OBJECTIVES: The purpose of this study was to map the care journeys of patients with MPS2 through the Value Care Journey for Patients with Rare Diseases (JAV-RARAS), a prospective nationwide cohort investigation that employs the Time-Driven Activity-Based Costing (TDABC) methodology to evaluate the processes and costs associated with the management of rare diseases in several regions of Brazil (Midwest, North, Southeast, and South) and compare them with the Clinical Protocol and Therapeutic Guidelines (PCDT) established by the Ministry of Health.

METHODS: The study was conducted in seven medical centers that provide services to the Unified Health System (SUS), and the study identified the real annual costs of managing patients with MPS2. This was achieved through interviews with health professionals and an analysis of administrative and care processes in the participating centers. The cost defined by the Clinical Protocol and Therapeutic Guidelines (PCDT) was calculated based on the definitions contained within it, allowing for a direct comparison with current clinical practices.

RESULTS: The preliminary results indicate that, in actual practice, the national average annual cost was US$17,022.29, while the cost based on the Clinical Protocol and Therapeutic Guidelines (PCDT) was US$15,034.90. Significant differences in costs were observed between the treatments proposed in the PCDT and those implemented in real life. Specifically, the average cost estimated in the PCDT was US$14,061.11, while the actual cost implemented by the care institutions was US$16,226.93.

CONCLUSIONS: The preliminary cost analysis revealed that the current clinical practice in medical centers is more expensive than that defined in the PCDT for the treatment of MPS2. The observed discrepancy in costs suggests that the specific needs of patients may not be fully addressed by the existing PCDT.