JAV-RARAS: Analysis of Adherence to Clinical Protocols and Therapeutic Guidelines (PCDT) for Rare Diseases – A Comparison of Real-World Clinical Practice in the Context of the Value-Based Care Journey for RARAS.
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, MD3, RARAS Study Group, MD2;
1MAPE Solutions, São Paulo, Brazil, 2Porto Alegre Clinical Hospital, Porto Alegre, Brazil, 3Porto Alegre Clinical Hospital, São Paulo, Brazil
1MAPE Solutions, São Paulo, Brazil, 2Porto Alegre Clinical Hospital, Porto Alegre, Brazil, 3Porto Alegre Clinical Hospital, São Paulo, Brazil
OBJECTIVES: The Clinical Protocol and Therapeutic Guidelines (PCDT) establish criteria for the diagnosis and treatment of rare diseases. This study aimed to map the care pathways of patients with rare diseases in reference centers and compare them with the PCDT from the Ministry of Health (MS), aiming to evaluate compliance, identify discrepancies, and propose improvements in care.
METHODS: The study, titled "Value-Based Care Journey for Patients with Rare Diseases (JAV-RARAS)," is a national prospective cohort that uses the Time-Driven Activity-Based Costing (TDABC) methodology to analyze the care journey, and the costs associated with the management of 21 rare diseases. The research identified the actual annual treatment costs in the Unified Health System (SUS) through interviews with healthcare professionals and administrative and care analyses. The cost stipulated by the PCDT was calculated based on the definitions in the document, allowing for a direct comparison with clinical practice.
RESULTS: The care journeys and service processes in clinical centers were mapped, measuring adherence to the PCDTs. Preliminary data indicate that the average annual cost for Acromegaly was R$ 1,049,864.46 (approximately $209,972.89), while the cost stipulated by the PCDT was R$ 4,265,225.84 (approximately $853,045.17). For Angioedema Associated with C1 Deficiency, the annual cost at the center was R$ 300,278.91 (approximately $60,055.78), in contrast to R$ 66,850.22 (approximately $13,370.04) from the PCDT. A significant difference was observed between the costs and processes proposed by PCDT and those actually practiced.
CONCLUSIONS: The preliminary analysis suggests that current clinical practice is more costly than what is outlined in the PCDT, indicating that patient needs may not be adequately met. The results highlight the importance of updating clinical protocols, monitoring compliance, and adapting them to patient demands to ensure quality medical care.
METHODS: The study, titled "Value-Based Care Journey for Patients with Rare Diseases (JAV-RARAS)," is a national prospective cohort that uses the Time-Driven Activity-Based Costing (TDABC) methodology to analyze the care journey, and the costs associated with the management of 21 rare diseases. The research identified the actual annual treatment costs in the Unified Health System (SUS) through interviews with healthcare professionals and administrative and care analyses. The cost stipulated by the PCDT was calculated based on the definitions in the document, allowing for a direct comparison with clinical practice.
RESULTS: The care journeys and service processes in clinical centers were mapped, measuring adherence to the PCDTs. Preliminary data indicate that the average annual cost for Acromegaly was R$ 1,049,864.46 (approximately $209,972.89), while the cost stipulated by the PCDT was R$ 4,265,225.84 (approximately $853,045.17). For Angioedema Associated with C1 Deficiency, the annual cost at the center was R$ 300,278.91 (approximately $60,055.78), in contrast to R$ 66,850.22 (approximately $13,370.04) from the PCDT. A significant difference was observed between the costs and processes proposed by PCDT and those actually practiced.
CONCLUSIONS: The preliminary analysis suggests that current clinical practice is more costly than what is outlined in the PCDT, indicating that patient needs may not be adequately met. The results highlight the importance of updating clinical protocols, monitoring compliance, and adapting them to patient demands to ensure quality medical care.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
EE68
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies, Value of Information
Disease
SDC: Rare & Orphan Diseases