卫生技术评估 Health Technology Assessment

卫生技术评估Health Technology Assessment 复旦大学医学技术评估研究中心陈英耀陈洁

Overview 提纲 • 卫生技术和卫生技术评估概述 • 卫生技术评估的内容 • 卫生技术评估的步骤 • 各国卫生技术评估发展回顾 • 我国卫生技术评估的发展国际华夏医药学会－陈英耀发言

一、卫生技术和卫生技术评估概述

Technology Definitions 技术定义 Technology (gr - tekhné) 技术 • The application of science to the arts 科学应用的艺术 • Art - skill, especially human skill opposed to nature 艺术-技能，尤其是不同于自然的人类技能 • Science - as systematic and formulated knowledge 科学，系统化和程式化的知识 • The systematic application of scientific or other organized knowledge to practical tasks 将科学和其它有组织的系统知识运用到实践国际华夏医药学会－陈英耀发言

Medical Device Procedure Knowledge Drugs Support system Components of HT卫生技术的组成 Equipment 国际华夏医药学会－陈英耀发言

Health Technology Definition卫生技术的定义 • Health Technology is considered to include the drugs, devices, medical and surgical procedures used in healthcare delivery, the knowledge associated with this, as well as the organisational and support systems within which such care is provided • 卫生技术是指用于医疗保健的药物，仪器设备，内、外科程序及相关的组织管理系统和支持系统的特定的知识体系国际华夏医药学会－陈英耀发言

Three ways to describe HT从三个方面描述卫生技术 Physical nature 物理性质 Clinical purpose 临床目的 Stage of diffusion 传播阶段

Technology: physical nature 技术：物理特性 Drugs药物 Aspirin阿斯匹林 Antibiotics抗生素 Vaccines疫苗 Devices, equipment, supplies 仪器设备和物资供应 Cardiac pacemaker心脏起搏器 Computed Topography scanner CT Surgical gloves外科手套 Medical and surgical procedures内外科诊疗程序 Acupuncture针灸 Cancer chemotherapy癌症化疗 Cesarean section剖腹产手术 Support systems支持系统 Drug formulary药物报销目录 Clinical laboratory临床实验室 Patient record system病案系统 Organizational, delivery, and managerial systems组织管理和服务提供系统 Vaccination program免疫计划 Clinical pathway临床路径 Health care payment systems健康保健支付系统

Technology: Clinical purpose技术：临床目的 预防 Prevention 筛检 Screening 诊断 Diagnosis 治疗 Treatment 康复 Rehabilitation

Technology: Stage of diffusion技术：传播阶段 Future 未来 Experimental (Laboratory or animal testing) 试验阶段（实验室或动物试验） Investigational (Clinical studies) 观察阶段（临床研究） Established (Standard approach) 应用阶段（标准方法） Obsolete 淘汰

Technology Life Cycle 技术寿命周期 David & Judd, Medical Technology Management, 1993

Manufacture 生产 Marketing 市场 PROVISION 提供 Testing 实验 Transfer 转让 Development 发展 Distribution 分配 Research 研究 Assessment of Needs 需要评估 De-commissioning 报废 Technology Assessment 技术评估 Evaluation 评价 Maintenance 维护 Planning 规划 Training 培训 Procurement 获得 Operation 操作 Installation 安置 UTILISATION 使用 ACQUISITION 获得 Commissioning 使用 Healthcare Technology Life Cycle卫生技术的生命周期国际华夏医药学会－陈英耀发言

Why HTA is necessary?卫生技术评估的必要性 • Effective therapies were rare until recently • 60% of the remedies in 1927 textbook of medicine as harmful, dubious, or merely symptomatic, while 3% provided fully effective treatment or prevention • By 1975, effective regimens increased sevenfold and dubious ones decreased by two-thirds • It was not until Bradford Hill formulated the principles of the RCT, testing a vaccine for pertussis in the mid 1930 • The most important problem was lack of a control or comparison group to assure the observed effect was due to the intervention 国际华夏医药学会－陈英耀发言 • Beeson in 1980

Why HTA is necessary?卫生技术评估的必要性 Visibility of new technologies 新技术的广泛使用 High expenditures for health care 医疗费用的不断上涨 Necessity to begin to rationalize health care technology 卫生技术资源优化的必要性

Why HTA is necessary?卫生技术评估的必要性 技术是一把双刃剑（两重性）利技术创新改善医疗质量提高人群健康弊技术带来的负面后果伦理争论医疗费用过度增长

What is HTA?什么是卫生技术评估？ • 卫生技术评估是对卫生技术的性质，效果和其他影响进行系统评价 • 卫生技术评估的主要目的是给卫生技术领域的政策制订提供信息 • 卫生技术评估可以针对技术直接的、预期的后果，也能处理间接的、非预期的后果 • 卫生技术评估由多学科团体来完成（不同专业领域的专家一起工作） • 卫生技术评估采用明确的分析框架和多种方法国际华夏医药学会－陈英耀发言

二、卫生技术评估的内容 国际华夏医药学会－陈英耀发言

Properties And Impacts Assessed技术评估的内容 Technical properties 技术特性 Safety安全性 Efficacy and effectiveness功效和效果 Cost and other economic attributes经济性 Social, legal, ethical, or political impacts社会、法律、伦理道德和政治影响

安全性 • Rezulin’s slow removal in the US • Dec 1997: Rezulin taken off in the UK • By Mar 1999: 400 patients suffered liver failure • Mar 2000: FDA withdraw the Rezulin in the US • Four safety labeling changed in almost 2 years • liver functions monitored by blood test every 2-3 months during the first half-year use • test monthly for the first half-year • test monthly in the first 8 month of use • test monthly for the entire first year 国际华夏医药学会－陈英耀发言

The stages of pharmaceutical R&D 药品研发的阶段

Efficacy and Effectiveness功效与效果 功效是指在理想情况下，使用某项技术对某一特定的健康问题所带来的效益，如在随机对照试验中或在著名医学中心效果：在一般情况下或常规情况下使用某项技术对某一特定的健康问题所带来的效益, 如在社区医院中测量指标死亡率(死亡率、期望寿命等) 发病率生命质量失能调节生命年或失能调节期望寿命应用：FDA的药品审批

Clinical Epidemiology / EBHC Iterative Measurement Loop 1 BURDEN OF ILLNESS Efficacy? 2 COMMUNITY EFFECTIVENESS 6 REASSESSMENT *Needs Based *Action oriented *Systematic subsets *Populations and individuals *Different Methods 5 MONITORING OF PROGRAM 3 ECONOMIC EFFICIENCY 4 KNOWLEDGE TRANSLATION & IMPLEMENTATION 国际华夏医药学会－陈英耀发言

Effectiveness [not Efficacy]效果而非功效 Health Provider Systems Compliance Patient Subject Concordance Effectiveness = Efficacy x x x Coverage DOES it work in reality? Can it Work in Ideal Circumstances? 国际华夏医药学会－陈英耀发言

经济性 比较和选择 在给定的情况下选择何种临床治疗方案  肾衰病人是选择肾移植还是肾透析  干预的时间选择  高血压防治：人群筛检、健康促进项目  在什么地点提供服务 医院、社区、还是在病人家里国际华夏医药学会－陈英耀发言

经济性 针对不同卫生问题的可选方案  流感免疫还是建立冠心病监护中心  同一方案的不同应用规模  某免疫方案是针对高危人群还是整个人群  提高健康水平的不同途径  控制污染、加强公路安全保障还是采用其他的卫生技术国际华夏医药学会－陈英耀发言

Model of economic evaluation经济学评价的模型 国际华夏医药学会－陈英耀发言

经济学评价解决什么问题？ • Rising expenditure for health care • payment decision • insurance coverage • Economic analysis is a valuable tool in decision making, but it is only one factor 国际华夏医药学会－陈英耀发言

Social impact社会影响 社会、伦理、道德和法律等正负影响 genetic testing fertility treatment computer-based record system transplantation of scarce organs brain death

三、卫生技术评估的步骤 国际华夏医药学会－陈英耀发言

A Process for HTA卫生技术评估的过程 Identification Testing Synthesis Dissemination Banta, D. Health care technology and its assessment 国际华夏医药学会－陈英耀发言

Ten Steps of HTA卫生技术评估的十大步骤 Identify assessment topics确定评估项目 Specify assessment problem确定所要评估的问题 Determine locus or responsibility of assessment 决定评估的焦点和任务 Retrieve (gather) evidence收集数据 Collect new primary data (as appropriate)收集新的原始数据 (只要合适) Interpret evidence解释数据 Synthesize (combine or consolidate) evidence综合分析数据(联合或者加强) Formulate findings and recommendations形成结果和建议 Disseminate (Communicate) findings and recommendations传播（交流）结果和建议 Monitor impact监督评估的影响

Two Main Groups of Studies (Methods)两种主要研究方法 • Primary data collection原始资料收集 • Collect original new data, for example, using experiments收集原始的新数据，例如，利用试验数据 • Secondary data analyses第二手资料分析 • Combine (Synthesize or integrate) data from existing sources采用已有资料 (综合或者整合) 国际华夏医药学会－陈英耀发言

原始资料研究方法的证据强弱 • 前瞻性研究比回顾性研究更有说服力 • 有对照研究比无对照研究更有说服力 • 随机研究比非随机研究更有说服力 • 大样本（有足够的病人供检测治疗的真实效果）研究比小样本研究更有说服力 • 当前对照组研究比历史对照组研究更有说服力 • 双盲法研究（指病人和医生不知道所采用的是何种技术）比非盲法研究更有说服力国际华夏医药学会－陈英耀发言

The relation between levels of evidence and grades of recommendation原始资料研究方法的证据强弱排序 (stronger) (说服力强) • Large randomized controlled trials with clear-cut results大规模随机对照试验 • Small randomized trials with uncertain results小规模随机对照试验 • Nonrandomized, contemporaneous controls 非随机,同期对照研究 (不同种类) • Nonrandomized, historical controls 非随机，历史对照 • No controls, case series only 连续多个案例研究 • No controls, single case study 单个案例研究 (weaker) (说服力弱) A B C 国际华夏医药学会－陈英耀发言

Secondary Data Analyses第二手资料分析 • Expert opinion专家意见 • Group judgment (Consensus development) 团体判断（得出一致意见） • Literature review文献评阅 • Meta-analysis文献综合分析 • Decision analysis, other modeling决策分析及其他模型国际华夏医药学会－陈英耀发言

Timing of Assessment评估的时间选择 • 对一项技术而言并不存在开展技术评估的单一的正确时间 • 在整个技术寿命过程中，执行技术评估的目的是为了满足不同的政策制订者的需要 • 在选择评估时间的时候，我们需要权衡下列因素： • 一方面，对一项技术评估得越早，我们就越有可能限制其使用（如果这项技术是有害的或者无效的话） • 另外一方面，早期技术评估的结果可能会引起误导，因为在技术应用的早期我们并没有足够的数据和信息来进行评估 • “目标变动”问题 • 教训：“人们总是觉得现在开展评估太早了，而当危害发生的时候却又已经太晚了。” (M.BUXTON 1987) 国际华夏医药学会－陈英耀发言

Types of Experts For HTA参与技术评估的专家 Clinicians (physicians, other)临床医生 Pharmacists药师 Laboratory and radiology technicians实验室和放射技术人员 Hospital managers医院管理者 Bio-medical engineers生物医学工程师 Patients and community representatives病人和社区代表 Economists经济学家 Epidemiologists流行病学家 Bio-statisticians 生物统计学家 Decision scientists 决策科学家 Lawyers律师 Ethicists 伦理学家 Librarians or information specialists图书和信息专业人员

四、各国卫生技术评估发展回顾 国际华夏医药学会－陈英耀发言

Development of HTA卫生技术评估的发展 • 1967 the term used in the US Congress • 1972 Office of Technology Assessment (OTA) was established • Technology assessment: A comprehensive form of policy research that examines the short- and long-term social consequences of the application or use of technology • CT scanner was OTA’s first target for HTA 国际华夏医药学会－陈英耀发言

卫生技术评估在各国的发展 几乎和美国同时，瑞典开始进行技术评估工作 1985年，国际卫生技术评估组织(ISTAHC)成立 1987年，瑞典建立了卫生部与HTA项目合作机制 90年代，几乎所有的欧盟成员国建立了国家和区域的HTA机构和项目 1993年，国际卫生技术评估机构网络(INAHTA)成立 1994年1月卫生部在上海医科大学成立了国内第一家卫生技术评估研究中心 2003年， ISTAHC更名为HTAi

Health technologies typically regulated for placement 加强规制的主要技术 • Surgery • Neurosurgery • Cardiac surgery (with cardiac catheterization, PTCA, etc.) • Intensive care – especially neonatal intensive care • Genetic screening and counseling • In vitro fertilization • Imaging devices • CT • MRI • PET • Radiation therapy • Transplants • Kidney (and renal dialysis) • Heart • Heart-lung • Liver • Pancreas • Bone marrow 国际华夏医药学会－陈英耀发言

Relations between payment and HTA 运用于支付或补偿 • Every system is characterized by incentives and disincentives 各种补偿机制优劣并存 • Rising expenditures related to incentives for technological intervention 技术造成费用上涨 • Rising expenditures addressed by prospective fixed budgets 前瞻性的固定预算来解决费用上涨问题 • Fixed budgets can control costs and technology， but not assure cost-effectiveness or efficiency 固定预算可以控制成本和技术，但不能保证效率国际华夏医药学会－陈英耀发言

Coverage and HTA 运用于保险报销范围 • Traditionally the “package” is defined by medical doctors 报销范围传统上由医生来确定 • New technology not admitted to package until shown to be effective (and possibly cost-effectiveness) 新技术必须证明是有效而且经济，才能纳入报销范围 • Old technology removed from the package when shown to be ineffective or very expensive for the benefit gained 若老技术显示无效或昂贵，它会被从报销范围中删除国际华夏医药学会－陈英耀发言

Quality assurance 运用于质量保证 • Wide range of activities, including • Routine statistics (for example, health outcome)常规统计 • Audits 评审 • Licensing 许可证 • Accreditation 认证 • Certification 证明 • Informational strategies 信息化策略 • Clinical practice guidelines 临床指南或诊疗常规国际华夏医药学会－陈英耀发言

Relationship of HTA, CC and EBM HTA to work mainly to influence health policy CC: Cochrane Collaboration EBM: Evidence-based Medicine CC to develop the evidence on which to base decision EBM to seek primarily to affect medical (healthcare) practice 国际华夏医药学会－陈英耀发言 Banta. The development of health technology assessment. 2003

HTA in the US • The US has not a national HTA framework • 1972-1997:OTA • 1978-1981: National Center for Health Care Technology • Public and private mix structure • Main goals: • cost containment • improving quality or innovation • micro management 国际华夏医药学会－陈英耀发言

HTA participants • Public • NIH:clinical trials • AHCPR (Agency for Health Care Policy and Research): clinical guideline • HCFA: insurance coverage • VA (Veterans Administration) • FDA • Private • AHA and AMA • Health insurance and managed care companies • Industrial companies • Hospitals 国际华夏医药学会－陈英耀发言

HTA in UK • UK, along with Sweden, has the best organized and best funded HTA activities in the world • HTA leadership: a national agency or council for HTA • embedded in the Department of Health’s Research and Development Program • a national HTA committee, a coordinating center and centers • To enhance the role of the consumer in decision-making 国际华夏医药学会－陈英耀发言

HTA in Sweden • Swedish Council for Technology Assessment in Health Care (SBU) • Extend HTA activities into mental health care and social services by the government • Swedish patients are increasingly aware of HTA 国际华夏医药学会－陈英耀发言

International Society of Technology Assessment in Health Care, ISTAHC国际卫生技术评估协会 1985年5月成立秘书处设在加拿大，会员来自45个国家宗旨是加强卫生技术安全性、有效性、经济性和社会影响的研究、教育、合作与信息交流，促进制定技术合理使用的相关政策出版《国际卫生技术评估杂志》及简报每年举办一次年会 http://www.istahc.org

卫生技术评估 Health Technology Assessment