A pharmacoeconomic study of pegaspargase in the treatment of pediatric acute lymphoblastic leukemia in Russia

Pegaspargase is an L-asparaginase that is conjugated to monomethoxypolyethylene glycol. It is currently approved for use in children and adults for acute lymphoblastic leukemia (ALL) in the Russian Federation, is included in the recommendations of clinical practice guidelines and is recommended for inclusion in the Vital and Essential Drug List. The aim of this research is to perform a pharmacoeconomic analysis of pegaspargase in the treatment of pediatric patients with ALL in the Russian healthcare setting. The study was approved by the Independent Ethics Committee and the Scientific Council of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of the Ministry of Healthcare of the Russian Federation. The target population of the study were pediatric patients with intermediate-risk ALL (according to the Moscow–Berlin protocol) who had not previously received treatment. We compared two treatment scenarios: therapy according to the Moscow–Berlin protocol with and without pegaspargase. A decision tree model was used to analyze the costs of ALL treatment strategies. The costs of first-line therapy, relapse therapy (in accordance with the ALL-REZ-MB-2014 protocol), hematopoietic allogenic stem cell transplantation, and third-line therapy with blinatumomab (in patients with B-cell ALL) were considered. Costs were calculated separately for patients receiving different treatments and were weight-averaged according to the total number of intermediate-risk patients. The size of the target population was determined by the incidence of ALL in 2020 and risk group distribution data obtained from the Moscow-Berlin protocol study. The costs were calculated for a cohort of patients available for therapy in 2023. The use of pegaspargase leads to a decrease in the average discounted cost of therapy for children with intermediate risk ALL of 93,273 rubles due to a decrease in the need for costly relapse therapy. Estimates show that up to 418 new patients are diagnosed with intermediate-risk ALL annually and belong the target population of this study. If 100% of ALL patients receive pegaspargase on the third day of remission induction therapy, the total cost of therapy for the target patient population will decrease by 38.99 million rubles. The use of pegaspargase on the third day of remission induction therapy in children with intermediate-risk ALL leads to a reduction in the relapse rate (superior efficacy) and lower costs, compared with the treatment regimen without pegaspargase.