JAV-RARAS: Analysis of the Value-Driven Care Journey for Patients With Rare Diseases in Brazil Applying the Time-Driven Activity-Based Costing Methodology
Author(s)
Camila Azevedo, MBA1, Marcelo E. Nita, MSc, PhD, MD1, Myrianne Barbosa, MSc1, Luana Lopes, MSc1, Thiago Godoy, MSc1, Claudia Lorea, MSc, MD2, Temis Felix, MSc, PhD, MD2, RARAS Study Group, MD2;
1MAPE Solutions, São Paulo, Brazil, 2Porto Alegre Clinical Hospital, Porto Alegre, Brazil
1MAPE Solutions, São Paulo, Brazil, 2Porto 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 conditions, establish value parameters, and evaluate cost-effectiveness to optimize resource allocation. The study seeks to quantify and describe the costs associated with the care journey of patients diagnosed with 21 rare diseases, involving 35 medical centers and utilizing the Time-Driven Activity-Based Costing (TDABC) methodology.
METHODS: The research using the TDABC methodology collects data on diagnosis, treatment, and follow-up of patients in the JAV-RARAS study. A mapping of the care journey for 21 rare diseases was conducted, quantifying time and resources in the diagnosis, treatment, and follow-up phases. Direct costs were obtained through interviews and analysis of health professionals' records. The analysis included identifying activities in diseases such as Osteogenesis Imperfecta (OI), Acromegaly (ACRO), and Duchenne Muscular Dystrophy (DMD), as well as calculating costs per unit and measuring the duration of activities.
RESULTS: The average annual cost for a patient undergoing treatment for OI is $1,080.43, for Acromegaly it is $209,972.89, and for DMD it is $53,102.02. Most costs are allocated to treatment, with $523.32 for OI, $209,493.64 for Acromegaly, and $52,339.81 for DMD. The majority of the annual investment is directed exclusively to treatment. Regarding the source of funding, some comes from judicialization in the case of Acromegaly, from participating centers, the patient, and the Unified Health System (SUS) in other locations.
CONCLUSIONS: This study highlights the importance of assessing and analyzing costs and access to therapeutic interventions in the treatment of rare diseases. The predominance of annual expenses related to medications underscores 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. A mapping of the care journey for 21 rare diseases was conducted, quantifying time and resources in the diagnosis, treatment, and follow-up phases. Direct costs were obtained through interviews and analysis of health professionals' records. The analysis included identifying activities in diseases such as Osteogenesis Imperfecta (OI), Acromegaly (ACRO), and Duchenne Muscular Dystrophy (DMD), as well as calculating costs per unit and measuring the duration of activities.
RESULTS: The average annual cost for a patient undergoing treatment for OI is $1,080.43, for Acromegaly it is $209,972.89, and for DMD it is $53,102.02. Most costs are allocated to treatment, with $523.32 for OI, $209,493.64 for Acromegaly, and $52,339.81 for DMD. The majority of the annual investment is directed exclusively to treatment. Regarding the source of funding, some comes from judicialization in the case of Acromegaly, from participating centers, the patient, and the Unified Health System (SUS) in other locations.
CONCLUSIONS: This study highlights the importance of assessing and analyzing costs and access to therapeutic interventions in the treatment of rare diseases. The predominance of annual expenses related to medications underscores 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
EE67
Topic
Economic Evaluation
Topic Subcategory
Budget Impact Analysis, Cost/Cost of Illness/Resource Use Studies
Disease
SDC: Rare & Orphan Diseases