药物经济学基本概念与基本研究方法简介

药物经济学基本概念

与基本研究方法简介张延军2015年7月3日上海概要卫生经济学和健康结果研究，HealthEconomicsandOutcomesResearch(HEOR)卫生技术评估,HealthTechnologyAssessment(HTA)药物经济学，Pharmacoeconomics视角(perspective)、成本分类、时间范围(timehorizon)、贴现(discounting)、敏感性分析药物经济学研究的基本方法成本最小化分析,cost-minimizationanalysis(CMA)成本-效果分析,cost-effectivenessanalysis(CEA)成本-效用分析,cost-utilityanalysis(CUA)成本-效益分析,cost-benefitanalysis(CBA)疾病成本研究,costofillness预算影响分析,budgetimpactanalysis(BIA)临床试验(RCT)、数据库(database)决策树,decisiontree;马可夫模型,MarkovmodelTreeAge,MSExcel,R...合理用药与药品价值合理用药：安全、有效、经济临床价值+经济价值安全性疗效（临床试验、临床实践）成本-效果比Demonstratingproductvalue安全性:Benefit/risk(harm)eg,苯丙醇胺（PPA）、罗非昔布（万络，Vioxx）经济性，Cost-effectiveness/journals/cpm/2008/04-April%202008/023-IMS.pdfDemonstratingproductvalue–cont’dEfficacy:疗效（随机对照临床试验，RCT）“Caninterventionworkintheidealstudysetting?”Effectiveness:效果（真实世界数据、证据）“Doesitwork,generalizedtoreal-worldsettingsandappliedtoindividualpatients?”Cost-effectiveness“Isitworthitandshoulditbepaidfor?”Source:Djulbegovic,B.andA.Paul,Fromefficacytoeffectivenessinthefaceofuncertainty:indicationcreepandpreventioncreep.JAMA,2011.305(19):p.2005-6.EBM,HTA,andCERSource:LuceBR,DrummondM,JonssonB,etal.EBM,HTA,andCER:clearingtheconfusion.MilbankQ2010;88:256-76.HealthEconomicsandOutcomesResearchWhyHEORandPE?更加有效地配置有限的卫生资源（少花钱，多办事，办好事；性价比高）健康结果(Healthoutcomes)：ECHO模型人文结果，又称健康相关生活质量(HealthRelatedQualityofLife,HRQoL)及患者报告的健康结果(Patient-ReportedOutcomes,PROs)ClinicalOutcomesHumanisticOutcomesEconomicOutcomesHRQoL研究表明在结直肠癌继发肝癌患者中，健康相关生活质量(HRQoL)是比CT扫描和肿瘤大小测定都更强及更好的生存预测因素。1HRQoL在慢性髓性白血病(CML)研究中已经成为一个重要的研究兴趣。2EarlamS,GloverC,FordyC,BurkeD,Allen-MershTG.Relationbetweentumorsize,qualityoflife,andsurvivalinpatientswithcolorectallivermetastases.JClinOncol1996;14:171–175.Guilhot,J.,etal.,Definitions,methodologicalandstatisticalissuesforphase3clinicaltrialsinchronicmyeloidleukemia:aproposalbytheEuropeanLeukemiaNet.Blood,2012.119(25):p.5963-71.Pharmacoeconomics

Definition药物经济学是对药物治疗相对于医药卫生系统及社会的成本（和结果）的描述和分析。药物经济学研究识别、测量、比较药品和药学服务的成本（即消费的资源）和结果。TownsendRJ,PostmarketingDrugResearchandDevelopmentDrugIntellClinPharm,1987:21COSTS($)RXOUTCOMESCosts=costsassociatedw/providingpharmacyproductorserviceRx

=pharmacyproductorserviceOutcomes=outcomeoftheproductorservicePharmacoeconomicsRelationshipofPharmacoeconomicstoOtherResearchPharmacy-relatedclinicalorhumanisticoutcomesresearch

Healthcareeconomics

Pharmaco-economicsWhentodoeconomicevaluation?Source:CoxER,SchafermeyerKW.Chapter18--Pharmacoeconomics.In:McCarthyRL,SchafermeyerKW,eds.IntroductiontoHealthCareDelivery:APrimerforPharmacists.Sudbury,MA:JonesandBartlettPublishers;2007:481-508.成本：

研究视角、分类、贴现社会Society患者Patient医院Hospital保险商Insurer政府GovernmentHealthCareProfessionalPerspective药物经济学评价可能的研究视角成本(cost)的分类成本分为：直接成本直接医疗成本(directmedicalcost)直接非医疗成本(directnon-medicalcost)间接成本(indirectcost)无形成本/隐性成本(intangiblecost)另一成本分类：卫生服务部门的成本(coststohealthcaresector)其他部门的成本(coststoothersectors)患者及其家庭的成本(patientandfamilycosts)生产力成本(productivitycosts)视角/角度(perspective)直接医疗成本(DirectMedicalCosts)Medications药品Medicationmonitoring药物监测Medicationadministration给药Patientcounseling&consultations患者咨询Diagnostictests诊断测试Hospitalizations住院（含ICU）Clinicvisits门诊/诊所EmergencyDept.visits急诊Homemedicalvisits家庭医疗服务Ambulanceservices救护车直接非医疗成本(DirectNonmedicalCosts)Travelcoststoreceivehealthcare差旅Nonmedicalassistancerelatedtocondition非医疗救助，如对残疾人患者的生活服务等Hotelstaysforpatientorfamilyrelatedtocondition住宿Childcareservicesforchildrenofparents由于父/母患病而对未成年人的监护间接成本(IndirectCosts)Nomoneyisexchanged.没有金钱的交换Lostproductivityforpatient患者失去的生产力Lostproductivityforunpaidcaregiver未支付报酬的护理人员失去的生产力Lostproductivitybecauseofprematuremortality早亡而导致的失去生产力隐性成本(IntangibleCosts)Painandsuffering疼痛和痛苦Fatigue疲乏Anxiety焦虑金钱的时间价值今天借出￥10,000,对方5年后归还￥10,000Inflation?Deflation?Stagflation?通货膨胀/通货紧缩/滞胀（经济停滞、高通胀）Discountrate（贴现率）将不同年份的金钱价值转化为当前价值“如果疾病治疗的时间超过一年，就应该对成本进行贴现。贴现率（DiscountRate）一般为市场利率，建议采用一年期的国家指导利率或国债利率进行贴现。贴现率应该进行敏感性分析，波动范围建议在0%~8%之间。”*国家统计局发布的CPI及GDP折算Source:中国药物经济学评价指南.http:///PEguidelines/source/China-Guidelines-for-Pharmacoeconomic-Evaluations_2011_Chinese.pdfAnExampleTotalhospitalcostsin2004$22,538.10Convertingto2007USDMedicalComponentofCPI2004=4.4% 2005=4.3%2006=4.1% 2007=4.1%Formula$???in2007USD药物经济学研究的基本方法PharmacoeconomicMethodologiesMethodofAnalysisCostOutcomeMeasurementCostminimization$AssumedequalCostbenefit$$/$(netbenefit)Costeffectiveness$$/outcome-goalCostutility$$/qualityoflifeindex[Quality-AdjustedLifeYear(QALY)]质量调整生命年(QALY)QALY等于剩余的生命年数乘以这段时间内的健康效用值综合反映生命的“质”和“量”的变化计算QALY的关键在健康效用值的测量上健康效用值的测量工具：直接间接：欧洲五维健康量表（EuroQol-5Dimensions，EQ-5D）、短表36（shortform36,SF-36）及疾病专用量表等HRQoL/PROHRQoL包括对身体、社交、职业、心理、情绪等方面的健康调查。HRQoL是而且应当是患者报告的健康情况，而不能是由医药卫生专业人员通过她/他的主观判断而进行的测定。Patient-ReportedOutcomeMeasures:UseinMedicalProductDevelopmenttoSupportLabelingClaimshttp:///downloads/Drugs/Guidances/UCM193282.pdfHRQoL通用和特定疾病专用量表SF-36,EQ-5D;FunctionalAssessmentofCancerTherapy-General(FACT-G),CancerTherapySatisfactionQuestionnaire(CTSQ),MDAndersonSymptomInventory-CML(MDASI-CML)“当没有相应中文版量表时应按规定先进行汉化，汉化完后才可以使用。汉化时需要依据翻译（Translation）、回译（BackTranslation）、文化调试（CultureAdjustment）和跨文化验证（Cross-cultureValidation）等步骤来进行”(Ref:中国药物经济学评价指南.2011)http:///PEguidelines/index.aspApproachestoConductingPEStudiesRandomizedcontrolledtrialsDecisionanalysisInformationpooledfrommultiplesources(e.g.,RCT,NIR,expertopinion,…)Twomodels:decisiontree&MarkovmodelRetrospectiveanalysis(claimsdatabase)InformationsourcesSystematicreview,meta-analysis(Lancet,JAMA,BMJ,AnnInternMed...)(RC)T(NEJM...)Cohortstudy,case-controlstudy(database...)NIR=non-interventionalresearch,orobservationalresearch（非干预性研究，观察性研究）Caseseries,casereportExpertopinions/wiki/Research_designDatabasesMEPS(MedicalExpenditurePanelSurvey,/mepsweb/)NHANES(NationalHealthandNutritionExaminationSurvey)Claimsdatabases(MarketScan®，中国医保数据...)GPRD(GeneralPracticeResearchDatabase,1994–2012,followedbyCPRD,ClinicalPracticeResearchDatalink)台湾全民健康保险（全民健保）研究数据库(Taiwan’snationalhealthinsuranceresearchdatabase,NHIRD)*ElectronicHealth/MedicalRecord(EHR/EMR)NationalDrugCode(NDC),ICD-9/ICD-10,CurrentProceduralTerminology(CPT)...ChenYC,WuJC,ChenTJ,WetterT.Apubliclyavailabledatabaseacceleratesacademicproduction.BMJ.2011;342:d637.Databases–cont’dDeidentified(identifier?)InstitutionalReviewBoard(IRB)/EthicalCommitteereviewandapprovalSAS,SPSS,Stata...Demographic(age,gender),socioeconomic(employment,income,healthinsurance)datasetsMedicalconditions,healthservices(outpatientvisit,emergencyroomvisit,hospitalization,expense),pharmacydatasetsDocumentation,codebook...DecisiontreeSource:CoxER,SchafermeyerKW.Chapter18--Pharmacoeconomics.In:McCarthyRL,SchafermeyerKW,eds.IntroductiontoHealthCareDelivery:APrimerforPharmacists.Sudbury,MA:JonesandBartlettPublishers;2007:481-508.Decisiontree–cont’dDrugCPhysicianofficevisitSour