OBJECTIVES:This study aims to quantify the expenses associated with treating Familial Amyloidotic Polyneuropathy (FAP) as part of the JAV-RARAS initiative, which is a component of the National Rare Diseases Network (RARAS) project. The overarching goal of JAV-RARAS is to assess the value of the care journey for patients in Brazil.

METHODS: The Clinical Protocol and Therapeutic Guidelines (PCDT) served as the basis for analyzing Time-Driven Activity-Based Costing (TDABC). Costs were collected through interviews and records of administrative data from nursing and health professionals at health centers in the Southeast and Northeast regions of the country. Additionally, the study included the analysis of direct costs, identification of activities within the PAF (Procedure, Activities, and Functions) framework, resource allocation, cost per unit of time, and measurement of activity duration.

RESULTS: On average, the annual cost for a patient undergoing FAP treatment is R$198,853.14, primarily allocated to medication expenses. The cost breakdown includes human resources (R$253.20), materials (R$489.54), medicines (R$197,240.40), and exams (R$870.00). The entire annual expenditure is dedicated exclusively to treating the disease. Regarding the origin of resources, approximately 99% is sourced from treatment centers, while the remaining 1% is funded by the Unified Health System (SUS) in other locations.

CONCLUSIONS: This study emphasizes the importance of gathering and analyzing costs and access to therapeutic interventions in the treatment of FAP. The largest portion of annual expenses is attributed to medications, emphasizing the need for a more comprehensive understanding of the factors influencing these costs. Exploring more effective therapeutic alternatives holds the potential to lower long-term costs and enhance patients' clinical outcomes.