. EDZ costs incorporated the 5-year outcomes-based payment agreed in Spain. Annual and cumulative treatment costs were reported for each cohort.
RESULTS: EDZ resulted in higher initial treatment cost; however, cost neutrality was achieved in years 7-8, after which EDZ generated savings. EDZ prevented 193 bleeding events, providing savings of €994,539 in this conservative analysis. Over 20 years, EDZ resulted in lower cumulative treatment costs versus EHL FIX prophylaxis, generating total savings of €50.51 million (€57.46 million vs €107.96 million) driven by sustained FIX activity and nearly eliminating the need for FIX prophylaxis (91% of patients FIX ≥5% at 20 years).
CONCLUSIONS: EDZ is expected to deliver substantial long-term economic benefits versus EHL FIX prophylaxis in the treatment of haemophilia B in Spain. Assuming sustained long-term efficacy, EDZ could generate significant savings through offsetting prophylaxis costs and bleed reduction.
METHODS: A cost-offset model was developed comparing two hypothetical patient cohorts (n=20), receiving EDZ or EHL FIX prophylaxis over a 20-year time horizon. EHL FIX prophylaxis assumed a weighted average of Alprolix®, Refixia® and Idelvion® based on Spanish utilization data. Clinical inputs included long-term efficacy (FIX activity ≥5%) and annual bleeding rate (ABR). Maintenance of FIX≥5% over time following EDZ was derived from a Bayesian non-linear model based on HOPE-B 5-year data, the EHL FIX prophylaxis cohort was assumed to maintain FIX activity≥5% over the time horizon. ABRs for the first 5 years were obtained from HOPE-B 5-year data for EDZ and indirect treatment comparison for EHL FIX prophylaxis after which ABR was assumed equal. Breakthrough bleeds incurred on demand FIX therapy and EDZ patients FIX <5% returned to EHL FIX prophylaxis. Drug costs used local list prices. EDZ costs incorporated the 5-year outcomes-based payment agreed in Spain. Annual and cumulative treatment costs were reported for each cohort.
RESULTS: EDZ resulted in higher initial treatment cost; however, cost neutrality was achieved in years 7-8, after which EDZ generated savings. EDZ prevented 193 bleeding events, providing savings of €994,539 in this conservative analysis. Over 20 years, EDZ resulted in lower cumulative treatment costs versus EHL FIX prophylaxis, generating total savings of €50.51 million (€57.46 million vs €107.96 million) driven by sustained FIX activity and nearly eliminating the need for FIX prophylaxis (91% of patients FIX ≥5% at 20 years).
CONCLUSIONS: EDZ is expected to deliver substantial long-term economic benefits versus EHL FIX prophylaxis in the treatment of haemophilia B in Spain. Assuming sustained long-term efficacy, EDZ could generate significant savings through offsetting prophylaxis costs and bleed reduction.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE250
Topic
Economic Evaluation, Health Technology Assessment
Topic Subcategory
Budget Impact Analysis, Cost/Cost of Illness/Resource Use Studies
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)