APPROPRIATE METHOD FOR FARMACOECONOMIC ANALYSIS OF MONOCLONAL ANTIBODIES EFFICACY IN CHILDREN WITH ACUTE LYMPHOBLASTIC LEUKEMIA

Abstract

Pharmacoeconomics of therapeutic strategies for orphan diseases in pediatrics remains an understudied area, despite the critical importance of this type of work in determining the optimal and most rational treatment tactics from a clinical and economic perspective. Currently, great advances have been made in the treatment of acute lymphoblastic leukemia (ALL), including the use of monoclonal antibodies (MA). The aim of this article is to evaluate the most rational method of clinical and economic evaluation of the use of MA in children with primary ALL in comparison with the standard therapy from the perspective of the public health system of the Russian Federation. Data from the TOWER and INO-VATE-ALL studies were used for clinical and economic evaluation of the efficacy of MA in the treatment of ALL. Complete response or complete response with partial or incomplete hematological remission were used as endpoints. Partitioned survival model B-GEM (transitional model for chronic conditions), which used 3 transitional states to which the patient progressed from the initial state (first 12 weeks, according to the TOWER study): response, refractory/relapsed, death was used for the analysis. Taking into account the described required adjustments, this clinical and economic analysis of the use of MA in the therapy of acute lymphoblastic leukemia in pediatric patients from the perspective of organization of medical care for children in the Russian Federation can be conducted using the B-GEM model after making the required changes in the presented model, considering the urgency of the problem and the absence of similar studies in pediatric patients in global practice.