Cost of prophylaxis versus on-demand treatment for paediatric haemophilia at a Moroccan referral centre: a bottom-up cost analysis

In low- and middle-income countries, the main barrier to haemophilia prophylaxis is the price of clotting factor concentrate rather than clinical knowledge. Morocco issued its first National Haemophilia Management Guide in 2023, but no local cost estimate exists to inform how far prophylaxis can be extended. We estimated the direct treatment cost of prophylaxis and on-demand treatment for a paediatric patient at a single national referral centre, using Moroccan tariffs. We used a bottom-up basket-of-care model for a reference patient (severe haemophilia A, no inhibitors, no established arthropathy; base-case weight 15 kg, ~ 3–4 years). Nine direct medical categories were priced from the 2023 Guide and national tariff schedules (NABM; NGAP), with factor concentrate priced from Ibn Sina Hospital pharmacy procurement records reflecting actual public-sector acquisition prices (FVIII US$0.50/IU, FIX US$0.70/IU; 1 USD = 10 MAD, 2024). The primary perspective was the healthcare system; a limited societal extension added parental productivity loss. A one-way (univariate) deterministic sensitivity analysis varied one input at a time. Scenario analyses covered heavier weights (30 kg, 50 kg) and haemophilia B. For the 15-kg patient, annual direct medical cost was US$20,136 (prophylaxis) versus US$10,092 (on-demand). Factor concentrate represented 89% of the prophylaxis basket and 59% of the on-demand basket. A 20% change in factor price or utilisation changed annual prophylaxis cost by 17.9%; no other single parameter changed it by more than 0.5%. Cost rose steeply with weight (annual prophylaxis US$20,136, US$38,136, and US$62,136 at 15, 30, and 50 kg). Haemophilia B prophylaxis cost about 87% more than haemophilia A. Providing prophylaxis to all 34 children would raise the centre’s annual cost from US$523,920 to US$684,624. At this centre, prophylaxis costs roughly twice as much per year as on-demand treatment, and almost all of the difference is factor concentrate; procurement price is therefore the dominant lever on affordability. These are short-term annual treatment costs, not a lifetime valuation, and do not capture the joint disease that on-demand treatment allows to accumulate, which is addressed in a companion cost-effectiveness analysis. Prophylaxis remains the standard of care where resources permit.