OBJECTIVES:

Hemophilia A is a rare disease characterized by uncontrolled and persistent bleeding. The mainstay of treatment in patients with hemophilia A is to monitor patients in accredited blood centers continuously. This study estimated the direct and indirect costs per patient with hemophilia type A in Brazil.

METHODS:

This is applied prospective observational research with retrospective data collection in patients followed at 3 referral blood centers from Brazil. Time-driven Activity-based Costing (TDABC) method was used to guide the data collection and analyses of direct costs. The indirect costs were estimated based on interviews. Patients were stratified in groups of age ranges (0-11;12-18 or older than 19) and, according to inhibitor status for the cost analyses, calculated as the annual cost per patient. The non-parametric Mann-Whitney test was used to confirm the differences in costs between groups. The costs are expressed in Brazilian national currency (R$) in 2022.

RESULTS:

Data from 140 patients were analyzed; 53 are 0-11, 29 are 12-18, and the remaining are older than 19. Only 14% had inhibitors, and most were 0-11 years old (n=12). The median cost per patient per year was R$450,831 (IQR R$219,842; R$785,149), and it was possible to demonstrate that older patients had higher costs (p=0.001; median cost: 0-11yrs - R$299,320; 12-18 yrs-R$521,936; ≥19yrs R$718,969). There were no significative differences between patients with or without inhibitors (p=0.18; median cost: R$702,444 and R$440,752, respectively).

CONCLUSIONS:

This study is innovating by providing cost information for hemophilia type A using a microcosting technique. The variation in costs across patient age ranges and justified by the health care multiprofessional service suggests the importance of considering that in health policies.

This research was financed by the company Produtos Roche Químicos e Farmacêuticos S/A of Brazil. The Institute of Health Technology Assessment was responsible for conducting the study, writing the manuscript and editorial review.