一个关于药物经济学的分析模型的Case study研究报告

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1、Development of an Economic Model to Assess the Cost Effectiveness of Asthma Management Strategies,Martin J.Price 1 and Andrew H.Briggs2 1 Global health outcomes, GlaxoSmithKline Research Development, Greenford, Middlesex, United Kingdom 2 Health Economics Research Centre, University of Oxford, Oxfor

2、d, United Kingdom,Pharmaceconomics 2002;20(3):183-194,Disease,Disease:Asthma 慢性发作性疾病 急性症候(不同严重程度)性发作:wheeze, chest tightness, breathlessness, cough, nacturnal waking Treatment: A:salmeterol/flucticasone propionate combination(SFC,沙美特罗/丙酸氟替卡松联合给药)50/100g B:flucticasone propionate(FP,丙酸氟替卡松)100g 实验条件:

3、一天两次,吸入式给药,持续12周,随机, 双盲的临床实验 Treatment success: Asthma control(defined in world-wide asthma management guidelines and in terms of QALYs) Results: 1, Effectiveness:Proportion of successfully controlled weeks per patient: SFC(66%)FP(47%) 2,Cost:Mean weekly direct asthma management cost: SFC(15.77)FP(1

4、1.83) 3, Average incremental cost per successfully controlled week with SFC:20.83 4,PSA(probabilistic sensitivity analysis):ICER(95% uncertainty intervals):- 64.94 to 112.66 if decision maker WTP 45 for an additional successful controlled week, SFC more cost-effective in 80% of the time,Methods:Mark

5、ov model,Health states: 1,Successful control: table I 2,Hospital-managed exacerbation: hospital 3,Primary-care-managed exacerbation: outpatient 4,Sub-optimal control: between “successful control “ and “exacerbation” 5,Treatment failure : discontinue the study treatment Cycle length:1 week Transition

6、 parameters: figure I,Health states: 1,Successful control: table I, well-controlled 代替total control,原因:1,对于一部分病人,不清楚total control 是否是现实的和可以达到的。2,对于某些决策者来说, 2,Hospital-managed exacerbation:哮喘恶化病人在医院接受治疗,一些病人:急救室后回家,另一些病人:住院治疗 3,Primary-care-managed exacerbation:门诊病人接受专业治疗。英国:general practice clinic.美

7、国:urgent care centres 4,Sub-optimal control:成功控制和恶化之间,无法确诊,但又不值得干预 5,Treatment failure :1,由于时间短,哮喘致死率低,而无法使用death作为markov model的absorbing state, 而是用treatment failure 代替。2,而与一般的markov model 不同,处在treatment failure 的病人还会增加成本和效果,因为他们可能会选择另外的治疗方式。3,故使用B:flucticasone propionate(FP,丙酸氟替卡松)100g的成本效果来代替treat

8、ment failure 的成本效果 Cycle length:对于哮喘的周期长度来说,1周是比较合适的。 Transition parameters:定义了健康状态和转变方向,其中任意两个状态是可以相互转化的,只有treatment failure 是absorbing state,是单向的。,Data source,Population: 1,patients with asthma aged 12-70, 2,FEV1(forced expiratory volume in 1 second)40%-85%, 3,receiving treatment corticosteroids (

9、beclomethasone dipropionate (二丙酸倍氯米松)252-420g/day or equivalent) or salmeterol (沙美特罗)42 g/day Grouping: 1, salmeterol/flucticasone propionate combination(SFC,沙美特罗/丙酸氟替 卡松联合给药)50/100g 2, B:flucticasone propionate(FP,丙酸氟替卡松)100g 3, salmeterol(沙美特罗)50g 4,placebo Recording: 1,Patient diary card(symptoms

10、, lung function, nocturnal waking, use of rescue salbutamol(舒喘宁) 2,case report form,Assignment to health states,Successful control(1): well-controlled Hospital-managed exacerbation(2):weekly data examined Primary-care-managed exacerbation(3):weekly data examined Sub-optimal control:not 2 and 3, any

11、of criteria of 1 not met Treatment failure :withdraw from the study for prematurely clinical reasons,Determining transition probabilities(1),1,存在的问题:有些transition 的数量为0,这意味着这些transition很小,而不是不可能发生。 2,采用bayesian methods,Determining transition probabilities(2),Model cost,Medication cost(medication usag

12、e, physician time, hospital costs):2000 UK pharmacy prices(taking from MIMS(Monthly Index of Medical Specialities) Cost associated with Primary and secondary exacerbation health states(from a paper published in 1998) Inflation(HCHS(hospital and community health services) inflation index),Hospital-ma

13、naged exacerbation中,cost采用急救室和住院的平均费用计算,Hoskins et al.,Health outcomes,Outcomes:Proportion of successfully controlled weeks All other states except treatment failure are assigned 0 benefit Costs and Outcomes of Treatment failure states: use FP treatment arm(lacking “usual care” sub-model ),Fitting d

14、istributions,Transition probabilities: dirichlet distribution(multinomial equivalent of the -distribution) Costs: 1,drug: no distribution 2,unit costs of primary care consultations and the cost of an inpatient day: normal distribution 3,the length of stay for a hospital-managed exacerbation: log nor

15、mal distribution 4,costs of primary and secondary care-managed exacerbations: normal distribution Treatment failure: probabilistically(FP arm),Data analysis,Cost-effectiveness PSA(Probabilistic sensitivity analysis),1,控制周数增加比例:0.10.26. 2,成本增加:-10.9717.51每周 3,ICER:-64.94112.66 4,25%SFC处于dominant,75%,

16、SFC效果更好,成本更高 5,WTP=45时,80%SFC的NB0,Discussion,Limitations management exacerbation cost factors usual care cost data QALYs Further improvement usual care prior distribution asthma control QALYs,限制: ,恶化成本因素(临床实验时间短) ,一般治疗使用了FP arm代替 ,成本数据来源于不同的病人群体 ,的效用值说服力不够(个哮喘病人,轻度中度,平均效用值医院.,门诊.,次级健康. 进一步研究: ,组可以用来作为一般治疗 ,先验性的数据可以代替根据

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