JAV-RARAS: Value-Driven Care Journey in Patients with Duchenne Muscular Dystrophy: A Time-Driven Activity-Based Costing Study at the Bahiana School of Medicine and Public Health
Author(s)
Camila Azevedo, MBA1, Marcelo E. Nita, MSc, PhD, MD1, Myrianne Barbosa, MSc1, Luana Lopes, MSc1, Thiago Godoy, MSc1, Marilaine Ferreira, MSc, PhD2, Marcela Costa, PhD, MD2, Claudia Lorea, MSc, MD3, Temis Felix, MSc, PhD, MD3, RARAS Study Group, MD3;
1MAPE Solutions, São Paulo, Brazil, 2Bahiana School of Public Health, Salvador, Brazil, 3Porto Alegre Clinical Hospital, Porto Alegre, Brazil
1MAPE Solutions, São Paulo, Brazil, 2Bahiana School of Public Health, Salvador, Brazil, 3Porto Alegre Clinical Hospital, Porto Alegre, Brazil
OBJECTIVES: The JAV-RARAS project, part of the National Rare Diseases Network, analyzes the Value-Driven Care Journey of patients with rare diseases in Brazil. Its aim is to document these conditions, establish value parameters, and evaluate cost-effectiveness to optimize resources. The Bahiana School of Medicine and Public Health (EBMSP) participates with a focus on five rare diseases, including Duchenne Muscular Dystrophy (DMD), quantifying and describing the costs associated with the care journey of patients with DMD using the TDABC methodology.
METHODS: The research using the TDABC methodology collects data on diagnosis, treatment, and follow-up of patients in the JAV-RARAS study, mapping the care journey in 21 rare diseases. Direct costs were obtained through interviews and analysis of healthcare professionals' records, focusing on Duchenne Muscular Dystrophy (DMD). The analysis includes identification of activities, resource allocation, calculation of costs per unit, and measurement of activity duration.
RESULTS: The average annual cost for a patient undergoing DMD treatment is $ 79,010.57, with most expenses concentrated on medications. The cost distribution is as follows: human resources $ 253.38, equipment $ 798.00, medications $ 77,724.70, and exams $ 234.49. According to the data analysis, most of the annual investment is allocated to disease treatment. The source of funding comes from judicialization, accounting for approximately 98% of total resources. In contrast, only 0.09% of resources come from EBMSP, while 0.10% is funded by partner laboratories. Additionally, 1% of resources originate from the Unified Health System (SUS) in other locations.
CONCLUSIONS: This study emphasizes the importance of assessing and analyzing costs and access to therapeutic interventions in DMD treatment. The predominance of annual expenses related to medications highlights the need for a deeper understanding of the factors influencing these costs. Investigating more effective therapeutic alternatives could potentially reduce long-term costs and improve clinical outcomes for patients.
METHODS: The research using the TDABC methodology collects data on diagnosis, treatment, and follow-up of patients in the JAV-RARAS study, mapping the care journey in 21 rare diseases. Direct costs were obtained through interviews and analysis of healthcare professionals' records, focusing on Duchenne Muscular Dystrophy (DMD). The analysis includes identification of activities, resource allocation, calculation of costs per unit, and measurement of activity duration.
RESULTS: The average annual cost for a patient undergoing DMD treatment is $ 79,010.57, with most expenses concentrated on medications. The cost distribution is as follows: human resources $ 253.38, equipment $ 798.00, medications $ 77,724.70, and exams $ 234.49. According to the data analysis, most of the annual investment is allocated to disease treatment. The source of funding comes from judicialization, accounting for approximately 98% of total resources. In contrast, only 0.09% of resources come from EBMSP, while 0.10% is funded by partner laboratories. Additionally, 1% of resources originate from the Unified Health System (SUS) in other locations.
CONCLUSIONS: This study emphasizes the importance of assessing and analyzing costs and access to therapeutic interventions in DMD treatment. The predominance of annual expenses related to medications highlights the need for a deeper understanding of the factors influencing these costs. Investigating more effective therapeutic alternatives could potentially reduce long-term costs and improve clinical outcomes for patients.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
EE173
Topic
Economic Evaluation
Topic Subcategory
Budget Impact Analysis, Cost/Cost of Illness/Resource Use Studies
Disease
SDC: Rare & Orphan Diseases