Pharmacoeconomics and formulary decision making

Pharmacoeconomic assessment of formulary actions has become increasingly common in local, national, and international formulary decision making. Tactics for managing medication use include formulary management and drug policies. Pharmacoeconomic data can provide support for these formulary decisions. For example, pharmacoeconomic data can support the inclusion or exclusion of a drug on or from the formulary and support practice guidelines that promote the most cost-effective or appropriate utilisation of pharmaceutical products. Various strategies can be used to incorporate pharmacoeconomics into formulary decision making. These include using published pharmacoeconomic studies and economic modelling techniques, and conducting local pharmacoeconomic research. Criteria for evaluating the pharmacoeconomic literature, suggestions for employing economic models, and suggested guidelines for conducting pharmacoeconomic projects are discussed. Although most formularies are viewed as cost-containment tools, formularies should not be a list of the 'cheapest' alternatives. Today's formulary should contain agents that optimise therapeutic outcomes while controlling cost. Pharmacoeconomic assessments of formulary decisions help to ensure that the agents promoted by our formularies yield the highest outcome per dollar spent. A discussion of the process for formulary action in a US hospital, the influence of pharmacoeconomics on US formularies, and strategies for incorporating pharmacoeconomics into formulary decision making are presented in this paper.     

Mycophenolate mofetil: a pharmacoeconomic review of its use in solid organ transplantation

Most pharmacoeconomic studies of mycophenolate mofetil have focused on its use as part of maintenance immunosuppression for renal transplantation, involving short-term (3 to 12 months) time frames. In general, mycophenolate mofetil reduced the treatment costs for rejection episodes and graft failure which offset its higher drug acquisition cost compared with azathioprine. Several cost analyses have been modelled on the large multicentre trials of adult renal transplant recipients. The use of mycophenolate mofetil was associated with either cost savings or no additional costs after 6 or 12 months in French, US and Canadian analyses of triple or quadruple immunosuppressant therapy. A further cost analysis utilising a registry database of renal transplant recipients in the US found mycophenolate mofetil to be cost saving compared with azathioprine after 6.4 years when evaluating costs due to graft loss only. Of the limited cost-effectiveness analyses with the drug, one US study modelled the 1- and 10-year cost effectiveness of mycophenolate mofetil and various other immunosuppressants used in combined regimens. Long-term use of mycophenolate mofetil was less cost effective than other regimens, but the use of long-term mycophenolate mofetil in high-risk patients was shown to be a relatively cost-effective strategy. In another US analysis comparing mycophenolate mofetil with azathioprine as part of quadruple therapy, mycophenolate mofetil was associated with slightly lower costs during the first year after renal transplantation as well as improved clinical outcomes. Conclusion: Pharmacoeconomic studies support the use of mycophenolate mofetil as part of immunosuppressant therapy in renal transplantation, at least in the short term. Although the cost effectiveness of mycophenolate mofetil in the long term is less clear, limited pharmacoeconomic data available appear promising. Among issues to be examined in future economic analyses in renal transplantation are the calcineurin-sparing potential of mycophenolate mofetil and the feasibility of using more efficient mycophenolate mofetil dosage regimens when using the drug on a long-term basis. Additional pharmacoeconomic analyses of mycophenolate mofetil are also needed in other types of solid organ transplantation.     

Evaluation of the pharmacoeconomic literature

Pharmacoeconomics identifies, measures, and compares the costs and consequences of pharmaceutical products and services. The cost effectiveness of a pharmacotherapy in a particular indication depends on the molecular configuration of the drug, its safety and efficacy, and local market factors such as the acquisition cost of the drug, the cost of a hospital bed, physician fees and nursing time. The types of study include cost minimisation, cost effectiveness, cost utility, cost benefit and cost of illness. Modelling studies are used to predict long term economic consequences of therapy. Retrospective studies apply local costs to clinical trials that may be international in scope. Full prospective pharmacoeconomic studies provide more complete information but are rare and expensive to perform. The principles of pharmacoeconomics are illustrated by 2 retrospective case studies from the literature. In the first study, ondansetron was compared with metoclopramide on the basis of efficacy and tolerability inferred from a previous well controlled clinical trial. A range of economic outputs provided answers from several perspectives. In the second study, a sound pharmacoeconomic analysis of corticosteroids in paediatric asthma used a comparator therapy that is now deemed clinically inappropriate. The case studies illustrate how conclusions from pharmacoeconomic studies must be interpreted with caution before they can be applied in a particular clinical setting.     

Pharmacoeconomics: disease-based management applications

Pharmacoeconomic information is rapidly becoming an accepted format for evaluating and comparing various treatment options. Such information may either supplement or replace standard methods for evaluating new drugs for possible inclusion on the formulary. It is important to recognize the pitfalls that may accompany different methods of collecting and evaluating pharmacoeconomic studies. Such information is important because drug use and outcomes in a real-world setting may differ substantially from those within the confines of a clinical trial setting.     

利用药物经济学评价指导药品定价的探讨

目的:探讨如何利用药物经济学评价指导药品定价,以提高药品定价的科学性和合理性。方法:首先指出药物经济学评价在药品定价中所能解决的问题即其应用必要性,然后从全社会的角度出发,对药品的成本和收益进行评估,以对药品的经济性进行评价。结果与结论:药物经济学评价能够用于确定新药的价格范围,指导药品定价,促进药品定价的合理性。     

Applied pharmacoeconomics. When can publication be legitimately withheld?

Pharmacoeconomic studies can help decision-makers choose the most efficient drug treatments in our internationally cost-constrained healthcare environment. However, perceptions of bias about the nature of many economic evaluations limit the usefulness of pharmacoeconomic data to decision-makers. In an effort to increase the credibility of pharmacoeconomic studies, several groups have developed methodological guidelines, and one has developed ethical guidelines for these evaluations. In this article, we evaluate issues related to the publication of the results of pharmacoeconomic studies. Pharmacoeconomics is a true science (and should be so treated), rather than a form of marketing. Pharmacoeconomic studies must undergo the same peer review process and be published in serious research journals, as are other types of scientific investigations. Investigators should attempt to publish the results of pharmacoeconomic studies, even (and, perhaps, especially) when the results are not favourable to the sponsor. However, there are acceptable reasons to withhold publication of 'negative' results. For example, when methodological problems plague a study, or when the study addresses an investigational drug not likely to be approved, then researchers are justified in giving up on publication, if they so choose. Similarly, feasibility studies to test methods of data collection or analyses conducted very early in the drug development process need not always be published. Nonetheless, access to all important investigations--regardless of whether the results are positive or negative--will become more important as healthcare becomes more evidence-based, as decisions have impact on large populations of people, and as those in charge of formularies actually begin to use cost-effectiveness analysis to help make choices among competing drugs.     

Institutional formularies: the relevance of pharmacoeconomic analysis to formulary decisions

Formularies, in one form or another, have been in existence for nearly 100 years. Beginning simply as a list of available agents, the formulary has evolved into a complex system which acts as a guide to prescribing practices. As the importance of the formulary has increased, so has the need for formulary managers to make an appropriate decision about each drug's formulary status. Several systematic approaches to drug evaluations have been developed to aid in the decision process. However, while some reviews of drug utilisation contain fairly rigorous analyses of their clinical efficacy, very few include an economic evaluation that goes beyond the cost of drug acquisition, preparation, distribution and administration. This is surprising, since formulary managers rank economic data second only to clinical data when making formulary decisions. In the past this apparent oversight has been due, in part, to the absence of a sophisticated model which can both approximate a drug's true economic impact and express cost and quality in similar terms. The explosion of new and very expensive biotechnology drugs into the market has the potential to improve patient care significantly. Such drugs also have the potential to increase institutional pharmacy budgets significantly; with some analysts predicting a spending of $US60 million yearly for these drugs by the year 2000, critical evaluation will be mandatory. Fortunately, advances in the relatively new science of pharmacoeconomics have made it possible to conduct appropriate estimates of the true economic impact of new drug therapies. Pharmacoeconomic studies can be very useful in evaluating drugs for formulary inclusion and in assessing the effects of formulary changes on institutional budgets. Cost-effectiveness and cost-benefit analyses, utilising decision analysis models and/or data gathered from clinical studies, are used most frequently. Relatively simple models can be used to evaluate drugs within the same class if sufficient published data on their clinical efficacy and safety are available. More complex analyses are necessary when comparing dissimilar agents or when comparing agents with non-drug therapy. Pharmacoeconomic studies have frequently been used to demonstrate that very substantial direct costs of drug therapy are often offset by equal or greater reductions in other institutional direct and indirect patient care costs. Pharmacoeconomic studies have also been used to calculate the relative cost-effectiveness of drug therapies for different disease states, although such evaluations are more useful to governmental and regulatory agencies than to individual institutions.(ABSTRACT TRUNCATED AT 400 WORDS)     

Methods for claims-based pharmacoeconomic studies in psychosis

Pharmacoeconomic studies using claims data are frequently employed to compare the healthcare costs associated with competing drugs. Different methodological approaches with varying limitations for evaluating claims data are reviewed within the context of psychosis. Intent-to-treat paradigms and mirror-image designs have limitations that can bias comparisons of health resource use and can be addressed through the use of drug treatment episodes. Health resource use is better measured in financial rather than volume amounts, to account for service intensity. However, financial measures reported in claims records need to be carefully chosen. Between-group comparisons are frequently affected by selection bias, and within-group comparisons are often limited by period bias. While selection bias can be addressed by controlling for patient factors such as disease severity, and period bias by controlling for trend, devising appropriate control measures from the limited information in claims data can be challenging. Healthcare data are often skewed, which affects statistical testing. While skewed data are commonly handled through log transformation, this method has serious limitations, potentially distorting pharmacoeconomic comparisons. Determining the most appropriate methods for claims-based data involves careful evaluation of the alternate approaches to best achieve the goals of a pharmacoeconomic investigation.     

Pharmacoeconomic burden of undertreating hypertension

Many studies have shown the importance of antihypertensive drug therapy as a factor in reducing the risk of cardiovascular morbidity and mortality, and in containing the cost of managing hypertension and its complications. Nevertheless, the evidence in clinical practice indicates about half of hypertensive patients do not receive pharmacological treatment and about half of treated patients do not achieve blood pressure level control. Undertreating hypertension is the leading cause of failure in drug therapy effectiveness and cost effectiveness. The pharmacoeconomic burden of undertreating hypertension can be defined as the clinical (number of cardiovascular events) and economic (costs of managing cardiovascular events) consequences that would have been avoided by adequate control of blood pressure levels. In the last few years, the increase in this burden and the restriction of budget constraints has raised the awareness of healthcare providers with regards to the need to achieve better performance and to improve disease management of hypertension. This review aims to present the current situation regarding the pharmacoeconomic burden of undertreating hypertension by identifying the key issues of this medical condition, defining and measuring the extent of undertreatment, defining and measuring costs associated with undertreatment, and discussing some fundamental aspects of disease management for hypertension. The pharmacoeconomic burden of undertreating hypertension appears to be an extremely important phenomenon for which there is currently only very limited adequate research. The present dearth of appropriate data can be largely attributed to the lack of epidemiological studies in clinical practice. Future studies are necessary for a more precise quantification of the therapeutic and economic impact of undertreating arterial hypertension in clinical practice (appropriateness studies) and for more precise selection of antihypertensive drugs on the basis of the different cost-effectiveness profiles detected in 'real world' settings (cost-effectiveness studies).     

The pharmacoeconomics of cognitive enhancers in moderate to severe Alzheimer's disease

Alzheimer's disease is associated with a substantial economic impact on patients, their caregivers and society. Due to the current rise in the aging population, the prevalence and impact of Alzheimer's disease are expected to increase greatly. The cost of caring for someone with Alzheimer's disease is magnified in the more severe stages of the disease. There are four cognitive enhancers commonly used for the treatment of Alzheimer's disease: three cholinesterase inhibitors (donepezil, rivastigmine and galantamine) and one NMDA receptor antagonist (memantine). Of these, donepezil and memantine have been approved in many countries as pharmacological treatments for moderate to severe Alzheimer's disease, while donepezil, rivastigmine and galantamine are approved treatments for mild to moderate Alzheimer's disease. While cost effectiveness has been well studied in mild to moderate Alzheimer's disease, the cost-benefit information for drug therapy in moderate to severe Alzheimer's disease is less clear. This article reviews the pharmacoeconomic data available on these four drugs, with a specific focus on moderate to severe Alzheimer's disease, including economic burden, cost drivers, clinical outcomes and pharmacoeconomic studies. A key driver of the cost of Alzheimer's disease is the severity of the disease, indicating that the ability to stabilize the disease state is a potential source of cost savings. Drug therapies that can limit increases in behavioural problems and cognitive and functional impairment, and postpone institutionalization without an increase in longevity may serve to reduce the economic burden on Alzheimer's disease patients. The data suggest that, while the available, approved agents offer only modest improvements in clinical outcomes, they could be cost-effective treatments for moderate to severe Alzheimer's disease when viewed from the societal perspective. For memantine and donepezil, data are available that suggest that the cost of these drugs is offset by the clinical and societal benefits provided by slowing the progression of Alzheimer's disease. While there are few head-to-head comparison trials, the similarity in costs of the treatments and efficacy against placebo suggest that cost effectiveness will not be substantially different among treatments. More studies that examine longitudinal resource utilization and its relationship to drug treatment in the moderate to severe stages are needed to clarify cost benefit in this population and possibly differentiate between individual medications.     

Zidovudine: a review of pharmacoeconomic and quality-of-life considerations for its use in patients with human immunodeficiency virus

In patients with human immunodeficiency virus (HIV) infections or the acquired immunodeficiency syndrome (AIDS), zidovudine is a first-line therapy that reduces morbidity and may reduce mortality. By delaying progression to AIDS, the drug reduces the duration and incidence of hospitalisations in a given time period, resulting in overall decreases in the cost of medical treatment per unit of survival time. In current therapeutic dosages zidovudine is generally well tolerated. Most pharmacoeconomic and quality-of-life studies of this agent were conducted using data relating to higher dosages and higher drug acquisition costs than those currently applicable, but nevertheless generally support the cost-effectiveness of zidovudine in patients with HIV disease. Studies examining the use of the drug in higher dosages demonstrate neither clear positive nor negative effects of the drug on quality of life. The cost effectiveness of the drug as prophylaxis against seroconversion after occupational exposure to HIV is dependent primarily on the establishment of clinical effectiveness in this condition. Further pharmacoeconomic studies should examine changes to dosage and cost factors, along with direct nonmedical treatment costs, indirect medical treatment costs and the effects of the drug on quality of life. An evaluation of existing studies suggests that if these factors were accounted for, zidovudine might be shown to be more clearly cost effective, and indeed its use in the treatment of patients with HIV disease might be found to result in cost savings.     

三种治疗甲真菌病药物的成本——效果分析

目的：探讨3种抗真菌药（伊曲康唑、特比奈芬、氟康唑）治疗甲真菌病的经济效果,找出既经济又有效的治疗方案。方法：根据文献选择3种不同的治疗方案（伊曲康唑组、特比奈芬组、氟康唑组）,应用药物经济学成本-效果分析法。结果：治疗指甲真菌病所需药品成本分别是666.68,634.42,459.96元;治疗趾甲真菌病所需的药品成本分别是1000.02,911.98,689.94元。远期治愈率指甲真菌病分别是94.4%,100.0%和77.8%;趾甲真菌病分别是86.4%,95.2%和68.2%。结论：根据药物经济学成本-效果分析,特比奈芬是治疗甲真菌病的最佳药物。     

3种十二指肠球部溃疡药物治疗方案近期疗效的成本-效果分析

目的 :探讨不同药物治疗同一疾病产生的经济效果。方法 :运用药物经济学的成本 -效果分析方法 ,对3种十二指肠球部溃疡药物治疗方案 (即A、B、C方案 )的近期疗效进行回顾性分析评价。结果 :A方案为最佳治疗方案。结论 :通过分析 ,说明药物经济学的成本 -效果分析方法在优化治疗方案 ,指导合理用药 ,提高经济效益方面具有重要作用。     

3种十二指肠球部溃疡药物治疗方案近期疗效的成本－效果分析

目的探讨不同药物治疗同一疾病产生的经济效果。方法运用药物经济学的成本－效果分析方法,对3种十二指肠球部溃疡药物治疗方案（即A、B、C方案）的近期疗效进行回顾性分析评价。结果A方案为最佳治疗方案。结论通过分析,说明药物经济学的成本－效果分析方法在优化治疗方案,指导合理用药,提高经济效益方面具有重要作用。     

Propofol. A pharmacoeconomic appraisal of its use in day case surgery

Propofol is an intravenous anaesthetic agent that has become widely used in day case surgery. It induces anaesthesia rapidly and 'smoothly', is associated with a quick recovery and has a lower incidence of postoperative nausea and vomiting (PONV) than other agents. In studies comparing propofol with other intravenous anaesthetics (most commonly thiopental sodium) in day case surgery, the use of propofol as induction and/or maintenance anaesthesia was associated with a shorter time to intermediate recovery (street fitness or time to discharge), although the mean time difference was generally less than 1 hour. However, when compared with volatile anaesthetics (particularly desflurane), the differences in time to discharge were smaller. Propofol is also associated with less PONV than barbiturates, volatile anaesthetics or barbiturate/volatile anaesthetic combinations in the immediate postoperative period. The faster recovery time and the decreased incidence of PONV have potential pharmacoeconomic implications. Delayed recovery can increase the use of hospital resources and decrease patient throughput and PONV can incur costs due to an increase in adjunctive medications usage (e.g. antiemetics), nursing time or unintended admissions. The pharmacoeconomic significance of these properties of propofol requires formal evaluation. Pharmacoeconomic investigations, such as cost-effectiveness, cost-benefit and cost-minimisation studies, which include clinical outcome parameters are difficult to conduct in anaesthesia since there are no objective measures of equipotency between anaesthetic agents and because there is no specific health outcome associated with the delivery of anaesthesia. At present, cost-utility studies are not possible because there are no validated instruments for measuring utility in the provision of anaesthesia. To date, pharmacoeconomic analyses of propofol (and other anaesthetic agents) in day case surgery have been restricted to partial cost analyses. Only 2 of these have included the cost of drug wastage, an important consideration since propofol contains no preservative. With 1 exception, these studies have only included drug acquisition costs and propofol was reported to be approximately 1- to 3-fold as costly as other intravenous/inhalational agents. However, these limited analyses have little applicability since they do not include all relevant costs. In addition to drug acquisition costs, pharmacoeconomic studies should also include other direct costs such as the cost of adjunctive medications (including treatment necessary for adverse events), equipment and staff time, indirect costs such as loss of productivity and/or wages and intangible costs such as patient satisfaction and quality of life. A few studies have attempted to quantify some of these factors. Based on differences in recovery time, 2 studies have estimated a decreased demand for nursing staff time associated with the use of propofol compared with thiopental sodium/isoflurane. In addition, informal patient satisfaction assessments show propofol to be equal to or better than other anaesthetic agents. With these broader considerations, it thus remains for future studies to quantify the intangible and indirect costs associated with propofol anaesthesia, to determine whether differences in recovery between propofol and other agents (especially the newer inhalational anaesthetics) are of economic importance, and to identify those instances where propofol use provides the greatest value for available funds.     

Emerging role of pharmacoeconomics in the research and development decision-making process

OBJECTIVES: This study examines the organisational structure of pharmacoeconomics departments in major pharmaceutical and biotechnology companies, the impediments to optimal use of pharmacoeconomic evaluations by companies and the integration of pharmacoeconomic analysis with research and development decision making. DATA AND METHODS: The heads of the pharmacoeconomics departments of 40 companies were surveyed on the structure of pharmacoeconomics departments in their companies, the roles that pharmacoeconomic analyses are playing in the new drug development decision-making process, and the initiation of pharmacoeconomic studies during the development process for a random sample of their companies' investigational new drugs. RESULTS: 45 department heads from 31 parent companies responded to the survey. The pharmacoeconomics function in pharmaceutical and biotechnology companies is relatively new and growing rapidly. Most pharmacoeconomics department heads preferred a different reporting structure than what they currently have and indicated that the strategic role that pharmacoeconomics can play is not well understood within the organisation. Pharmacoeconomic analyses have been increasingly initiated early in clinical development and have been a factor in clinical trial design and in key decisions made during the development process. CONCLUSIONS: Given the continued emphasis on containing healthcare costs worldwide, demand will increase for evidence that drugs provide good value for the money spent on them. Companies will likely respond not only with more economic evaluations for purchasers, but also with greater use of pharmacoeconomics early in the development process to aid in rationalising key research and development decisions, and in guiding final pricing decisions and reimbursement planning, thereby improving resource allocations.     

Simvastatin. A reappraisal of its cost effectiveness in dyslipidaemia and coronary heart disease

The Scandinavian Simvastatin Survival Study (4S) has confirmed the link between the cholesterol-reducing effects of simvastatin and improved survival in patients with hypercholesterolaemia and pre-existing coronary heart disease (CHD). Pharmacoeconomic analyses of the 4S trial, using prospectively collected data for cost-generating events, demonstrate that the cost per year of life saved for simvastatin in such patients falls within the range considered cost effective. Reductions in resource utilisation costs (numbers of hospitalisations and revascularisation procedures) largely offset the acquisition cost of long term simvastatin treatment in the US. Models of primary prevention incorporating epidemiological data to predict CHD events generally suffer from deficiencies in the methods and assumptions used, and no firm conclusions can be made at present regarding relative cost effectiveness of the drugs studied, including simvastatin. It is generally agreed that cost effectiveness will improve in patients with higher absolute risk of CHD. In summary, simvastatin has been shown in a major clinical trial and its companion economic analyses to reduce mortality and to be cost effective in patients with hypercholesterolaemia and existing CHD. As is the case for others of its class, its cost-effectiveness ratio in primary prevention remains to be ascertained. This issue aside, simvastatin is a rational choice of cholesterol-lowering agent in secondary prevention whose use can be justified on an economic basis.     

运用药物经济学手段有效遏制药品费用的不合理增长

目的 :寻求控制药品费用不合理增长的有效方法。方法 :分析和评价药物经济学研究在遏制药品费用不合理增长方面的作用。结果 :药物经济学研究通过对药物治疗的成本和药物治疗的结果两方面进行测量和比较 ,确定最佳的药物治疗方案 ;从5个方面控制药品费用的不合理增长。结论 :药物经济学研究在控制药品费用的不合理增长方面是切实可行的方法