OBJECTIVES : In Brazil, cost-effectiveness analysis is not the only indicator to orient the decision-making process, especially due to the absence of a willingness-to-pay threshold. Using an alternative health technology assessment (HTA) model, this study aimed to evaluate the incorporation of eltrombopag olamine for the treatment of refractory severe aplastic anemia (SAA) in the Brazilian supplementary healthcare system (SHS).

METHODS : An alternative HTA model, consisting of a Multi-Criteria Decision Analysis (MCDA) presented the following flow: problem context description, criteria selection, process structuring, criteria rationale construction, score attribution, mathematical model elaboration and software development. Criteria and score scales were based on literature review and validated by specialists in a second moment. The criteria were organized according to relevance level in a proposed HTA diagram, and they were classified as eliminators, decision-making and complementary. Eliminators criteria were the minimum requirements to enable the technology assessment on the next steps. Decision-making criteria represented the main aspects/indicators during the HTA processes, including clinical benefit and economic impact of eltrombopag. Complementary criteria were only evaluated in case the previous result demonstrated any uncertainty. Scores were indicated by a scale of 0 to 10. A sensitivity analysis was performed to verify the result robustness.

RESULTS : Eltrombopag attended all requirements of eliminators criteria. Regarding the decision-making criteria, the new technology indicated to have an assertive clinical evidence (7.00), with very high drug effect (8.50) and very low budget impact (0.02) due to very small use expectation. Final score was 7.69, which indicated recommendation of eltrombopag incorporation. There was no need to evaluate the complementary criteria. Sensitivity analysis results demonstrated consistency to the base case.

CONCLUSIONS : Considering the context of refractory SAA in Brazil, the incorporation of eltrombopag demonstrated to be favorable, being an option with high overall clinical benefit accompanied by a very low budget impact in the Brazilian SHS.