OBJECTIVES: The objective of the study was to propose a protocol for measuring and analyzing adherence to Clinical Protocols and Therapeutic Guidelines (PCDTs) developed within the scope of the JAV-RARAS Project, as part of the National Rare Diseases Network survey.

METHODS: The protocol involved reviewing Clinical Protocols and Therapeutic Guidelines (PCDTs), identifying adherence indicators, selecting measurement methods, developing personalized and validated measurement instruments (through pilot tests with a representative sample of healthcare professionals and patients), implementing adherence measurement, analyzing and interpreting data, continuous evaluation, and improvement. Time-Driven Activity-Based Costing (TDABC) was utilized to assess resources and costs associated with healthcare, while the TPValue system was employed to support management by aggregating and organizing data, simplifying process analysis.

RESULTS: In the analysis of the Clinical Protocol and Therapeutic Guidelines (PCDT) for Type II Mucopolysaccharidosis, it is evident that the PCDT allocates 4.63% of costs to diagnosis, 93.5% to treatment, and 1.77% to follow-up. In contrast, Time-Driven Activity-Based Costing (TDABC) indicates 0.79%, 95.3%, and 3.82%, respectively. The cost breakdown by the type of treatment reveals that the PCDT adopts an all-drug allocation, while TDABC shows 87.2% for medication and 12.8% for surgery.

Similarly, for Osteogenesis Imperfecta, the PCDT allocates 2.05% of costs to diagnosis, 85.6% to treatment, and 11.5% to follow-up. In contrast, TDABC indicates 2.43%, 42.4%, and 34.5%, respectively. The allocation by the type of treatment shows that the PCDT allocates 69.7% for medication, 21.5% for surgical, and 8.7% for non-medication. On the other hand, TDABC indicates 47.2% for medication, 50.2% for non-drug allocation, and 2.6% for fractures.

CONCLUSIONS: This study presents a novel protocol using TDABC to measure and analyze adherence to PCDTs, aiming to optimize resources, establish approaches aligned with healthcare reality, and enhance the quality of healthcare for patients with rare diseases in Brazil.