Objective To evaluate the cost-effectiveness of chemotherapy in children with newly diagnosed Hodgkin lymphoma at low-, intermediate-, and high-risk. Methods From the perspective of health system, a decision-tree model was designed for cost-effectiveness analysis. The chemotherapy regimens of low-risk group included OEPA (vincristine, etoposide, prednisone, doxorubicin), AV-PC (doxorubicin, vincristine, prednisone, cyclophosphamide), and ABVD (doxorubicin, bleomycin, vincristine, dacarbazine); intermediate-risk group included OEPA, ABVE-PC (doxorubicin, bleomycin, vincristine, etoposide, prednisone, cyclophosphamide) and ABVD; high-risk group included OEPA, ABVE-PC, ABVD and BEACOPP (bleomycin, etoposide, doxorubicin, cyclophosphamide, vincristine, procarbazine, prednisone). The effectiveness and cost parameters were derived from the event-free survival rate reported in the literature, the drug linked reference price in Sichuan province, and treatment price of medical institutions. Univariate and probabilistic sensitivity analysis were performed to explore the impact of uncertainty. Results In the low-risk group, compared with AV-PC, the incremental cost-effectiveness ratios (ICER) of OEPA and ABVD were 80 700 yuan and 108 799 yuan, respectively. In the intermediate-risk group, compared with OEPA, the ICER of ABVE-PC and ABVD were −17 737 yuan and −4 701 yuan respectively. In the high-risk group, compared with ABVE-PC, the ICER of OEPA, ABVD and BEACOPP were 149 262, 472 090 and 64 652 yuan, respectively. Univariate sensitivity analysis showed that in low-risk group, the most influential factors were cost of OEPA and cost of ABVD; in moderate-risk group were cost of ABVE-PC and cost of OEPA; in the high-risk group were cost of OEPA, cost of ABVD, and cost of BEACOPP, respectively. The results of probabilistic sensitivity analysis are basically consistent with those of the main analysis. Conclusion If China's per capita gross domestic product in 2023 (89 358 yuan) was used as the willingness-to-pay (WTP) threshold, OEPA in the low-risk group, OEPA in the intermediate-risk group and BEACOPP in the high-risk group are cost-effective.
With the continuous progress of national medical insurance strategic purchasing and value-based healthcare, pharmacoeconomic evaluation, serving as a technical tool for assessing the cost-effectiveness of healthcare interventions, has played an important role in policy decision support. Comparative efficacy evidence is the core data source for pharmacoeconomic evaluation, and also the foundation for conducting pharmacoeconomic research. In recent years, the number of innovative drugs approved based on single-arm trial has been increasing. Most existing randomized controlled clinical trials are also placebo-controlled or compared with traditional treatments, unable to directly meet the need for efficacy evidence of comparisons with conventional or standard treatments in pharmacoeconomic evaluations. In the absence of direct comparative efficacy evidence, exploring indirect comparison methods for efficacy has become a cutting-edge direction in pharmacoeconomic evaluation. Through a comprehensive literature review and systematic analysis, this study focuses on five indirect comparison methods based on individual patient data for population adjustment, including match adjusted indirect comparison (MAIC), simulated treatment comparison (STC), propensity score matching (PSM), inverse probability of treatment weighting (IPTW) and network meta regression (NMR), and discussing their basic concepts, advantages and disadvantages and application comparisons. Finally, it provides methodological suggestions on how to choose an indirect comparison method for efficacy, with the aim of promoting the generation of higher-quality indirect comparison evidence for efficacy and advancing pharmacoeconomic evaluation to provide high-quality evidence references for healthcare policy decision-making.