Cost-Effectiveness Analysis of a Three-Drug Regimen Containing Bevacizumab for the Treatment of Recurrent Pediatric Medulloblastoma in China: Based on a COG Randomized Phase II Screening Trial.

Background: Medulloblastoma is the most common malignant brain tumor of childhood, accounting for 6 to 7 percent of all childhood CNS tumors. The purpose of this study was to evaluate the economic efficacy of a bevacizumab combined with temozolomide + irinotecan regimen for the treatment of recurrent pediatric medulloblastoma in China.
Methods: The data analyzed were from a randomized phase II screening trial that showed an improved survival benefit in child patients with recurrent medulloblastoma treated with a T+I+B combination regimen. A Markov model is constructed to estimate the incremental cost-effectiveness ratio (ICER) from the perspective of Chinese society. The uncertainty in the model is solved by one-way certainty and probabilistic sensitivity analysis.
Results: Our base case analysis showed that the total costs of treatment increased from $8,786.403 to $27,603.420 with the combination bevacizumab vs. the two-agent chemotherapy regimen. Treatment with T+I+B combination therapy was associated with an increase in effectiveness of 0.280 QALYs from 0.867 to 1.147 QALYs T+I regimen. The incremental cost-effectiveness ratio was $67,203.632/QALY, which exceeded our pre-specified willingness-to-pay threshold ($38,136.26/QALY). Cost changes associated with grade 3-4 AE management, tests used, or hospitalization costs had little effect on the ICER values predicted by sensitivity analysis.
Conclusions: Taken together, the results of this study suggest that the combination of bevacizumab with temozolomide and irinotecan is not a cost-effective option from the perspective of Chinese payers as a first-line treatment option for children with recurrent medulloblastoma in China.
Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.
(Copyright © 2022 Chen, Tian and Chen.)