Na Li1,2, Bin Zheng1,2, Hongfu Cai1,2, Ting Yang3, Yunda Hong3, Maobai Liu4,5, Jianda Hu6. 1. Department of Pharmacy, Fujian Medical University Union Hospital, Fuzhou, China. 2. The School of Pharmacy, Fujian Medical University, Fuzhou, China. 3. Department of Hematology, Fujian Provincial Key Laboratory On Hematology, Fujian Institute of Hematology, Fujian Medical University Union Hospital, Fuzhou, China. 4. Department of Pharmacy, Fujian Medical University Union Hospital, Fuzhou, China. liumaobai@163.com. 5. The School of Pharmacy, Fujian Medical University, Fuzhou, China. liumaobai@163.com. 6. Department of Hematology, Fujian Provincial Key Laboratory On Hematology, Fujian Institute of Hematology, Fujian Medical University Union Hospital, Fuzhou, China. drjiandahu@163.com.
Abstract
PURPOSE: Axicabtagene ciloleucel (Axi-Cel, 2 × 106 CAR-T cells/kg, single intravenous injection) is a chimeric antigen receptor cell immunotherapy that exhibits favorable clinical efficacy and safety in patients with relapsed or refractory diffuse large B-cell lymphoma (R/R DLBCL). However, this treatment is expensive in China. This study aimed to evaluate the cost-effectiveness of Axi-Cel versus salvage chemotherapy for the treatment of R/R DLBCL from the perspective of the Chinese healthcare system. METHODS: A decision analysis model containing a short-term decision tree and long-term semi-Markov partitioned survival model was developed. The time horizon was 40 years and the period from 10 to 40 years was included in sensitivity analysis. The model was developed based on data from the ZUMA-1 and SCHOLAR-1 trials. Life years, quality-adjusted life years (QALYs), overall costs, and the incremental cost-effectiveness ratio (ICER) were estimated at a willingness to pay (WTP) threshold of US $31,320 per QALY, which is three times the gross domestic product per capita. RESULTS: The base case analysis revealed that treatment with Axi-Cel is associated with an increased overall cost of US $175,380 and improved effectiveness of 3.43 LYs and 2.61 QALYs compared to salvage chemotherapy, leading to an ICER of US $51,190 per LY and US $67,250 per QALY. The developed model is sensitive to the discount rate, utility of progression-free survival (PFS), and cost of Axi-Cel. The ICER of Axi-Cel was greater than the WTP threshold in the sensitivity and scenario analyses. To achieve cost-effectiveness, the price of Axi-Cel must be reduced by 59.19% to US $71,000. CONCLUSION: At its current price, Axi-Cel is not likely to be a cost-effective option compared to salvage chemotherapy for adult patients with R/R DLBCL.
PURPOSE: Axicabtagene ciloleucel (Axi-Cel, 2 × 106 CAR-T cells/kg, single intravenous injection) is a chimeric antigen receptor cell immunotherapy that exhibits favorable clinical efficacy and safety in patients with relapsed or refractory diffuse large B-cell lymphoma (R/R DLBCL). However, this treatment is expensive in China. This study aimed to evaluate the cost-effectiveness of Axi-Cel versus salvage chemotherapy for the treatment of R/R DLBCL from the perspective of the Chinese healthcare system. METHODS: A decision analysis model containing a short-term decision tree and long-term semi-Markov partitioned survival model was developed. The time horizon was 40 years and the period from 10 to 40 years was included in sensitivity analysis. The model was developed based on data from the ZUMA-1 and SCHOLAR-1 trials. Life years, quality-adjusted life years (QALYs), overall costs, and the incremental cost-effectiveness ratio (ICER) were estimated at a willingness to pay (WTP) threshold of US $31,320 per QALY, which is three times the gross domestic product per capita. RESULTS: The base case analysis revealed that treatment with Axi-Cel is associated with an increased overall cost of US $175,380 and improved effectiveness of 3.43 LYs and 2.61 QALYs compared to salvage chemotherapy, leading to an ICER of US $51,190 per LY and US $67,250 per QALY. The developed model is sensitive to the discount rate, utility of progression-free survival (PFS), and cost of Axi-Cel. The ICER of Axi-Cel was greater than the WTP threshold in the sensitivity and scenario analyses. To achieve cost-effectiveness, the price of Axi-Cel must be reduced by 59.19% to US $71,000. CONCLUSION: At its current price, Axi-Cel is not likely to be a cost-effective option compared to salvage chemotherapy for adult patients with R/R DLBCL.
Authors: Michael Crump; John Kuruvilla; Stephen Couban; David A MacDonald; Vishal Kukreti; C Tom Kouroukis; Morel Rubinger; Rena Buckstein; Kevin R Imrie; Massimo Federico; Nicola Di Renzo; Kang Howson-Jan; Tara Baetz; Leonard Kaizer; Michael Voralia; Harold J Olney; A Robert Turner; Jonathan Sussman; Annette E Hay; Marina S Djurfeldt; Ralph M Meyer; Bingshu E Chen; Lois E Shepherd Journal: J Clin Oncol Date: 2014-09-29 Impact factor: 44.544