Presentation (CTI)

OBJECTIVES: Pembrolizumab, with 39 indications approved in Brazil, presents significant resource allocation challenges for the Brazilian Public Health System (SUS). This study introduces an innovative cost-effectiveness analysis framework to simultaneously evaluate five high-clinical-benefit indications (NSCLC, Esophagus, Breast, Melanoma, and Cervical cancer), representing an estimated 17,895 patients, to determine the conditions under which broad implementation is an efficient investment.
METHODS: A cost-effectiveness analysis was performed from the SUS perspective with a lifetime horizon and a 5% discount rate, using life years gained as the outcome. The target population was selected based on ESMO-ScoreCards. The model compared 31 possible combinations of using Pembrolizumab or chemotherapy across the five cancer types. Survival curves (OS and PFS) were extracted from clinical trials, fitted to survival, and calibrated to avoid parametrization issues during extrapolation. Each cancer type in each alternative was weighted according to its prevalence.
RESULTS: The base-case analysis, using the standard Pembrolizumab price (U$2,945.40/vial), showed that all strategies were above the SUS cost-effectiveness threshold (U$19,626/YLS). A deterministic sensitivity analysis on price revealed that reducing the vial price not only lowered the ICER but also changed the ranking of efficient strategies. To be cost-effective, the most efficient strategy was using Pembrolizumab only for melanoma patients, requiring a price reduction to U$711.40/vial. The price needed to be progressively reduced to U$490,46/vial, U$421,12/vial, U$383,31/vial and U$278.50/vial to include it for breast, cervical, esophagus and NSCLC respectively while remaining below the threshold in each inclusion.
CONCLUSIONS: This multi-indication HTA framework provides a powerful tool for efficient price negotiation, quantifying the price reduction necessary for each increase in the number of treated patients. This approach is critical for improving patient access to high-cost oncological drugs while maintaining the ICER under the cost-effectiveness threshold.