COST-MINIMIZATION ANALYSIS OF EFANESOCTOCOG ALFA VERSUS STANDARD HALF-LIFE FVIII CONCENTRATES FOR MAJOR SURGERY IN PATIENTS WITH MILD/MODERATE HEMOPHILIA A WITHOUT SELF-ADMINISTRATION CAPABILITY
Author(s)
Manuel R. Lopez, MD1, Maria Teresa Alvarez Roman, PhD, MD2, Michael Calviño Suarez, MD3, Ramiro Núñez, PhD, MD4, Carmen Albo Lopez, PhD, MD1.
1Hospital Universitario Alvaro Cunqueiro, EOXI VIGO, Vigo, Spain, 2Hospital Universitario La Paz, IdIPaz, Madrid, Madrid, Spain, 3Complejo Hospitalario Universitario A Coruña, A Coruña, Spain, 4Hospital Universitario Virgen del Rocio, Sevilla, Spain.
1Hospital Universitario Alvaro Cunqueiro, EOXI VIGO, Vigo, Spain, 2Hospital Universitario La Paz, IdIPaz, Madrid, Madrid, Spain, 3Complejo Hospitalario Universitario A Coruña, A Coruña, Spain, 4Hospital Universitario Virgen del Rocio, Sevilla, Spain.
OBJECTIVES: To evaluate the total perioperative cost per episode of efanesoctocog alfa (EFA) versus other alternatives (Standard or Extended CFVIII) in persons with mild/moderate hemophilia A on on-demand therapy without self-administration capability undergoing major surgery, from a Spanish hospital formulary perspective.
METHODS: A cost-minimization model anchored on WFH perioperative FVIII targets was developed across two sub-models: A1 (pharmacokinetic-guided dosing) and A2 (per EMA label). Baseline FVIII was simulated at 5%, 10%, 20%, and 30% (reference weight: 80 kg). Unit costs: SIM €0.34/IU; TAP €0.37/IU; EFA €0.78/IU; hospitalization €528.95/day; day-hospital €250/visit. For SHL or EHL-FVIII, 10 inpatient days and 4 post-discharge day-hospital visits were assumed, reflecting 8-12h redosing constraints in non-self-administering patients. For EFA, 7 inpatient days and zero day-hospital visits were modeled, supported by sustained FVIII activity >40% at 96-120h post-dose (XTEND-1 data). A real-world surgical episode (trabeculectomy, mild HA, FVIII:C 27%) validated the IV administration burden (18 doses/8 days). Univariate sensitivity analysis and two-dimensional sensitivity mapping (FVIII baseline × day-hospital cost) were performed. Vial wastage was excluded, yielding conservative estimates.
RESULTS: In the base case (FVIII 5-20%, day-hospital €250/visit), EFA generated €315-€1,315 in episode-level savings versus SHL-FVIII, driven by a €1,587 reduction in inpatient costs and elimination of day-hospital visits (up to €1,000), despite a 2.3-fold higher unit price. The sole unfavorable scenario was FVIII 10% vs EHL-FVIII (+€161); cost-neutrality was achieved at €41/day-hospital visit. Sensitivity analyses confirmed robustness across ±20% hospitalization cost, inpatient days, and ±10% unit costs. Real-world validation showed cost-neutrality with concomitant desmopressin (+€115); without desmopressin, EFA yielded −€1,707.
CONCLUSIONS: In mild/moderate HA persons lacking self-administration capability, EFA reduces or neutralizes total perioperative episode costs versus classical FVIII concentrates across all clinically relevant scenarios, despite a higher unit price. These findings support cost-per-episode as the appropriate metric for hospital formulary decisions in this population.
METHODS: A cost-minimization model anchored on WFH perioperative FVIII targets was developed across two sub-models: A1 (pharmacokinetic-guided dosing) and A2 (per EMA label). Baseline FVIII was simulated at 5%, 10%, 20%, and 30% (reference weight: 80 kg). Unit costs: SIM €0.34/IU; TAP €0.37/IU; EFA €0.78/IU; hospitalization €528.95/day; day-hospital €250/visit. For SHL or EHL-FVIII, 10 inpatient days and 4 post-discharge day-hospital visits were assumed, reflecting 8-12h redosing constraints in non-self-administering patients. For EFA, 7 inpatient days and zero day-hospital visits were modeled, supported by sustained FVIII activity >40% at 96-120h post-dose (XTEND-1 data). A real-world surgical episode (trabeculectomy, mild HA, FVIII:C 27%) validated the IV administration burden (18 doses/8 days). Univariate sensitivity analysis and two-dimensional sensitivity mapping (FVIII baseline × day-hospital cost) were performed. Vial wastage was excluded, yielding conservative estimates.
RESULTS: In the base case (FVIII 5-20%, day-hospital €250/visit), EFA generated €315-€1,315 in episode-level savings versus SHL-FVIII, driven by a €1,587 reduction in inpatient costs and elimination of day-hospital visits (up to €1,000), despite a 2.3-fold higher unit price. The sole unfavorable scenario was FVIII 10% vs EHL-FVIII (+€161); cost-neutrality was achieved at €41/day-hospital visit. Sensitivity analyses confirmed robustness across ±20% hospitalization cost, inpatient days, and ±10% unit costs. Real-world validation showed cost-neutrality with concomitant desmopressin (+€115); without desmopressin, EFA yielded −€1,707.
CONCLUSIONS: In mild/moderate HA persons lacking self-administration capability, EFA reduces or neutralizes total perioperative episode costs versus classical FVIII concentrates across all clinically relevant scenarios, despite a higher unit price. These findings support cost-per-episode as the appropriate metric for hospital formulary decisions in this population.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
MSR33
Topic
Methodological & Statistical Research
Disease
Rare & Orphan Diseases