The November 1995 revised Australian guidelines for the economic evaluation of pharmaceuticals

In November 1995, the revised Australian Guidelines for the Economic Evaluation of Pharmaceuticals ('the Guidelines') were published. The new document is to be seen as a measured bureaucratic response to the perceived shortcomings of the August 1992 document. The new document sets substantially more demanding and more rigorous evidentiary standards in the reporting of randomised clinical trials and in the justification of the selected evaluation methodology. It also introduces the requirement for a trial- or efficacy-based preliminary economic evaluation, and it recognises the need, under certain circumstances, to model economic evaluations. Although this document has an immediate appeal to those coming to pharmacoeconomic evaluations from a clinical perspective, the approach taken is unlikely to appeal either to economists (the Guidelines continue to discourage cost-benefit analysis) or to health system evaluators working in a competitive delivery environment (such as the US). The Guidelines, in a US environment, would be seen as not only unreasonable in their evidentiary demands and in the task imposed on evaluators, but limited in their failure to take an explicit modeling or system approach to therapy intervention evaluations.     

Recommended structure for reporting economic evaluation on pharmaceuticals in Belgium

Pharmacoeconomic evaluations become more important for the reimbursement of pharmaceuticals, and will be obligatory for new pharmaceutical drugs for which an added therapeutic value is claimed and a price premium is proposed by the manufacturer. Therefore, it is important to guide purchasers and providers of pharmaceutical care in their efforts related to the evaluation process. Standard Report Format can support the quality, transparency and exhaustiveness of the data submitted. A multidisciplinary task force developed a Standard Report Format for pharmacoeconomic evaluations in Belgium.     

Canadian guidelines for economic evaluation of pharmaceuticals. Canadian Collaborative Workshop for Pharmacoeconomics

In 1994, Canada became the second country to release national guidelines for the economic evaluation of pharmaceuticals. The guidelines were developed over a period of 18 months through an elaborate process of broad consultation with a wide variety of relevant stakeholders. The intent of the guidelines is to provide guidance to doers and users of studies, by laying out the general 'state of the art' regarding methods, and by providing specific methodological advice on many matters. The aim is to improve the scientific quality and integrity of studies, and to enhance consistency and comparability across studies. This article presents the Canadian guidelines, both in summary and in detail. Because the techniques of economic evaluation are widely applicable beyond pharmaceuticals, the guidelines will be of interest to researchers and decision makers in all fields of healthcare. Because the methods are not country specific, the guidelines will be of interest to those in other countries as well as in Canada.     

蛋白降解靶向嵌合体药物的非临床评价策略分析

蛋白降解靶向嵌合体(PROTAC)将靶向蛋白募集到E3泛素连接酶进行泛素化标记,然后通过泛素-蛋白酶体途径将其降解,从而将过表达和突变的致病蛋白清除。本文基于已有的文献报道,总结PROTAC药物研发进展,针对其存在的相对分子质量较大、生物利用度低、稳定性和血管穿透能力差等问题提出应对策略,并从药理药效、代谢和安全性评价等方面对该类药物的非临床评价研究需要考虑的问题进行分析,为制定PROTAC药物非临床评价研究方案提供参考。     

The Danish approach to standards for economic evaluation methodologies

Standards for economic evaluation have now been proposed in several countries. The background for this article is a report commissioned in 1994 by the Danish health authorities (the National Board of Health and the Danish Ministry of Health) on the state of the art of economic evaluation: are the methods ready to be used for systematic decision-making in the Danish healthcare sector? The themes of this article are the attitude towards economic evaluation of medical technologies and healthcare services in Denmark, the areas for application and the desirability and feasibility of a standard set of methods for conducting economic evaluations. In addition, the current state of methodologies in economic evaluation is briefly outlined. On the background of a roundtable discussion of interested parties, the Danish health authorities have decided to introduce economic evaluation of new pharmaceutical products, on a voluntary basis for 2 to 3 years, when application has been made for public reimbursement.     

Influenza. Cost of illness and considerations in the economic evaluation of new and emerging therapies

Influenza infection has been a burden to humans for thousands of years. Despite the fact that epidemics could be predicted with regularity, the lack of available prevention or treatment measures left humankind vulnerable to the harmful effects of this ubiquitous virus. While the pandemics of 1918 and 1957 are recent examples of the devastation that influenza may inflict, even in a typical year influenza infection and related complications cause significant morbidity and mortality. The development of an influenza vaccine during the 1940s marked a major turning point in the management of this disease. Vaccination of the elderly and other high risk patients has been shown to reduce morbidity and mortality and to be a worthwhile investment from an economic perspective. Despite these benefits, vaccine use in this group remains suboptimal. The role of annual vaccination for individuals at lower risk for influenza-related complications remains controversial. While prevention by vaccination is relatively straightforward, the treatment of symptomatic influenza-like illness with medication is more complicated. Differentiating symptoms caused by the influenza viruses from those caused by other common viruses is difficult. Currently available tests to document influenza as the cause of illness are either too expensive, too inaccurate or too time consuming to impact treatment. Symptom-based diagnosis remains the most commonly used strategy in clinical practice. The approval of the neuraminidase inhibitors (NIs)--zanamivir and oseltamivir--remind healthcare providers of the difficulties in diagnosing and treating influenza. NIs have been shown to reduce the duration of symptoms of individuals infected with influenza when prescribed within the first 2 days of symptoms. Whether these innovative agents are cost effective, however, requires a more detailed understanding of the benefits that these agents may offer above and beyond existing therapies. In this review, we examine the burden of influenza infection, diagnostic challenges and the clinical and economic impact of available interventions. Clinical controversies and potential areas for further investigation are also explored.     

青光眼卫生经济学评价模型综述研究

本文通过对国内外研究文献中青光眼卫生经济学评价模型的综述,归纳了现有模型的基本架构和逻辑,主要包括针对短期性评价的决策树模型和针对长期性评价的马尔科夫模型,以期为中国青光眼经济学评价的模型设计和研究开展提供参考。     

蛋白降解靶向嵌合体药物(PROTAC)的非临床评价策略分析

蛋白降解靶向嵌合体(PROTAC)将靶向蛋白募集到E3泛素连接酶进行泛素化标记,然后通过泛素-蛋白酶体途径将其降解,从而将过表达和突变的致病蛋白清除。本文基于已有的文献报道,总结PROTAC药物研发进展,针对其存在的相对分子质量较大、生物利用度低、稳定性和血管穿透能力差等问题提出应对策略,并从药理药效、代谢和安全性评价等方面对该类药物的非临床评价研究需要考虑的问题进行分析,为制定PROTAC药物非临床评价研究方案提供参考。     

The changing environment for US pharmaceuticals

Health reform is currently the predominant health policy issue in the US. It carries profound implications for the pharmaceutical field, including the possibility of price controls that could stifle pharmaceutical research. While policy makers are contemplating alternative approaches to reform, the marketplace for pharmaceuticals has changed dramatically. For example, price increases have lessened, price discounting has increased, and new drugs are typically launched at prices lower than those of the leading product in the therapeutic class. These changes are driven in part by the growth of managed care. Further evidence of change in the industry is the number of job reductions announced and the decline in market valuation of pharmaceutical companies. Policy makers need to take the changed marketplace into consideration as they proceed with health reform, to avoid layering additional policy impediments on top of an increasingly harsh and unforgiving market. Such an approach could seriously compromise incentives for pharmaceutical research.     

新药非临床研究与评价中应关注的问题

近些年来,国家和业界对药物创新的整体认知水平不断提高,研发能力不断加强,新药非临床研究与评价中出现了一些新的值得关注的问题:首先在安全性研究与评价技术方面,中药、化药没有实质性差异,研究评价的基本思路和技术标准应统一,相应研究的技术指导原则应统一。其次,在非临床安全性研究的质量方面,一是支持药品注册的非临床安全性研究均应严格执行GLP,保障数据的真实性、完整性、可靠性以及可溯源性,提高药物研究质量和非临床安全性评价水平,二是应加强受试物分析,是非临床安全性研究与评价获取可靠结果的物质基础。此外,毒代与药代动力学研究、致癌性研究等也是值得关注的问题。     

Economic evaluation of pharmaceuticals: a European perspective

In recent years there has been a large increase in the number of economic evaluations of pharmaceuticals. Many of these studies have been commissioned by individual pharmaceutical companies, in support of new or existing products. In 2 countries, Australia and Canada (in the province of Ontario), draft guidelines issued by the government have outlined the requirements for economic evaluations to be submitted in support of requests for reimbursement (government subsidy) of particular products. One consequence of the guidelines is that they clarify what is required, and in specifying the procedure for submission of dossiers, identify a clear audience for the economic evaluation. In contrast, the situation in Europe is diverse. A wide range of healthcare systems exist, including national health services and more liberal systems, involving a wide range of insurers and providers. European countries also differ widely in their approach to the pricing and reimbursement of pharmaceuticals. Because of this diversity, the nature, conduct and impact of economic evaluation in Europe is not clear. It is therefore difficult for pharmaceutical companies to develop appropriate strategies for economic evaluation and for analysts to decide on appropriate study methodology. This article reviews the nature of any official guidance or requirements for economic evaluation, the potential for use of economic evaluation, the range of studies carried out and the identifiable impacts. There is currently no official guidance in any country, although some countries are considering issuing guidelines. In some countries there is official encouragement to pharmaceutical companies to undertake studies, and where economic data have been presented they have been considered by the relevant committees. The potential uses of economic evaluation vary widely from country to country. These can be classified in terms of a potential role in undertaking national price negotiations, deciding on reimbursement status or copayment level, deciding on inclusion in local formularies or in treatment guidelines, or in improving prescribing decisions. Approximately 80 economic evaluations of pharmaceutical products have been conducted to date in Europe, covering a wide range of clinical areas. There are relatively few examples of identifiable effects of such studies. In part this is because it is often difficult to assess the part played by various items of data. Nevertheless, the overriding conclusion is that economic evaluation of medicines is likely to be more relevant in Europe in the future. The problem for the pharmaceutical industry is in determining when and how.     

生物技术药物临床前安全性评价研究进展

生物技术药物是用现代生物技术生产的一类特定药物,主要种类包括基因重组的细胞因子或生长因子、单克隆抗体、疫苗、基因治疗制 反义核酸等。本文介绍了国际协调会议（LCH）有着生物技术药物临床前安全性评价指导原则一些要点,阐明了各类生物技术药物临床前安全性评价的具体要求并举例说明。     

The increasingly complex fourth hurdle for pharmaceuticals

There are three known criteria that underlie drug reimbursement decisions: therapeutic value, cost effectiveness and burden of disease. However, evidence from recent reimbursement decisions in several jurisdictions points to residual unexplained variables, one of which may be budget impact. An economic rationale for carrying out budget impact analyses is opportunity cost, measured by the economic benefits foregone by using resources in one way rather than another. Under certain assumptions, cost-effectiveness analysis accounts for opportunity cost while conveying to the decision maker the price of maximising health gains, subject to a budget constraint. However, the underlying assumptions are implausible, particularly in the context of pharmaceutical care. Although drugs that are cost effective may lead to unambiguous health gains among patient groups for whom the drugs are indicated, the opportunity costs could conceivably lead to a reduction in aggregate health gains, or failure to meet different kinds of equity considerations. The pertinent policy question is where to find the resources to fund new innovations, such as cost-effective pharmaceuticals, or drugs targeting severe diseases. It may be a matter of redeployment of resources across healthcare sectors, cancelling the funding of (older) pharmaceuticals that are less cost effective, or delisting drugs that are cost effective but target less burdensome conditions.