OBJECTIVES:

Hemophilia is rare genetic disorder affecting blood’s ability to clot due to clotting factor deficiency. Hemophilia A (HA), the most common form of Hemophilia, is due to factor VIII (FVIII) deficiency. HA treatment options include plasma-derived or recombinant-FVIII concentrates. Approximately 30% of HA patients develop inhibitors against FVIII concentrates. Patients with inhibitors are usually managed using bypassing agents, e.g., Anti-Inhibitor Coagulant Complex (ACC) or recombinant human coagulation Factor VIIa. (rFVIIa). In 2016, the Kingdom of Saudi Arabia (KSA) reported 31 pediatric HA inhibitor patients, among whom 26 patients were treated in the ministry of health (MOH) facilities. To assess the budget impact (BI) of switching MOH pediatric HA patients with inhibitors from-rFVIIa to ACC both as prophylaxis and an on-demand therapy.

METHODS:

A global BI excel model was adapted from payer’s perspective. Model inputs were retrieved from literature and validated by key experts through face-to-face interviews. The model was adapted for two scenarios: Switching patients from (1) rFVIIa prophylaxis therapy to ACC prophylaxis therapy and (2) rFVIIa on-demand therapy to ACC on-demand therapy. The model considered direct costs such as drug acquisition, drug administration and bleeding episodes management. A sensitivity analysis was performed to assess the robustness of the results.

RESULTS:

The BI analysis showed that switching patients from rFVIIa prophylaxis to ACC prophylaxis would result in three times (-112 million Saudi Riyal [SAR]) and eight times (-418 million SAR) savings in the annual payers’ budget using rFVIIa standard dose and megadose. Similarly, switching patients from rFVIIa on-demand to ACC on-demand therapy would result in approximately 1.5 times (-15 million SAR) savings in the payers’ budget.

CONCLUSIONS:

Switching pediatric HA patients with inhibitors from rFVIIa to ACC as prophylaxis and on-demand therapies could be cost saving options for KSA payers thereby freeing more resources for other diseases management.