Omalizumab in the treatment of children with asthma: a rapid health technology assessment_Chinese Journal of Evidence-Based Medicine

Authors：

LIU Jianing ^1,2,3,4,5 , ZENG Linan ^2,3,4 , WANG Lili ⁶ , LI Hailong ^2,3,4 , HUANG Liang ^2,3,4 , CHENG Guo ^4,7,8 ,  YANG Chunsong ^2,3,4 ,  ZHANG Lingli ^1,2,3,4

1. Chinese Evidence-based Medicine Center, West China Hospital, Sichuan University, Chengdu 610041, P. R. China;
2. Department of Pharmacy/Evidence-Based Pharmacy Center, West China Second University Hospital, Sichuan University; Children's Medicine Key Laboratory of Sichuan Province, Chengdu 610041, P. R. China;
3. NMPA Key Laboratory for Technical Research on Drug Products in vitro and in vivo Correlation, Chengdu 610041, P. R. China;
4. Key Laboratory of Birth Defects and Related Diseases of Women and Children, Sichuan University, Ministry of Education, Chengdu 610041, P. R. China;
5. West China School of Medicine, Sichuan University, Chengdu 610041, P. R. China;
6. Department of Pediatric Pulmonology and Lmmunology, West China Second University Hospital, Sichuan University, Chengdu 610041, P. R. China;
7. Department of Pediatrics, West China Second University Hospital, Sichuan University, Chengdu 610041, P. R. China;
8. Laboratory of Molecular Translational Medicine, Center for Translational Medicine, Sichuan University, Chengdu 610041, P. R. China;

Corresponding author：

YANG Chunsong, Email: yangchunsong_123@126.com; ZHANG Lingli, Email: zhanglingli@scu.edu.cn

Keywords：

Omalizumab; Pediatric asthma; Efficacy; Safety; Economics; Health technology assessment

DOI：

10.7507/1672-2531.202405095

Video：

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Objective To evaluate the efficacy, safety, and economics of omalizumab for the treatment of pediatric asthma through a rapid health technology assessment (HTA). Methods The search was conducted on INAHTA website and databases such as PubMed, Embase, Cochrane Library, SinoMed, CNKI, VIP, and WanFang Data from inception to August 2024. Literature screening, quality evaluation, and data extraction were conducted independently by two investigators. An interview was conducted to consult the medication and treatment opinions of doctors and patients to investigate its clinical application. Results A total of 28 articles, including HTA articles (4), SR/Meta analyses (18), and pharmacoeconomics articles (6) were included. Omalizumab could reduce the incidence of clinical exacerbations, decrease the number of asthma attacks/days with asthma symptoms, and improve qualification of life. The results of the safety evaluation showed that omalizumab could reduce the rate of serious adverse events, especially those related to worsening asthma. Foreign pharmacoeconomic studies showed ICER ranging from £30 109 to £78 009. A pharmacoeconomic study in China found an ICER of $211 217/QALY for omalizumab, which were both above the pre-set thresholds. Conclusion Omalizumab is a treatment for patients who suffer from moderate to severe persistent allergic asthma or moderate to severe allergic asthma. It has been found to improve disease symptoms without increasing serious adverse events. However, it is not considered cost-effective due to its high price.

1.	中华医学会儿科学分会呼吸学组, 《中华儿科杂志》编辑委员会. 儿童支气管哮喘诊断与防治指南(2016年版). 中华儿科杂志, 2016, 54(3): 167-181.
2.	全国儿科哮喘协作组, 中国疾病预防控制中心环境与健康相关产品安全所. 第三次中国城市儿童哮喘流行病学调查. 中华儿科杂志, 2013, 51(10): 729-735.
3.	Boulet LP, Reddel HK, Bateman E, et al. The global initiative for asthma (GINA): 25 years later. Eur Respir J, 2019, 54(2): 1900598.
4.	National Asthma Education and Prevention Program. Expert panel report 3 (EPR-3): guidelines for the diagnosis and management of asthma-summary report 2007. J Allergy Clin Immunol, 2007, 120(5 Suppl): S94-S138.
5.	杨祎, 姜明燕. 儿童支气管哮喘药物治疗研究与进展. 系统医学, 2019, 4(2): 193-195.
6.	Global Initiative For Asthma. Global strategy for asthma management and prevention. 2024.
7.	中国医药教育协会慢性气道疾病专业委员会, 中国哮喘联盟. 重度哮喘诊断与处理中国专家共识(2024). 中华医学杂志, 2024, 104(20): 1759-1789.
8.	中华医学会变态反应分会呼吸过敏学组(筹), 中华医学会呼吸病学分会哮喘学组. 中国过敏性哮喘诊治指南(第一版, 2019年). 中华内科杂志, 2019, 58(9): 636-655.
9.	Arakawa H, Adachi Y, Ebisawa M, et al. Japanese guidelines for childhood asthma 2020. Allergol Int, 2020, 69(3): 314-330.
10.	Holguin F, Cardet JC, Chung KF, et al. Management of severe asthma: a European Respiratory Society/American Thoracic Society guideline. Eur Respir J, 2020, 55(1): 1900588.
11.	Asher I, Pearce N. Global burden of asthma among children. Int J Tuberc Lung Dis, 2014, 18(11): 1269-1278.
12.	Hong J, Bao Y, Chen A, et al. Chinese guidelines for childhood asthma 2016: major updates, recommendations and key regional data. J Asthma, 2018, 55(10): 1138-1146.
13.	Lanier B, Bridges T, Kulus M, et al. Omalizumab for the treatment of exacerbations in children with inadequately controlled allergic (IgE-mediated) asthma. J Allergy Clin Immunol, 2009, 124(6): 1210-1216.
14.	唐密, 张雪艳, 杨燕, 等. 三级医疗机构医院卫生技术评估实施现况. 中国卫生资源, 2023, 26(3): 281-285.
15.	COOTHA. Omalizumab as add-on therapy to inhaled steroids for asthma. 2004.
16.	NICE. Omalizumab for treating severe persistent allergic asthma. 2013.
17.	NIHR. Omalizumab for the treatment of severe persistent allergic asthma: a systematic review and economic evaluation. 2013.
18.	周华, 车大钿. 奥马珠单抗治疗儿童重度过敏性哮喘的快速卫生技术评估. 上海医药, 2020, 41(21): 13-16,24.
19.	Lang D, Liu Z, Li D. Safety and tolerability of omalizumab in children with allergic (IgE-Mediated) asthma: a systematic review and meta-analysis. Discov Med, 2023, 35(176): 233-241.
20.	Fenu G, La Tessa A, Calogero C, et al. Severe pediatric asthma therapy: omalizumab-a systematic review and meta-analysis of efficacy and safety profile. Front Pediatr, 2023, 10: 1033511.
21.	Hanania NA, Niven R, Chanez P, et al. Long-term effectiveness and safety of omalizumab in pediatric and adult patients with moderate-to-severe inadequately controlled allergic asthma. World Allergy Organ J, 2022, 15(10): 100695.
22.	Liu L, Zhou P, Wang Z, et al. Efficacy and safety of omalizumab for the treatment of severe or poorly controlled allergic diseases in children: a systematic review and meta-analysis. Front Pediatr, 2022, 10: 851177.
23.	杨琰茗, 任丹阳, 翁稚颖, 等. 奥马珠单抗治疗中-重度过敏性哮喘儿童的文献分析. 海峡药学, 2022, 34(7): 113-117.
24.	Busse WW, Szefler SJ, Haselkorn T, et al. Possible protective effect of omalizumab on lung function decline in patients experiencing asthma exacerbations. J Allergy Clin Immunol Pract, 2021, 9(3): 1201-1211.
25.	王亚芹, 樊鹏利, 吕品, 等. 奥马珠单抗治疗儿童及青少年过敏性哮喘的系统评价. 中国药物评价, 2021, 38(2): 111-114.
26.	姜红玉, 李娟, 胡文凤, 等. 奥马珠单抗治疗儿童中重度过敏性哮喘的有效性和安全性系统评价. 中国医院用药评价与分析, 2021, 21(9): 1091-1096.
27.	Fu Z, Xu Y, Cai C. Efficacy and safety of omalizumab in children with moderate-to-severe asthma: a meta-analysis. J Asthma, 2021, 58(10): 1350-1358.
28.	Henriksen DP, Bodtger U, Sidenius K, et al. Efficacy of omalizumab in children, adolescents, and adults with severe allergic asthma: a systematic review, meta-analysis, and call for new trials using current guidelines for assessment of severe asthma. Allergy Asthma Clin Immunol, 2020, 16: 49.
29.	Corren J, Kavati A, Ortiz B, et al. Patient-reported outcomes in moderate-to-severe allergic asthmatics treated with omalizumab: a systematic literature review of randomized controlled trials. Curr Med Res Opin, 2018, 34(1): 65-80.
30.	Tortajada-Girbés M, Bousquet R, Bosque M, et al. Efficacy and effectiveness of omalizumab in the treatment of childhood asthma. Expert Rev Respir Med, 2018, 12(9): 745-754.
31.	Corren J, Kavati A, Ortiz B, et al. Efficacy and safety of omalizumab in children and adolescents with moderate-to-severe asthma: a systematic literature review. Allergy Asthma Proc, 2017, 38(4): 250-263.
32.	Rodrigo GJ, Neffen H. Systematic review on the use of omalizumab for the treatment of asthmatic children and adolescents. Pediatr Allergy Immunol, 2015, 26(6): 551-556.
33.	Turner SW, Friend AJ, Okpapi A. Asthma and other recurrent wheezing disorders in children (chronic). BMJ Clin Evid, 2012, 2012.
34.	Milgrom H, Fowler-Taylor A, Vidaurre CF, et al. Safety and tolerability of omalizumab in children with allergic (IgE-mediated) asthma. Curr Med Res Opin, 2011, 27(1): 163-169.
35.	Massanari M, Milgrom H, Pollard S, et al. Adding omalizumab to the therapy of adolescents with persistent uncontrolled moderate--severe allergic asthma. Clin Pediatr (Phila), 2009, 48(8): 859-865.
36.	Chapman KR, Cartier A, Hébert J, et al. The role of omalizumab in the treatment of severe allergic asthma. Can Respir J, 2006, 13(Suppl B): 1B-9B.
37.	Nieto-Cid M, Garriga-Baraut T, Plaza-Martín AM, et al. Cost-effectiveness of omalizumab for the treatment of severe pediatric allergic asthma-results of a real-life study in Spain. Pediatr Allergy Immunol, 2023, 34(4): e13942.
38.	Rodriguez-Martinez CE, Sossa-Briceño MP, Castro-Rodriguez JA. Cost-utility of omalizumab for the treatment of uncontrolled moderate-to-severe persistent pediatric allergic asthma in a middle-income country. Pediatr Pulmonol, 2021, 56(9): 2987-2996.
39.	Pamuk G, Le Bourgeois M, Abou Taam R, et al. The economic burden of severe asthma in children: a comprehensive study. J Asthma, 2021, 58(11): 1467-1477.
40.	Zhou H, Lu Y, Wu B, et al. Cost-effectiveness of omalizumab for the treatment of inadequately controlled severe allergic asthma in Chinese children. J Asthma, 2020, 57(1): 87-94.
41.	Yoshikawa H, Iwata M, Matsuzaki H, et al. Impact of omalizumab on medical cost of childhood asthma in Japan. Pediatr Int, 2016, 58(5): 425-428.
42.	Faria R, McKenna C, Palmer S. Optimizing the position and use of omalizumab for severe persistent allergic asthma using cost-effectiveness analysis. Value Health, 2014, 17(8): 772-782.
43.	深圳政府在线. 深圳市医疗保障局答奥马珠单抗报销. 2023.
44.	国家医疗保障局. 2023年国家医保药品目录调整申报材料(公示版). 2023.

1. 中华医学会儿科学分会呼吸学组, 《中华儿科杂志》编辑委员会. 儿童支气管哮喘诊断与防治指南(2016年版). 中华儿科杂志, 2016, 54(3): 167-181.
2. 全国儿科哮喘协作组, 中国疾病预防控制中心环境与健康相关产品安全所. 第三次中国城市儿童哮喘流行病学调查. 中华儿科杂志, 2013, 51(10): 729-735.
3. Boulet LP, Reddel HK, Bateman E, et al. The global initiative for asthma (GINA): 25 years later. Eur Respir J, 2019, 54(2): 1900598.
4. National Asthma Education and Prevention Program. Expert panel report 3 (EPR-3): guidelines for the diagnosis and management of asthma-summary report 2007. J Allergy Clin Immunol, 2007, 120(5 Suppl): S94-S138.
5. 杨祎, 姜明燕. 儿童支气管哮喘药物治疗研究与进展. 系统医学, 2019, 4(2): 193-195.
6. Global Initiative For Asthma. Global strategy for asthma management and prevention. 2024.
7. 中国医药教育协会慢性气道疾病专业委员会, 中国哮喘联盟. 重度哮喘诊断与处理中国专家共识(2024). 中华医学杂志, 2024, 104(20): 1759-1789.
8. 中华医学会变态反应分会呼吸过敏学组(筹), 中华医学会呼吸病学分会哮喘学组. 中国过敏性哮喘诊治指南(第一版, 2019年). 中华内科杂志, 2019, 58(9): 636-655.
9. Arakawa H, Adachi Y, Ebisawa M, et al. Japanese guidelines for childhood asthma 2020. Allergol Int, 2020, 69(3): 314-330.
10. Holguin F, Cardet JC, Chung KF, et al. Management of severe asthma: a European Respiratory Society/American Thoracic Society guideline. Eur Respir J, 2020, 55(1): 1900588.
11. Asher I, Pearce N. Global burden of asthma among children. Int J Tuberc Lung Dis, 2014, 18(11): 1269-1278.
12. Hong J, Bao Y, Chen A, et al. Chinese guidelines for childhood asthma 2016: major updates, recommendations and key regional data. J Asthma, 2018, 55(10): 1138-1146.
13. Lanier B, Bridges T, Kulus M, et al. Omalizumab for the treatment of exacerbations in children with inadequately controlled allergic (IgE-mediated) asthma. J Allergy Clin Immunol, 2009, 124(6): 1210-1216.
14. 唐密, 张雪艳, 杨燕, 等. 三级医疗机构医院卫生技术评估实施现况. 中国卫生资源, 2023, 26(3): 281-285.
15. COOTHA. Omalizumab as add-on therapy to inhaled steroids for asthma. 2004.
16. NICE. Omalizumab for treating severe persistent allergic asthma. 2013.
17. NIHR. Omalizumab for the treatment of severe persistent allergic asthma: a systematic review and economic evaluation. 2013.
18. 周华, 车大钿. 奥马珠单抗治疗儿童重度过敏性哮喘的快速卫生技术评估. 上海医药, 2020, 41(21): 13-16,24.
19. Lang D, Liu Z, Li D. Safety and tolerability of omalizumab in children with allergic (IgE-Mediated) asthma: a systematic review and meta-analysis. Discov Med, 2023, 35(176): 233-241.
20. Fenu G, La Tessa A, Calogero C, et al. Severe pediatric asthma therapy: omalizumab-a systematic review and meta-analysis of efficacy and safety profile. Front Pediatr, 2023, 10: 1033511.
21. Hanania NA, Niven R, Chanez P, et al. Long-term effectiveness and safety of omalizumab in pediatric and adult patients with moderate-to-severe inadequately controlled allergic asthma. World Allergy Organ J, 2022, 15(10): 100695.
22. Liu L, Zhou P, Wang Z, et al. Efficacy and safety of omalizumab for the treatment of severe or poorly controlled allergic diseases in children: a systematic review and meta-analysis. Front Pediatr, 2022, 10: 851177.
23. 杨琰茗, 任丹阳, 翁稚颖, 等. 奥马珠单抗治疗中-重度过敏性哮喘儿童的文献分析. 海峡药学, 2022, 34(7): 113-117.
24. Busse WW, Szefler SJ, Haselkorn T, et al. Possible protective effect of omalizumab on lung function decline in patients experiencing asthma exacerbations. J Allergy Clin Immunol Pract, 2021, 9(3): 1201-1211.
25. 王亚芹, 樊鹏利, 吕品, 等. 奥马珠单抗治疗儿童及青少年过敏性哮喘的系统评价. 中国药物评价, 2021, 38(2): 111-114.
26. 姜红玉, 李娟, 胡文凤, 等. 奥马珠单抗治疗儿童中重度过敏性哮喘的有效性和安全性系统评价. 中国医院用药评价与分析, 2021, 21(9): 1091-1096.
27. Fu Z, Xu Y, Cai C. Efficacy and safety of omalizumab in children with moderate-to-severe asthma: a meta-analysis. J Asthma, 2021, 58(10): 1350-1358.
28. Henriksen DP, Bodtger U, Sidenius K, et al. Efficacy of omalizumab in children, adolescents, and adults with severe allergic asthma: a systematic review, meta-analysis, and call for new trials using current guidelines for assessment of severe asthma. Allergy Asthma Clin Immunol, 2020, 16: 49.
29. Corren J, Kavati A, Ortiz B, et al. Patient-reported outcomes in moderate-to-severe allergic asthmatics treated with omalizumab: a systematic literature review of randomized controlled trials. Curr Med Res Opin, 2018, 34(1): 65-80.
30. Tortajada-Girbés M, Bousquet R, Bosque M, et al. Efficacy and effectiveness of omalizumab in the treatment of childhood asthma. Expert Rev Respir Med, 2018, 12(9): 745-754.
31. Corren J, Kavati A, Ortiz B, et al. Efficacy and safety of omalizumab in children and adolescents with moderate-to-severe asthma: a systematic literature review. Allergy Asthma Proc, 2017, 38(4): 250-263.
32. Rodrigo GJ, Neffen H. Systematic review on the use of omalizumab for the treatment of asthmatic children and adolescents. Pediatr Allergy Immunol, 2015, 26(6): 551-556.
33. Turner SW, Friend AJ, Okpapi A. Asthma and other recurrent wheezing disorders in children (chronic). BMJ Clin Evid, 2012, 2012.
34. Milgrom H, Fowler-Taylor A, Vidaurre CF, et al. Safety and tolerability of omalizumab in children with allergic (IgE-mediated) asthma. Curr Med Res Opin, 2011, 27(1): 163-169.
35. Massanari M, Milgrom H, Pollard S, et al. Adding omalizumab to the therapy of adolescents with persistent uncontrolled moderate--severe allergic asthma. Clin Pediatr (Phila), 2009, 48(8): 859-865.
36. Chapman KR, Cartier A, Hébert J, et al. The role of omalizumab in the treatment of severe allergic asthma. Can Respir J, 2006, 13(Suppl B): 1B-9B.
37. Nieto-Cid M, Garriga-Baraut T, Plaza-Martín AM, et al. Cost-effectiveness of omalizumab for the treatment of severe pediatric allergic asthma-results of a real-life study in Spain. Pediatr Allergy Immunol, 2023, 34(4): e13942.
38. Rodriguez-Martinez CE, Sossa-Briceño MP, Castro-Rodriguez JA. Cost-utility of omalizumab for the treatment of uncontrolled moderate-to-severe persistent pediatric allergic asthma in a middle-income country. Pediatr Pulmonol, 2021, 56(9): 2987-2996.
39. Pamuk G, Le Bourgeois M, Abou Taam R, et al. The economic burden of severe asthma in children: a comprehensive study. J Asthma, 2021, 58(11): 1467-1477.
40. Zhou H, Lu Y, Wu B, et al. Cost-effectiveness of omalizumab for the treatment of inadequately controlled severe allergic asthma in Chinese children. J Asthma, 2020, 57(1): 87-94.
41. Yoshikawa H, Iwata M, Matsuzaki H, et al. Impact of omalizumab on medical cost of childhood asthma in Japan. Pediatr Int, 2016, 58(5): 425-428.
42. Faria R, McKenna C, Palmer S. Optimizing the position and use of omalizumab for severe persistent allergic asthma using cost-effectiveness analysis. Value Health, 2014, 17(8): 772-782.
43. 深圳政府在线. 深圳市医疗保障局答奥马珠单抗报销. 2023.
44. 国家医疗保障局. 2023年国家医保药品目录调整申报材料(公示版). 2023.

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