Journal of medical economics · 2026
Bleeding rates, healthcare utilization, and costs among patients with hemophilia a without inhibitors treated with concomitant octocog alfa or extended half-life factor VIII while on emicizumab prophylaxis.
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Montcrieff Caitlin, Caicedo Jorge, Cerretani Alicia, Fan Angela Qi, Bullano Michael, Ponomareva Ekaterina
Аннотация
AIM: Emicizumab has changed prophylactic treatment for HA, but the need for FVIII replacement still exists for the treatment of breakthrough bleeds, perioperative management, and participation in high-risk activities/sports. The primary objectives of this study were to describe the characteristics of individuals with congenital hemophilia A (HA) without inhibitors receiving emicizumab prophylaxis and to compare clinical outcomes, healthcare resource utilization (HCRU), and costs among those prescribed octocog alfa [Advate, standard half-life factor VIII (FVIII)] versus extended half-life (EHL) FVIII agents.
METHODS: Patients with congenital HA without inhibitors and emicizumab use were identified retrospectively from Inovalon claims data and stratified by concomitant octocog alfa vs. EHL FVIII use. Inverse probability treatment weighting (IPTW) and multivariable regression were used to balance baseline covariates and compare billable annualized rate of bleeding (ABRb), healthcare utilization, and costs between subgroups.
RESULTS: Among the 1,282 patients receiving emicizumab prophylaxis, 501 received concomitant FVIII prescription for octocog alfa (n = 274) or EHL (n = 227) during follow-up. Following IPTW adjustment, total and medical costs were comparable between groups; however, pharmacy costs associated with concomitant octocog alfa therapy were significantly lower compared with EHL products ($35,381 vs. $61,739; p < 0.001). Importantly, ABRb (0.35 vs. 0.28; p = 0.34) and bleed-related healthcare utilization remained similar across cohorts.
LIMITATIONS: Findings are limited by the retrospective design and reliance on claims data, which may omit home-treated bleeds, and misrepresent FVIII utilization due to billing complexities and assumptions about dispensed product use.
CONCLUSIONS: While the choice of concomitant FVIII does not substantially influence ABRb or bleed-related HCRU, significantly lower pharmacy costs with octocog alfa compared with EHL agents highlight its potential cost saving and support its consideration as a preferred on-demand treatment option in patients with HA without inhibitors on emicizumab prophylaxis.
METHODS: Patients with congenital HA without inhibitors and emicizumab use were identified retrospectively from Inovalon claims data and stratified by concomitant octocog alfa vs. EHL FVIII use. Inverse probability treatment weighting (IPTW) and multivariable regression were used to balance baseline covariates and compare billable annualized rate of bleeding (ABRb), healthcare utilization, and costs between subgroups.
RESULTS: Among the 1,282 patients receiving emicizumab prophylaxis, 501 received concomitant FVIII prescription for octocog alfa (n = 274) or EHL (n = 227) during follow-up. Following IPTW adjustment, total and medical costs were comparable between groups; however, pharmacy costs associated with concomitant octocog alfa therapy were significantly lower compared with EHL products ($35,381 vs. $61,739; p < 0.001). Importantly, ABRb (0.35 vs. 0.28; p = 0.34) and bleed-related healthcare utilization remained similar across cohorts.
LIMITATIONS: Findings are limited by the retrospective design and reliance on claims data, which may omit home-treated bleeds, and misrepresent FVIII utilization due to billing complexities and assumptions about dispensed product use.
CONCLUSIONS: While the choice of concomitant FVIII does not substantially influence ABRb or bleed-related HCRU, significantly lower pharmacy costs with octocog alfa compared with EHL agents highlight its potential cost saving and support its consideration as a preferred on-demand treatment option in patients with HA without inhibitors on emicizumab prophylaxis.