Economic evaluation of different medicines for acute myeloid leukemia: a systematic review_Chinese Journal of Evidence-Based Medicine

Authors：

LI Rui ^1,2,3 , XIAO Feiyi ^1,4 , LIU Yuanyuan ⁵ , ZOU Xinru ⁶ , FU Jiani ^6,7 , YANG Kehu ^2,3 , ZHAO Kun ¹ ,  LI Xue ¹

1. China National Health Development Research Center, Beijing 100044, P. R. China;
2. Evidence Based Social Science Research Center, School of Public Health, Lanzhou University, Lanzhou 730000, P. R. China;
3. Evidence Based Medicine Center, School of Basic Medical Sciences, Lanzhou University, Lanzhou 730000, P. R. China;
4. School of Business Administration, Shenyang Pharmaceutical University, Shenyang 110016, P. R. China;
5. The First School of Clinical Medicine, Lanzhou University, Lanzhou 730000, P. R. China;
6. The Second School of Clinical Medicine, Lanzhou University, Lanzhou 730000, P. R. China;
7. First Affiliated Hospital of Xi’an Jiaotong University, Xi’an 710061, P. R. China;

Corresponding?author：

LI Xue, Email: lx0204@126.com

Keywords：

Acute myeloid leukemia; Hypomethylating agents; Targeted medicine; Economic evaluation; Systematic review

DOI：

10.7507/1672-2531.202211023

Video：

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Objective To systematically review the health economic evaluation studies of medicines for the treatment of acute myeloid leukemia (AML). Methods The PubMed, EMbase, Cochrane Library, CBM, CNKI, and WanFang Data, as well as the CRD database specifically for health economics were electronically searched from inception to June 2022, and related journals in the field of health economics and the websites of HTA institutions in various countries were manually searched. The quality of the studies was assessed using the CHEERS checklist. The basic characteristics of health economics evaluation publications were summarized, the quality of model structures and methodologies was assessed and economic evaluation results were compared among different treatments. Results A total of 17 studies were included, and cost-effectiveness analyses were conducted from the perspectives of the health system, patients, the whole society, and medical insurance payers. The economic evaluation models were relatively unified, but there were differences in methods and results reporting, and the quality needed to be improved. The research objects were mainly the comparison of hypomethylating agents, targeted medicine and traditional chemotherapy regimens, as well as the comparison of different chemotherapy combinations and different drug dosages. Conclusion Real-world studies are mainly focused on traditional chemotherapy regimens, and model-based health economic evaluations, such as Markov models, are more frequently applied to newly developed targeted drugs and demethylation drugs. Among all treatments, the chemotherapy regimens including cytarabine, midostaurin, and decitabine are found to be more cost-effective.

Citation： LI Rui, XIAO Feiyi, LIU Yuanyuan, ZOU Xinru, FU Jiani, YANG Kehu, ZHAO Kun, LI Xue. Economic evaluation of different medicines for acute myeloid leukemia: a systematic review. Chinese Journal of Evidence-Based Medicine, 2023, 23(6): 638-646. doi: 10.7507/1672-2531.202211023 Copy

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8.	趙爽, 喬文亮, 邱志新, 等. 吉非替尼治療晚期非小細胞肺癌的藥物經濟學系統評價. 華西醫學, 2018, 33(1): 76-83.
9.	王莉, 胡善聯, 孫鑫, 等. 衛生經濟學分析的系統評價方法與挑戰. 中國循證醫學雜志, 2008, 8(11): 1007-1011.
10.	Husereau D, Drummond M, Petrou S, et al. Consolidated health economic evaluation reporting standards (CHEERS) statement. Value Health, 2013, 16(2): e1-e5.
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12.	Huang BT, Wang Y, Du QF, et al. Analysis of efficacy and cost-effectiveness of high-dose arabinoside versus daunorubicin chemotherapy in older adult patients with acute myeloid leukemia by cytogenetic risk profile: retrospective review from China. Int J Hematol, 2011, 93(4): 474-481.
13.	Stein E, Xie J, Duchesneau E, et al. Cost effectiveness of midostaurin in the treatment of newly diagnosed FLT3-mutated acute myeloid Leukemia in the United States. Pharmacoeconomics, 2019, 37(2): 239-253.
14.	Tremblay G, Dolph M, Patel S, et al. Cost-effectiveness analysis for midostaurin versus standard of care in acute myeloid leukemia in the United Kingdom. Cost Eff Resour Alloc, 2018, 16: 33.
15.	Lambertenghi-Deliliers G, Oriani A, Pioltelli P, et al. Cost-effectiveness analysis of induction regimens containing anthracyclines in adult acute myelogenous leukemia. Leuk Lymphoma, 1991, 5(1): 33-41.
16.	Uyl-de Groot CA, L?wenberg B, Vellenga E, et al. Cost-effectiveness and quality-of-life assessment of GM-CSF as an adjunct to intensive remission induction chemotherapy in elderly patients with acute myeloid leukemia. Br J Haematol, 1998, 100(4): 629-636.
17.	Arenaza A, Diez R, Esteve J, et al. Cost-effectiveness of midostaurin in the treatment of acute myeloid leukemia with the FLT3 mutation in Spain. Clinicoecon Outcomes Res, 2019, 11: 683-694.
18.	Tremblay G, Cariou C, Recher C, et al. Cost-effectiveness of midostaurin in the treatment of newly diagnosed FLT3-mutated acute myeloid leukemia in France. Eur J Health Econ, 2020, 21(4): 543-555.
19.	Jacob LA, Aparna S, Lakshmaiah KC, et al. Decitabine compared with low-dose cytarabine for the treatment of older patients with newly diagnosed acute myeloid leukemia: a pilot study of safety, efficacy, and cost-effectiveness. Adv Hematol, 2015, 2015: 167029.
20.	Batty N, Yin Y, Wetzler M. Decitabine is more cost effective than cytarabine and daunorubicin in elderly acute myeloid leukemia patients. Journal of Cancer Research & Therapy, 2014, 2(4): 68-73.
21.	Coyle D, Villeneuve PJA. Economic evaluation of azacitidine in elderly patients with acute myeloid leukemia with high blast counts. Pharmacoecon Open, 2020, 4(2): 297-305.
22.	National Institute for Health and Care Excellence. TA399-Azacitidine for treating acute myeloid leukaemia with more than 30% bone marrow blasts. 2016.
23.	National Institute for Health and Care Excellence. TA545-Gemtuzumab ozogamicin for untreated acute myeloid leukaemia. 2018.
24.	National Institute for Health and Care Excellence. TA552-Liposomal cytarabine–daunorubicin for untreated acute myeloid leukaemia. 2018.
25.	National Institute for Health and Care Excellence. TA642-Gilteritinib for treating relapsed or refractory acute myeloid leukaemia. 2020.
26.	張伶俐, 顏建周, 袁力. 阿糖胞苷分別聯合伊達比星、柔紅霉素治療急性非淋巴細胞白血病成本效果的比較分析. 藥學與臨床研究, 2016, 24(1): 66-68.
27.	張志萍, 朱秀美, 郭代紅, 等. 急性非淋巴性白血病3種治療方案的成本效果分析. 藥物與臨床, 2001, 16(2): 9-10.
28.	郭代紅, 張志萍, 朱秀美, 等. 急性非淋巴性白血病7種化療方案的成本-效果分析. 中國藥業, 2003, 12(5): 51-52.

1. National Cancer Institute. SEER stat fact sheets: chronic myeloid leukemia. 2020.
2. 應江偉, 杜靈彬, 鄭艷澤, 等. 浙江省腫瘤登記地區2000~2009年白血病發病與死亡分析. 中國腫瘤, 2014, 23(7): 538-542.
3. 黃治虎, 陳寶安, 歐陽建, 等. 我國白血病流行病學調查的現狀和對策. 臨床血液學雜志, 2009, 22(2): 166-167.
4. 周建軍, 陳睿, 王燁菁, 等. 上海市盧灣區居民2004-2011年白血病發病和死亡資料分析. 中華腫瘤防治雜志, 2017, 24(8): 507-511.
5. 斯垚翔, 錢虹, 劉玉琴, 等. 甘肅省2010年白血病發病和死亡分析. 中國腫瘤, 2015, 24(2): 81-88.
6. 董金鳳. 2011-2012年福建省新發成人急性白血病的發病情況調查. 福建: 福建醫科大學, 2014.
7. 劉玉琴, 趙鳳菊, 陳萬青, 等. 中國2009年白血病發病和死亡資料分析. 中國腫瘤, 2013, 22(7): 528-534.
8. 趙爽, 喬文亮, 邱志新, 等. 吉非替尼治療晚期非小細胞肺癌的藥物經濟學系統評價. 華西醫學, 2018, 33(1): 76-83.
9. 王莉, 胡善聯, 孫鑫, 等. 衛生經濟學分析的系統評價方法與挑戰. 中國循證醫學雜志, 2008, 8(11): 1007-1011.
10. Husereau D, Drummond M, Petrou S, et al. Consolidated health economic evaluation reporting standards (CHEERS) statement. Value Health, 2013, 16(2): e1-e5.
11. 肖軍, 孫謹芳, 王琦琦, 等. 衛生經濟學評價報告指南及應用現狀. 中華預防醫學雜志, 2017, 51(3): 276-280.
12. Huang BT, Wang Y, Du QF, et al. Analysis of efficacy and cost-effectiveness of high-dose arabinoside versus daunorubicin chemotherapy in older adult patients with acute myeloid leukemia by cytogenetic risk profile: retrospective review from China. Int J Hematol, 2011, 93(4): 474-481.
13. Stein E, Xie J, Duchesneau E, et al. Cost effectiveness of midostaurin in the treatment of newly diagnosed FLT3-mutated acute myeloid Leukemia in the United States. Pharmacoeconomics, 2019, 37(2): 239-253.
14. Tremblay G, Dolph M, Patel S, et al. Cost-effectiveness analysis for midostaurin versus standard of care in acute myeloid leukemia in the United Kingdom. Cost Eff Resour Alloc, 2018, 16: 33.
15. Lambertenghi-Deliliers G, Oriani A, Pioltelli P, et al. Cost-effectiveness analysis of induction regimens containing anthracyclines in adult acute myelogenous leukemia. Leuk Lymphoma, 1991, 5(1): 33-41.
16. Uyl-de Groot CA, L?wenberg B, Vellenga E, et al. Cost-effectiveness and quality-of-life assessment of GM-CSF as an adjunct to intensive remission induction chemotherapy in elderly patients with acute myeloid leukemia. Br J Haematol, 1998, 100(4): 629-636.
17. Arenaza A, Diez R, Esteve J, et al. Cost-effectiveness of midostaurin in the treatment of acute myeloid leukemia with the FLT3 mutation in Spain. Clinicoecon Outcomes Res, 2019, 11: 683-694.
18. Tremblay G, Cariou C, Recher C, et al. Cost-effectiveness of midostaurin in the treatment of newly diagnosed FLT3-mutated acute myeloid leukemia in France. Eur J Health Econ, 2020, 21(4): 543-555.
19. Jacob LA, Aparna S, Lakshmaiah KC, et al. Decitabine compared with low-dose cytarabine for the treatment of older patients with newly diagnosed acute myeloid leukemia: a pilot study of safety, efficacy, and cost-effectiveness. Adv Hematol, 2015, 2015: 167029.
20. Batty N, Yin Y, Wetzler M. Decitabine is more cost effective than cytarabine and daunorubicin in elderly acute myeloid leukemia patients. Journal of Cancer Research & Therapy, 2014, 2(4): 68-73.
21. Coyle D, Villeneuve PJA. Economic evaluation of azacitidine in elderly patients with acute myeloid leukemia with high blast counts. Pharmacoecon Open, 2020, 4(2): 297-305.
22. National Institute for Health and Care Excellence. TA399-Azacitidine for treating acute myeloid leukaemia with more than 30% bone marrow blasts. 2016.
23. National Institute for Health and Care Excellence. TA545-Gemtuzumab ozogamicin for untreated acute myeloid leukaemia. 2018.
24. National Institute for Health and Care Excellence. TA552-Liposomal cytarabine–daunorubicin for untreated acute myeloid leukaemia. 2018.
25. National Institute for Health and Care Excellence. TA642-Gilteritinib for treating relapsed or refractory acute myeloid leukaemia. 2020.
26. 張伶俐, 顏建周, 袁力. 阿糖胞苷分別聯合伊達比星、柔紅霉素治療急性非淋巴細胞白血病成本效果的比較分析. 藥學與臨床研究, 2016, 24(1): 66-68.
27. 張志萍, 朱秀美, 郭代紅, 等. 急性非淋巴性白血病3種治療方案的成本效果分析. 藥物與臨床, 2001, 16(2): 9-10.
28. 郭代紅, 張志萍, 朱秀美, 等. 急性非淋巴性白血病7種化療方案的成本-效果分析. 中國藥業, 2003, 12(5): 51-52.

Chinese Journal of Evidence-Based Medicine

Economic evaluation of different medicines for acute myeloid leukemia: a systematic review

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