[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. |