参考文献
[1]Chen W,Zhang L,Hu M,et al.Use of health technology assessment in drug reimbursement decisions in China[J].BMJ,2023,381:e068915.DOI:10.1136/bmj-2021-068915.
[2]Hauser SL,Bar-Or A,Cohen JA,et al.Ofatumumab versus Teriflunomide in Multiple Sclerosis[J].New England Journal of Medicine,2020 Aug 6;383(6):546-557.
[3]Palace J,Bregenzer T,Tremlett H,et al.UK multiple sclerosis risk-sharing scheme:a new natural history dataset and an improved Markov model[J].BMJopen,2014,4(1):e004073.
[4]Scandinavica AN.Course of multiple sclerosis.First results of a prospective study carried out of 102 MS patients from 1976-1980[J].Acta Neurologica Scandinavica,2010,65(4):248-266.
[5]Orme M,Kerrigan J,Tyas D,et al.The effect of disease,functional status,and relapses on the utility of people with multiple sclerosis in the UK[J].Value in Health,2007,10(1):54-60.
[6]Confavreux C,O'Connor P,Comi G,et al.Oral teriflunomide for patients with relapsing multiple sclerosis(TOWER):a randomised,double-blind,placebo-controlled,phase 3 trial[J].The Lancet.Neurology,2014,13(3):247-256.
[7]Samjoo IA,Worthington E,Drudge C,et al.Comparison of ofatumumab and other diseasemodifying therapies for relapsing multiple sclerosis:a network meta-analysis[J].Journal of Comparative Effectiveness Research,2020,9(18):1255-1274.
[8]NLM.Economic Review[M/OL].In:Pharmacoeconomic report:ofatumumab(kesimpta):(novartis pharmaceuticals Canada Inc.):indication:multiple sclerosis,relapsing-remitting[internet].https://www.ncbi.nlm.nih.gov/books/NBK572535/.
[9]NICE.Ocrelizumab for treating primary progressive multiple sclerosis[EB/OL].(2019-06-12)[2023-08-31].https://www.nice.org.uk/guidance/TA585.
[10]Acaster S,Perard R,Chauhan D,et al.A forgotten aspect of the NICE reference case:an observational study of the health related quality of life impact on caregivers of people with multiple sclerosis[J].Bmc Health Services Research,2013,13(1):346.
[11]孙宇航,王萍.罕见病患者医疗保障的伦理思考[J].中国医学伦理学,2019,32(9):1143-1146.
[12]Friedmann C,Levy P,Hensel P,et al.Using multi-criteria decision analysis to appraise orphan drugs:a systematic review[J].Expert Review of Pharmacoeconomics&Outcomes Research,2018,18(2):135-146.
[13]北京协和医院罕见病多学科协作组,中国罕见病联盟.多准则决策分析应用于罕见病药品临床综合评价的专家共识(2022)[J].协和医学杂志,2022,13(2):235-254.
[14]Kolasa K,Zwolinski KM,Kalo Z,et al.Potential impact of the implementation of multiplecriteria decision analysis(MCDA)on the Polish pricing and reimbursement process of orphan drugs[J].Orphanet Journal of Rare Diseases,2016,11(1):1-12.
[15]Stafinski T,Glennie J,Young A,et al.HTA decision-making for drugs for rare diseases:comparison of processes across countries[J].Orphanet Journal of Rare Diseases,2022,17(1):258.
[16]Korchagina D,Rémuzat C,Rodrigues J,et al.Health technology assessment,price and reimbursement review for orphan drugs In France[J].Value in Health,2014,17(7):A540.(https://www.daowen.com)
[17]Nicod E,Annemans L,Bucsics A,et al.HTA programme response to the challenges of dealing with orphan medicinal products:Process evaluation in selected European countries[J].Health Policy,2019,123(2):140-151.
[18]Nicod E,Jd KBB,Durand-Zaleski I,et al.Dealing with uncertainty and accounting for social value judgments in assessments of orphan drugs:evidence from four european countries[J].Value in Health,2017,20(7):919-926.
[19]Ollendorf DA,Chapman RH,Pearson SD.Evaluating and valuing drugs for rare conditions:no easy answers[J].Value in Health,2018,21(5):547-552.
[20]Kawalec P,Sagan A,Pilc A.The correlation between HTA recommendations and reimbursement status of orphan drugs in Europe[J].Orphanet Journal of Rare Diseases,2016,11(1):122.
[21]FIT Consulting.Challenges in preserving access to orphan drugs under an HTA framework[R/OL].(2021-12-02)[2023-06-26].https://www.fticonsulting.com/insights/reports/-/media/285673be00f84138ab72fad286b4f369.ashx.
[22]宣建伟,孙巧.中国罕见病药物经济学评估适用模型与支付阈值参考标准探讨[J].国际药学研究杂志,2019,46(9):659-665.
[23]GLI.Pricing&Reimbursement Laws and Regulations|Korea[R].(2021-08-17)[2022-03-17].https://www.globallegalinsights.com/practice-areas/pricing-and-reimbursementlaws-and-regulations/korea.
[24]Lee JH.Pricing and reimbursement pathways of new orphan drugs in South Korea:a longitudinal comparison[J].Healthcare,2021,9(3):296.
[25]Fukuda T.Drug Pricing in Japan[M].Tokyo:Center for Outcomes Research and Economic Evaluation for Health National Institute of Public Health,2018.
[26]GLI.Pricing&Reimbursement Laws and Regulations|Japan[EB/OL].In:GLI.Global Legal Insights-International legal business solutions.https://www.globallegalinsights.com/practice-areas/pricing-and-reimbursement-laws-and-regulations/japan.
[27]Martin Wenzl,Valérie Paris.Pharmaceutical Reimbursement and Pricing in Germany[R/OL].(2018-06)[2023-09-18].https://www.oecd.org/health/health-systems/Pharmaceutical-Reimbursement-and-Pricing-in-Germany.pdf.
[28]Gammie T,Lu CY,Babar UD,et al.Access to orphan drugs:a comprehensive review of legislations,regulations and policies in 35 countries[J].Plos One,2015,10(10):e0140002.
[29]顾一纯,张琴华,黄镇,等.韩国高值罕用药使用保障机制[J/OL].中国卫生资源,2021,24(6):641-645,825.
[30]Bae EY.Role of health technology assessment in drug policies:Korea[J].Value in Health Regional Issues,2019,18:24-29.
[31]Co-Chair LPG,Co-Chair ATMM,Andrew Briggs D,et al.Performance-based risk-sharing arrangements—good practices for design,implementation,and evaluation:report of the ISPOR good practices for performance-based risk-sharing arrangements task force[J].Value in Health,2013,16(5):703-719.