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The prevention and detection of corruption in pharmaceutical companies 预防及侦测制药公司的贪污
Pub Date : 1900-01-01 DOI: 10.3233/PPL-170451
Dominic Peltier-Rivest
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引用次数: 8
HTAs and access to rare diseases therapies: How can clinicians assist in the healthcare assessment of treatments for patients with primary immune deficiencies? hta与罕见病治疗的可及性:临床医生如何协助对原发性免疫缺陷患者的治疗进行保健评估?
Pub Date : 1900-01-01 DOI: 10.3233/PPL-2011-0325
H. Chapel
1. Background Health Technology Assessments (HTA) in terms of therapies depend on established treatments for defined conditionswith provenoutcomes; the various treatment regimes can be compared with each other or with outcomes in the absence of treatment, in various healthcare settings. For example in the UK, the National Institute for Health Research – NIHR states that “The HTA programmeproducesindependent research about the effectiveness of different healthcare treatments ...for those who use, manage and provide care in the NHS”. Given limited financial resources, it is appropriate that HTA agencies are increasingly involved in assessment of the cost effectiveness of well -established therapies, includingthose for rare diseases. Ultimately HTAs are mechanismsthat help funders and governments to control healthcare budget expenditure. An excellent example is therecentdecisionoftheIrishAuthority’sHealthTechnologyAssessmentDirectorate “not to introduce a new technology to screen donated blood for Creutzfeldt-Jakob disease because it would not be cost effective to do so”. Such tools are widely used, though for rare diseases the background is often poorly understood. However their popularity is exemplified in the Journal Citation Reports for 2009, which shows that "Health Technology Assessment", the journal of the HTA programme, has seen a significant rise in its impact factor rising from 5.01 last year, to 6.91. In the current world-wide economic climate, clinicians, patients, healthcare providers and those with the budgets need to understand the economic strategy of HTAs. This is particularly true for those involved with rare diseases, such as primary immune deficiencies (PIDs), and applies in countries with good access to PID centres of excellence that may be at risk, as well as in those in which therapies are restricted for financial reasons. The role of HTAs in assessing the efficacy and cost effectiveness treatments for patientswithPIDsisdifficultforavarietyofreasons: difficultiesinsettingupclinical
1. 背景:就治疗而言,卫生技术评估(HTA)依赖于针对已确定疾病的既定治疗方法和已证实的结果;不同的治疗方案可以相互比较,也可以在不同的卫生保健环境中与没有治疗的结果进行比较。例如,在英国,国家健康研究所(NIHR)表示,“HTA项目对不同医疗保健治疗的有效性进行了独立研究……为那些在NHS中使用、管理和提供护理的人”。鉴于财政资源有限,HTA机构越来越多地参与评估已确立的治疗方法(包括罕见病治疗方法)的成本效益是适当的。最终,hta是帮助资助者和政府控制医疗预算支出的机制。一个很好的例子是卫生部卫生技术评估局最近决定“不引入一种新技术来筛查捐献的血液是否患有克雅氏病,因为这样做的成本效益不高”。这些工具被广泛使用,尽管对于罕见疾病的背景往往知之甚少。然而,它们的受欢迎程度在2009年的期刊引用报告中得到了体现,该报告显示,HTA项目的期刊“卫生技术评估”的影响因子显著上升,从去年的5.01上升到6.91。在当前世界范围的经济气候下,临床医生、患者、医疗保健提供者和那些有预算的人需要了解卫生保健协会的经济战略。对于那些患有原发性免疫缺陷等罕见疾病的人来说尤其如此,这适用于那些能够很好地利用可能存在风险的原发性免疫缺陷卓越中心的国家,以及那些因经济原因而限制治疗的国家。由于各种原因,hta在评估pidas患者治疗的疗效和成本效益方面的作用很困难:难以建立临床
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引用次数: 0
Understanding the pharmaceutical value chain 了解制药价值链
Pub Date : 1900-01-01 DOI: 10.3233/PPL-160432
Murray L. Aitken
Understanding the pharmaceutical value chain requires the identification of each component from manufacturer to end consumer of medicines – and to understand their interaction. In most cases, the manufacturer’s selling price represents only a fraction of the retail price of a drug. More than half of the end user price results from insurance, freight charges (CIF), import tariffs and charges, importer margin, distributor margin, retailer margin and taxes. The article describes the elements of the medicine value chain, outlines factors and costs that contribute to the difference between the net price a pharmaceutical manufacturer receives for a drug and the final amount paid for the drug by the end user. It quantifies the price build-up for specific therapy areas and countries and illustrates the diversity of approaches and costs associated with the value chain through case studies.
了解药品价值链需要识别从药品制造商到最终消费者的每个组成部分,并了解它们之间的相互作用。在大多数情况下,制造商的销售价格仅代表药品零售价格的一小部分。最终用户价格的一半以上来自保险、运费(CIF)、进口关税和费用、进口商利润、分销商利润、零售商利润和税收。本文描述了药品价值链的要素,概述了导致药品制造商获得的药品净价格与最终用户支付的药品最终金额之间差异的因素和成本。它量化了特定治疗领域和国家的价格积累,并通过案例研究说明了与价值链相关的方法和成本的多样性。
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引用次数: 26
Pharmacologic-technical progress and the economics of growth 药理学——技术进步与经济增长
Pub Date : 1900-01-01 DOI: 10.3233/PPL-130365
Christoph Heible
Over the past 200 years, the real GDP in western industrialized countries rose continuously. The decisive factor of long-term economic growth is technical progress. Beside its macroeconomic importance it is the key vehicle of competition between research-based pharmaceutical companies. Further innovative drugs are an integral part of national health care systems in most industrialized countries. Innovative medicines contribute to the increase in life expectancy and an improved quality of life. To evaluate the share of pharmacologic-technical progress in the increase in life expectancy at the microand macroeconomic level it is initially helpful to clarify the economic relevance of technical progress in general. This will be the purpose of chapter two. Furthermore, the discussion encompasses the different approaches to define pharmacological innovations and the associated difficulties to discriminate between the different types of innovation. The focus of chapter three is the description of incentive mechanisms, which are fundamental to the Research and Development (R&D) activities leading to innovative pharmaceuticals. Beyond that, the microand macroeconomic positive effects of pharmaceutical innovations will be represented and interpreted with the help of existing results stemming from selected empirical studies. The last chapter gives a detailed overview on the current empirical results, which illustrate the versatile interdependencies on macroeconomic level between health and growth. It demonstrates the channels through which better health can have growth-enhancing effects.
在过去的200年里,西方工业化国家的实际GDP持续上升。长期经济增长的决定性因素是技术进步。除了具有宏观经济重要性外,它还是研究型制药公司之间竞争的关键工具。在大多数工业化国家,进一步创新药物是国家卫生保健系统的一个组成部分。创新药物有助于延长预期寿命和提高生活质量。为了在微观和宏观经济层面上评估药理学技术进步在预期寿命增加中所占的份额,首先要澄清一般技术进步的经济相关性。这将是第二章的目的。此外,讨论还包括定义药理学创新的不同方法以及区分不同类型创新的相关困难。第三章的重点是激励机制的描述,激励机制是导致创新药物的研究与开发(R&D)活动的基础。除此之外,药物创新的微观和宏观经济的积极影响将表示和解释的帮助下,从选定的实证研究产生的现有结果。最后一章详细概述了当前的实证结果,这些结果说明了健康与增长之间在宏观经济层面上的多种相互依赖关系。它展示了增进健康可以促进生长的途径。
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引用次数: 2
The European Innovative Medicines Initiative 欧洲创新药物倡议
Pub Date : 1900-01-01 DOI: 10.3233/PPL-2010-0259
G. Ruberti
The Innovative Medicines Initiative is a unique and novel long term european public-private partnership designed by the European Commission and the European Federation of Pharmaceutical Industries and Associations (EFPIA). The initiative was promoted to accelerate the discovery and development of better medicines by removing bottlenecks in the drug development process. It focuses on creating better methods and tools that improve and enhance the drug development process, rather than on developing specific, new medicines.
创新药物倡议是由欧洲委员会和欧洲制药工业和协会联合会(EFPIA)设计的一项独特而新颖的长期欧洲公私伙伴关系。推动该倡议的目的是通过消除药物开发过程中的瓶颈,加速发现和开发更好的药物。它的重点是创造更好的方法和工具,以改进和加强药物开发过程,而不是开发特定的新药。
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引用次数: 0
The new Spanish regulation of the medicinal industry 西班牙对医药行业的新规定
Pub Date : 1900-01-01 DOI: 10.3233/PPL-2010-0302
J. Gillard, C. Escudero
The Spanish legislator has finally decided to approve Royal Decree 824/2010 concerning pharmaceutical laboratories, manufacturers of pharmaceutical active ingredients and foreign trade in medicines. We say ‘finally’ because this is an area of regulation that is obviously primordial in the pharmaceutical sector and which, nevertheless, has been waiting for regulatory development to give operators minimal security and to clarify numerous underlying conflicts. Therefore, since the approval of Act 29/2006 of July 26th on Guarantees and the Rational Use of Medicine and Health Products – LGURM, in Spanish –, the current framework law in Spain, four long years have passed in which many of the mandates set out in article 63 and following were in need of greater specification. Some questions were even in direct conflict with Royal Decree 1564/1992 of December 18th, regulating the authorisation system of Pharmaceutical Laboratories and Medicine Importers and the guarantee of quality in their industrial manufacture, which was developed by the previous Law of Medicines of 1990, and which remained in force in the absence of legal replacement. To give but one example, in this time there have been numerous headers of the former authorisations such as “Commercialising laboratory”, whose administrative recognition was revoked without their being legalised with any alternative formula, leaving them in an unacceptable legal limbo. With such lacunae, it was difficult to encourage domestic, and above all foreign, operators to invest in Spain without a sufficiently predictable entrepreneurial ecosystem.
西班牙立法者最终决定批准关于制药实验室、药物活性成分制造商和药品对外贸易的第824/2010号皇家法令。我们之所以说“最终”,是因为这是一个监管领域,显然是制药行业的原始领域,尽管如此,它一直在等待监管发展,以给予运营商最低限度的安全性,并澄清许多潜在的冲突。因此,自从7月26日关于保证和合理使用药品和保健产品的第29/2006号法令(西班牙语:LGURM)获得批准,即西班牙现行的框架法律以来,已经过去了四年,其中第63条及以下规定的许多任务需要更详细的说明。有些问题甚至与12月18日第1564/1992号皇家法令直接冲突,该法令规定了制药实验室和药品进口商的授权制度以及工业生产中的质量保证,这是由1990年以前的《药品法》制定的,在没有法律替代的情况下仍然有效。仅举一例,在此期间,有许多前授权的标题,如“商业化实验室”,其行政认可被撤销,而没有任何替代方案使其合法化,使他们处于不可接受的法律边缘。由于存在这样的空白,如果没有一个充分可预测的创业生态系统,就很难鼓励国内,尤其是外国的运营商在西班牙投资。
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引用次数: 0
Specific requirements for somatic cell therapy medicinal products and tissue engineered products 体细胞治疗药品和组织工程产品的特殊要求
Pub Date : 1900-01-01 DOI: 10.3233/PPL-140413
R. H. Martín, Eduardo L. Mariño Hernández, J. L. Pedraz Muñoz, Antonio M. Rabasco Álvarez
Advanced-therapymedicinal products (ATMPs) are medicines for human use that are based on genes (gene therapy), cells (somatic-cell therapy) or tissues (tissue engineering) and include autologous, allogeneic, or xenogenic origin products. They are new therapeutic strategies and their development will contribute to providing opportunities for some diseases that so far lack effective treatments. The main objective of the pharmaceutical industry is to introduce on the market effective medications that increase hope and quality of life of the population, not to mention the safety criteria. To do this, it is important to know well the existing advanced therapies regulation allowing you to more easily perform the translation of basic research carried out in research laboratories and that decision making is quality. Definitions from a point of view regulatory of these medications are in the Regulation (EC) N◦ 1394/2007 of the European Parliament and of the Council of 13 November 2007 on advanced therapy (Chapter 1, article 2) drugs and the Directive 2001/83/CE of the European Parliament and of the Council of 6 November 2001 by establishing a community relating to medicinal products for human use code (annex I part IV, according to its last modification).
先进治疗药物(atmp)是基于基因(基因治疗)、细胞(体细胞治疗)或组织(组织工程)的人用药物,包括自体、异体或异种来源产品。它们是新的治疗策略,它们的发展将有助于为迄今为止缺乏有效治疗的一些疾病提供机会。制药业的主要目标是向市场推出有效的药物,增加人们的希望和生活质量,更不用说安全标准了。要做到这一点,重要的是要了解现有的先进疗法法规,使您能够更容易地在研究实验室中进行基础研究的翻译,并且决策是质量。从这些药物监管的角度来看,这些药物的定义见欧洲议会和理事会2007年11月13日关于高级治疗药物的第(EC) N◦1394/2007号条例(第1章,第2条)和欧洲议会和理事会2001年11月6日关于建立一个与人用药品代码有关的共同体的指令2001/83/CE(根据其最后一次修改,附件I第四部分)。
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引用次数: 0
What is the evidence produced so far by the application of HTA to haemophilia care 到目前为止,HTA在血友病治疗中的应用有哪些证据
Pub Date : 1900-01-01 DOI: 10.3233/PPL-2011-0326
K. Berger, D. Schopohl, W. Schramm
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引用次数: 1
Latin American pharmaceutical overview 拉丁美洲制药概述
Pub Date : 1900-01-01 DOI: 10.3233/PPL-140384
J. Valverde
Latin America is a diverse, multi-coloured and dynamic region; boast the highest life expectancy among developing regions. In Latin America persistent social exclusion and inequities in wealth distribution and in access and use of services are reflected in health outcomes. With its population reaching 600 million people in 2011, Latin American pharmaceutical sales were at $62.9 billion. LA’s pharmaceutical market represents approximately 25% of global pharmaceutical sales. As a developing market, Latin America is quite complicated and diverse in terms of regulatory, reimbursement, market, demographic, and political characteristics. The harmonization of pharmaceutical regulation has been initiated in conjunction with the creation of regional free trade zones. Five main free trade zones are developing: North American Free Trade Agreement (NAFTA); MERCOSUR; Central America; Andean Area; and The Caribbean Community. Regulatory frameworks overall have improved as a result of free trade and intellectual property agreements. Regulatory enforcement bodies, quality investigators, proactive Good Clinical Practice (GCP) and policy development, have been cited as contributing to Latin America’s explosive growth. Latin American countries are currently moving policies to support productive development and innovation. The local biotechnology industry is developing rapidly.
拉丁美洲是一个多样化、多肤色和充满活力的地区;在发展中地区拥有最高的预期寿命。在拉丁美洲,持续存在的社会排斥和财富分配以及获得和使用服务方面的不公平现象反映在健康结果中。2011年拉美人口达到6亿,医药销售额为629亿美元。洛杉矶的药品市场约占全球药品销售额的25%。拉丁美洲作为一个发展中市场,在监管、报销、市场、人口和政治等方面都相当复杂和多样化。在建立区域自由贸易区的同时,已经开始协调药品管制。正在发展的五个主要自由贸易区是:北美自由贸易协定;南方共同市场;中美洲;安第斯地区;和加勒比共同体。由于自由贸易和知识产权协议,监管框架总体上得到了改善。监管执法机构、质量调查人员、积极主动的良好临床实践(GCP)和政策制定被认为是促进拉丁美洲爆炸性增长的因素。拉丁美洲国家目前正在制定支持生产性发展和创新的政策。当地生物技术产业发展迅速。
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引用次数: 5
Health Technology Assessment and patients' rights: Assess versus access? 卫生技术评估与患者权利:评估与获取?
Pub Date : 1900-01-01 DOI: 10.3233/PPL-2011-0335
F. Houÿez, Y. Cam, F. Bignami, F. Macchia, F. Faurisson
Patients’ organisations call for an assessment of the clinical added value of orphan drugs at the time of the marketing authorisation evaluation. The European Commission has commissioned Ernst & Young to explore the feasibility of a new Committee at the European Medicines Agency1. A new drug candidate faces a number of hurdles on its journey to reach the patient. Regulators are the first, and right after are the Health Technology Agencies and payers. Things can turn sour, from a political point of view, when that second scenario happens, when the regulators say “yes this drug is safe and effective” and the payers say “Oh well but we won’t reimburse it”. Regulators and health technology assessment experts have recently started to harmonise their respective methodologies to avoid some degree of redundancy and contradictions in their respective opinions. Patients are participating in the decision making at the European Medicines Agency, and work closely with some national regulatory agencies. More systematic involvement with health technology assessment agencies is only beginning. This paper illustrates some of the issues patients are discussing with health technology assessment bodies.
患者组织要求在上市许可评估时对孤儿药的临床附加价值进行评估。欧盟委员会已委托安永会计师事务所(Ernst & Young)研究在欧洲药品管理局设立一个新委员会的可行性。一种新的候选药物在到达患者手中的过程中面临着许多障碍。首先是监管机构,其次是卫生技术机构和支付方。从政治角度来看,当第二种情况发生时,事情可能会变得糟糕,当监管机构说“是的,这种药是安全有效的”而付款人说“哦,好吧,但我们不会报销”。监管机构和卫生技术评估专家最近开始协调各自的方法,以避免各自意见中出现某种程度的冗余和矛盾。患者正在参与欧洲药品管理局的决策,并与一些国家监管机构密切合作。更系统地参与卫生技术评估机构才刚刚开始。本文阐述了患者与卫生技术评估机构讨论的一些问题。
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引用次数: 0
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Pharmaceuticals, policy and law
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