定量药品获益-风险评价方法研究及启示

doi:10.19803/j.1672-8629.2021.07.12

中国药物警戒 ›› 2021, Vol. 18 ›› Issue (7): 649-654.
DOI: 10.19803/j.1672-8629.2021.07.12

定量药品获益-风险评价方法研究及启示

郭志坚, 胡方圆, 郑轶, 梁际洲, 许金芳, 郭晓晶, 叶小飞, 贺佳^*

海军军医大学卫生勤务学系卫生统计学教研室,上海200433

收稿日期:2020-06-19 出版日期:2021-07-15 发布日期:2021-07-23
通讯作者: ^*贺佳,女,教授•博导,药物流行病学及新药评价的统计分析。E-mail:hejia63@yeah.net;^#为共同通信作者。
作者简介:郭志坚,男,硕士,药物流行病学。
基金资助:
国家自然科学基金资助项目（82073671）; 上海市自然科学基金资助项目（18ZR1449500）; 上海市第四轮公共卫生三年行动计划重点学科建设项目循证公共卫生与卫生经济学(15GWZK0901); 上海市卫计委优秀青年医学人才培养计划（2018YQ47）; 上海市公共卫生学科带头人（GWV-10.2-XD22）; 上海市公共卫生优青计划（GWV-10.2-YQ33）

Current Research on Quantitative Drug Benefit-Risk Assessment Methods and Its Englightenment

GUO Zhijian, HU Fangyuan, ZHENG Yi, LIANG Jizhou, XU Jinfang, GUO Xiaojing, YE Xiaofei, HE Jia^*

Department of Medical Statistics, Faculty of Medical Services, Naval Medical University, Shanghai 200433, China

Received:2020-06-19 Online:2021-07-15 Published:2021-07-23

摘要/Abstract

摘要： 目的介绍目前定量药品获益-风险评价方法的基本原理和最新进展。方法查阅目前药品获益-风险评价相关指导原则和系统检索中外有关定量药品获益-风险评价方法文献进行综述。结果简要介绍几种理论依据较为充分的不同类别的定量药品获益-风险评价方法,分析和讨论定量药品获益-风险评价方法的应用现状和发展趋势。结论目前在定量药品获益-风险评价方法的选择与使用上尚未形成统一共识,较为新颖的方法尚未得到广泛应用,仍需真实世界药品评价的进一步检验。

关键词: 药品, 定量, 获益-风险评价, 方法, 药品不良反应

Abstract: Objective To introduce the basic principles and latest developments of current quantitative drug benefit-risk assessment methods. Methods Current guidelines for drug benefit-risk assessment were consulted while domestic and foreign literature on quantitative drug benefit-risk assessment methods was retrieved to conduct a literature review. Results Several well-grounded quantitative benefit-risk assessment methods were outlined. The current applications of quantitative benefit-risk assessment methods of drugs were analyzed and the developments of quantitative assessment methods were explored. Conclusion There is no specific consensus on the selection and use of quantitative drug benefit-risk assessment methods. The relatively novel methods have not been widely used and need to be verified and confirmed in the real drug benefit-risk assessment context.

Key words: drug, quantitative, benefit-risk assessment, method, adverse drug reaction

中图分类号:

R971.4R969.1

郭志坚, 胡方圆, 郑轶, 梁际洲, 许金芳, 郭晓晶, 叶小飞, 贺佳. 定量药品获益-风险评价方法研究及启示[J]. 中国药物警戒, 2021, 18(7): 649-654.

GUO Zhijian, HU Fangyuan, ZHENG Yi, LIANG Jizhou, XU Jinfang, GUO Xiaojing, YE Xiaofei, HE Jia. Current Research on Quantitative Drug Benefit-Risk Assessment Methods and Its Englightenment[J]. Chinese Journal of Pharmacovigilance, 2021, 18(7): 649-654.

参考文献

[1] International Conference on Harmonisation. ICH Harmonised Tripartite Guideline, Periodic Benefit-Risk Evaluation Report (PBRER) E2C (R2), 2012 [EB/OL]. (2012-12-17)[2020-11-19]. http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/ E2C/E2C_R2_Step4.pdf.
[2] Hammad TA, Pinto CA.Key changes in benefit risk assessment guidelines and implications for data analysis in drug development[J]. Statistics in Biopharmaceutical Research, 2016, 8(4): 366-372.
[3] Mt-Isa S, Hallgreen CE, Wang N, et al.Balancing benefit and risk of medicines: a systematic review and classification of available methodologies[J]. Pharmacoepidemiol Drug Saf, 2014, 23(7): 667-678.
[4] Hughes D, Waddingham E, Mt-Isa S, et al.Recommendations for benefit-risk assessment methodologies and visual representations[J]. Pharmacoepidemiol Drug Saf, 2016, 25(3): 251-262.
[5] Zhang XM, Jin YN, Zhang B, et al.Aconitum in treatment of rheumatoid arthritis: benefit-risk assessment[J]. China Journal of Chinese Materia Medica(中国中药杂志), 2018, 43(2): 234-241.
[6] Marcelon L, Verstraeten T, Dominiak-Felden G, et al.Quantitative benefit-risk assessment by MCDA of the quadrivalent HPV vaccine for preventing anal cancer in males[J]. Expert Rev Vaccines, 2016, 15(1): 139-48.
[7] De Greef-Van Der SI, Newgreen D, Schaddelee M, et al. A quantitative benefit-risk assessment approach to improve decision making in drug development: application of a multicriteria decision analysis model in the development of combination therapy for overactive bladder[J]. Clin Pharmacol Ther, 2016, 99(4): 442-451.
[8] Zhang XM, Lin ZJ, Zhang B, et al.Comparison of aconitum with tripterygium in treatment of axial spondylo-arthritis: a benefit-risk assessment[J]. Chinese Journal of Pharma-covigilance(中国药物警戒), 2018, 15(2): 103-109.
[9] Waddingham E, Mt-Isa S, Nixon R, et al.A bayesian approach to probabilistic sensitivity analysis in structured benefit-risk assessment[J]. Biometrical Journal, 2016, 58(1) : 28-42.
[10] Morton A.Treacle and smallpox: two tests for multicriteria decision analysis models in health technology assessment[J]. Value Health, 2017, 20(3): 512-515.
[11] Saint-Hilary G, Robert V, Gasparini M, et al.A novel measure of drug benefit-risk assessment based on scale loss score[J]. Stat Methods Med Res, 2019, 28(9): 2738-2753.
[12] Tervonen T, Van Valkenhoef G, Buskens E, et al.A stochastic multicriteria model for evidence-based decision making in drug benefit-risk analysis[J]. Statistics in Medicine, 2011, 30(12): 1419-1428.
[13] Zhang JC, Yu H, Huang LH, et al.Benefit-risk assessment of oral anticoagulants for stroke prevention in atrial fibrillation[J]. Chinese Journal of Health Statistics(中国卫生统计), 2016, 33(4): 612-615.
[14] Wang Y, Mai Y, He W.A quantitative approach for benefit-risk assessment using stochastic multi-criteria discriminatory method[J]. Statistics in Biopharmaceutical Research, 2016, 8(4): 373-378.
[15] Van Valkenhoef G, Tervonen T, Zhao J, et al.Multicriteria benefit-risk assessment using network meta-analysis[J]. Journal of Clinical Epidemiology, 2012, 65(4): 394-403.
[16] Saint-Hilary G, Cadour S, Robert V, et al.A simple way to unify multicriteria decision analysis (MCDA) and stochastic multicriteria acceptability analysis (SMAA) using a Dirichlet distribution in benefit-risk assessment[J]. Biometrical Journal, 2017, 59(3): 567-578.
[17] Costa MJ, He W, Jemiai Y, et al.The case for a bayesian approach to benefit-risk assessment:overview and future directions[J]. Ther Innov Regul Sci, 2017, 51(5): 568-574.
[18] Costa M J, Drury T.Bayesian joint modelling of benefit and risk in drug development[J]. Pharm Stat, 2018, 17(3): 248-263.
[19] Li K, Yuan SS, Wang W, et al.Periodic benefit-risk assessment using Bayesian stochastic multi-criteria acceptability analysis[J]. Contemporary Clinical Trials, 2018, 67(2): 100-108.
[20] Sutton AJ, Cooper NJ, Abrams KR, et al.A bayesian approach to evaluating net clinical benefit allowed for parameter uncertainty[J]. J Clin Epidemiol, 2005, 58(1): 26-40.
[21] Nixon R, Dierig C, Mt-Isa S, et al.A case study using the PrOACT-URL and BRAT frameworks for structured benefit risk assessment[J]. Biom J, 2016, 58(1): 8-27.
[22] Citrome L, Ketter TA.When does a difference make a difference? interpretation of number needed to treat, number needed to harm, and likelihood to be helped or harmed[J]. Int J Clin Pract, 2013, 67(5): 407-411.
[23] Catt H, Bodger K, Kirkham JJ, et al.Value assessment and quantitative benefit-risk modelling of biosimilar infliximab for Crohn's disease[J]. PharmacoEconomics, 2019, 37(12): 1509-1523.
[24] Huang M, Pietanza MC, Samkari A, et al.Q-TWiST analysis to assess benefit-risk of pembrolizumab in patients with PD-L1-positive advanced or metastatic non-small cell lung cancer[J]. PharmacoEconomics, 2019, 37(1): 105-116.
[25] Levitan B, Phillips LD, Walker S.Structured approaches to benefit-risk assessment: a case study and the patient perspective[J]. Ther Innov Regul Sci, 2014, 48(5): 564-573.
[26] Evans SR, Follmann D.Using outcomes to analyze patients rather than patients to analyze outcomes: a step toward pragmatism in benefit:risk evaluation[J]. Statistics in Biopharmaceutical Research, 2016, 8(4): 386-393.
[27] Irony T, Ho M, Christopher S, et al.Incorporating patient preferen-ces into medical device benefit-risk assessments[J]. Statistics in Biopharmaceutical Research, 2016, 8(3): 230-236.
[28] Byun JH, Kwon SH, Ha JH, et al.A benefit-risk assessment model for statins using multicriteria decision analysis based on a discrete choice experiment in Korean patients[J]. Ther Clin Risk Manag, 2016, 12(1): 965-974.
[29] Reed Johnson F, Lancsar E, Marshall D, et al.Constructing experimental designs for discrete-choice experiments: report of the ISPOR conjoint analysis experimental design good research practices task force[J]. Value Health, 2013, 16(1): 3-13.
[30] Mukhopadhyay S, Dilley K, Oladipo A, et al.Hierarchical bayesian benefit-risk modeling and assessment using choice based conjoint[J]. Statistics in Biopharmaceutical Research, 2019, 11(1): 52-60.
[31] Vass CM, Payne K.Using discrete choice experiments to inform the benefit-risk assessment of medicines: are we ready yet?[J]. PharmacoEconomics, 2017, 35(9): 859-866.
[32] Belton V, Stewart T.Multiple criteria decision analysis: an integrated approach[M]. Springer Science & Business Media, 2002.
[33] Li K, Luo S, Yuan S, et al.A Bayesian approach for individual-level drug benefit-risk assessment[J]. Stat Med, 2019, 38(16): 3040-3052.
[34] Johnson FR, Zhou M.Patient preferences in regulatory benefit-risk assessments: a US perspective[J]. Value Health, 2016, 19(6): 741-745.
[35] Muhlbacher AC, Juhnke C, Beyer AR, et al.Patient-focused benefit-risk analysis to inform regulatory decisions: The European Union Perspective[J]. Value Health, 2016, 19(6): 734-740.

定量药品获益-风险评价方法研究及启示

Current Research on Quantitative Drug Benefit-Risk Assessment Methods and Its Englightenment

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