Economics in Epilepsy Treatment Choices: Our Certain Fate?

Over the last 20 years, there is evidence to show that doctors increasingly accept the need to consider the financial implications of the treatments they prescribe. In the field of epilepsy treatment, economic evaluations have considered the financial impact of physicians' choices in newly diagnosed epilepsy, chronic active epilepsy, epilepsy surgery, and childhood epilepsy. A variety of economic methods have been applied including cost-effectiveness analysis, cost–utility analysis, and more recently, cost–benefit analysis. The real effect of this research on treatment decisions is yet to be established. We discuss the impact that economic data, in the form of cost-effectiveness and cost–benefit analyses, have on epilepsy-treatment decisions at the clinical and policy levels. The broader literature concerning physician attitudes and practices concerning cost-effectiveness is reviewed and, with examples from countries throughout the world, the extent to which health economic research has affected epilepsy-related treatment decisions is discussed. Epilepsy is one of the most common, serious, neurologic disorders worldwide (1). Although the cost of antiepileptic drug (AED) treatments is relatively modest compared with new treatments for other neurologic conditions, the high prevalence and chronicity of epilepsy mean that, in public health terms, the overall cost of its treatment is high. Furthermore, many of the recent advances for treating epilepsy, such as implantable vagal nerve stimulators and new techniques of epilepsy surgery, have high initial cost but may offer long-term savings if patients become seizure free (2,3). Economic factors are important at every level of epilepsy treatment, although the way in which costs affect medical decisions depends, to a large extent, on the country and health system that is considered. For example, in many developing countries, the choices that individual doctors make about the investigation and treatment of patients are governed primarily by a patient's ability to pay. In rural areas of a country such as India, patients with newly diagnosed epilepsy may be prescribed phenobarbitone, despite the poor long-term side-effect profile of this drug, as most patients find the cost of modern AEDs prohibitive (4). In countries with well-developed social health insurance systems, such as those that exist in Western Europe or Australia, treatment is often free, or heavily subsidized, to the patient at the point of delivery. In these countries, a doctor's ability to prescribe expensive AEDs may be limited to those included on approved formulary lists or government reimbursement schedules. Statutory protocols may restrict the situations in which a doctor may prescribe certain drugs. Drug prices are a crude means of comparing the economic impact of epilepsy treatments. Table 1 shows how they vary across three countries. Those who rely on drug prices as the sole means of assessing the economic impact of treatment fail to take into account the savings that might arise if a patient's condition improves: for example, in most cases, a seizure-free patient requires less medical follow-up and is more likely to be able to return to work (5). Treatments may also improve the quality of a patient's life, and this can also be considered in economic terms, using outcomes such as Quality Adjusted Life Years or contingent valuation measures such as Willingness to Pay (6). Cost-effectiveness studies have been proposed as a method by which decisions about finite resources may be reached and health care delivery optimized for the money spent (7). Economic evaluation involves the comparative assessment of all courses of action in terms of their clinical consequences and resource costs (6). By making all relevant costs and consequences explicit, cost-effectiveness analyses aim to provide clinical decision makers with information to choose rationally between treatment options. There are two distinct settings in which economic information may be considered: (a) at the clinical care context, where doctors make decisions in conjunction with their patients, as should be the case within a hospital or outpatient setting; and (b) in the “committee-room,” where policy decisions about resource allocation and rationing, which affect physicians' ability to treat patients, are made for heath plans and systems. In general, in deciding on appropriate epilepsy treatments, physicians must balance the risks and costs of a given treatment with its potential benefits. Potential treatment-related adverse events and the cost of medication must be weighed against the clinical, psychological, social, and economic impacts of inadequately treated seizures (in terms of frequency and/or severity). There is rarely an instance in which the risks and costs of not treating at all are thought to outweigh the risk and cost of treatment, although treatment during pregnancy may fall into this category. Rather, the treatment decision usually focuses on the comparative risk, benefits, and costs of alternative AEDs. In making these treatment decisions, economic information may be considered by doctors when investigating and treating their patients either at the bedside or in the outpatient clinic. There is evidence to suggest that the opinions of individual physicians and neurologists regarding the relevance of economic information in clinical practice have changed dramatically over recent years. Whether this change in opinion has translated into more cost-effective clinical practice is less clear (8). Before the 1980s, the effect of economic evaluations on health care decision-making was minimal (9). Little economic research had been performed and, in the case of epilepsy, only rudimentary studies of the cost of illness had been published (10). Since the 1990s, however, the number of economic evaluations of treatment has increased markedly. There is evidence to suggest that individual doctors in a variety of countries throughout the world, including Holland, Denmark, Germany Australia, Canada, Sweden, the United States, and the United Kingdom (11–15), have become aware of the need for cost-effectiveness analysis and economic evaluation in everyday practice. Although few surveys have been published, studies performed in Europe and the United States reveal that most physicians accept the need to consider economic factors when treating patients. In a study by Reichert et al. (16) of 134 U.S. physicians, 88% of surveyed physicians thought that medication costs were an important factor in making prescription decisions, and 71% were willing to sacrifice some degree of efficacy to make drugs more affordable for their patients. Almost all the physicians (94%) reported that cost was a more important factor when the patient was paying for his or her own medications. Safavi and Hayward (17) also surveyed U.S. physicians regarding factors influencing treatment decisions. Their results indicated that when deciding between medications with similar efficacies and side effects, cost, patient compliance, and patient preference were the most important factors. Hux et al. (18) also found that prescription decisions by Canadian physicians were strongly influenced by drug costs. In a survey of a disparate selection of physicians, hospital managers, and individuals in government agencies in nine Western European countries, generally positive attitudes toward economic-evaluation studies were demonstrated, although many misgivings toward the current state of the art were highlighted (19). Recently, United States neurologists were surveyed about restricting the prescription of five new costly AEDs as part of a wider study considering attitudes towards rationing (20). It was clear that most neurologists recognized the need to ration health care, and believed that cost-effectiveness research is one method to achieve efficient distribution of resources. Only 25% of respondents thought that there should be no restrictions placed on the prescription of any of the five newer AEDs described in the questionnaire. Half of the respondents (57%) indicated that only carbamazepine (CBZ) and phenytoin (PHT) should be available for the treatment of new-onset complex partial seizures unless there was a specific contraindication to such use. This study did not establish whether these attitudes reflect the influence of a number of recent economic studies in epilepsy or are a result of a more general acceptance of the need to consider cost issues in all treatment. Despite the apparent importance of economic information in treatment decisions, many physicians are unaware of medication costs. In the Reichert et al. (16) study, 80% of the participating physicians indicated that they often did not know the cost of medications. Other studies have also indicated little knowledge of medication costs by physicians (17,21–23). Once medication costs are known, these costs appear to be influential in making treatment decisions. Salman et al. (24) evaluated the impact of drug costs on treatment decisions for hypertension. Using hypothetical scenarios of patients with moderate hypertension, participating physicians were substantially more likely to choose a less expensive medication when the drug costs were explicitly presented. Similar effects of drug-cost knowledge were seen in a study of treatment choices for urinary tract infections (25). Based on these studies, it is likely that physicians treating individuals with epilepsy will consider cost an important factor. However, these physicians may have only limited knowledge regarding the relative costs of AEDs. Further, it is not clear if these attitudes translate into practice behaviour, as discussed later. Cost-effectiveness results are a step beyond information on drug cost alone, additionally requiring an understanding of how to interpret the results. Few studies have assessed physicians' attitudes towards cost-effectiveness studies. Ginsberg et al. (26) surveyed 1,000 randomly selected California physicians in the United States. Most physicians (88%) considered cost-effectiveness to be an important factor in making treatment decisions. However, implementation of cost-effectiveness in clinical practice may be problematic. Many of the surveyed physicians (72%) indicated that only the treating physician and patient should decide what treatments are cost worthy. Further, physicians were divided on whether they should offer treatments with little likelihood of success regardless of cost; 53% of those surveyed agreed with the statement “if a medical intervention has any chance of helping the patient, it is the physician's duty to offer it.” Given the lack of knowledge regarding drug costs and (once known) their role in influencing treatment decisions, it is likely that physicians are unaware of the cost-effectiveness of many AEDs. Physicians might use cost-effectiveness information in treatment decisions were it available to them. However, the thresholds at which physicians would consider an AED cost-effective are likely to vary substantially and be easier to define in abstract rather than for an individual patient. Especially for individuals with refractory epilepsy, physicians might be willing to recommend medications or other treatments despite minimal expected value for the intervention's cost. Spending on AEDs has been estimated to be increasing at a rate 10% per year in developed countries (27). This increase may provide indirect evidence that despite changing attitudes, cost considerations are low on the list of individual doctors' priorities in the clinical care context. Alternatively, the increase may reflect the baseline increase in costs for AEDs (which, as with most other medical goods and services, increase in price at rates greater than that of overall inflation) or may be due to physicians switching to newer and more expensive AEDs based on beliefs in their superior effectiveness and/or side-effect profiles. There does appear to be an “inherent contradiction” between research showing that physicians' believe that prescribing should be more cost-effective and data concerning their actual prescribing patterns (8). Recent qualitative and quantitative research on doctors' attitudes toward economic data may explain this apparent paradox. Physicians recognize the ethical dilemma of applying cost-effectiveness arguments to individual patients. Physicians increasingly accept their role as responsible stewards for society's finite resources, but this duty may conflict with their fiduciary duty to do everything possible for the patient facing them in a consulting room (28). For example, a doctor often has to choose between AEDs to treat a patient with recently diagnosed epilepsy. For partial seizures, PHT, CBZ, and lamotrigine (LTG) have been proven to be equally efficacious in terms of seizure control for newly diagnosed patients. It is argued that LTG may have benefits in terms of side-effect profile and tolerability, and thus improve quality of life, but it is many times the cost of PHT or CBZ. Other dilemmas may occur in some settings where there may be differential fee structures between consultants when patients are referred to specialists: the fee of the nationally prominent epilepsy specialist may be higher than that of a local specialist, although treatment by a knowledgeable epilepsy specialist may result in more appropriate treatments, improved patient outcomes, and decreased costs. Some doctors may feel less inclined to consider societal needs against those of an individual, particularly when faced with a patient who asks “but what if it were you, doctor?” and the ethical issues surrounding this “double agent” role have been well described (29). Doctors may have difficulty with the methods and validity of health economic methods. Medical training in most countries contains little emphasis on social and economic sciences, and many physicians may find the results of economic assessments difficult to understand or apply. Physicians may, therefore, rely on secondary or even tertiary sources of evidence about the cost-effectiveness of treatment. Economic evaluations are suspected of being biased, particularly if sponsored by the pharmaceutical industry (30). A survey of European health care workers revealed a view that economic studies rely on “too many assumptions,” and the significance of sensitivity analyses to test assumptions may be misunderstood. It could be argued that doctors may feel that the results of economic analyses are relevant only to large cohorts and do not apply to individual patients who by definition have unique circumstances. As discussed by Asch and Hershey (31), cost-effectiveness evaluates the relative costs and benefits of an intervention on a population basis. Conversely, physicians may be more concerned with the potential costs and benefits on an intervention on a given patient. The costs and benefits observed for a single individual might be quite different from those expected on average over a large population. Patients differ in terms of their medical needs and the priorities they place on certain outcomes. For example, an individual patient with epilepsy may place a high premium on avoiding adverse cosmetic side effects, whereas another might value seizure freedom most of all (32). When faced with this diversity of patients' needs, doctors may find it difficult to apply the findings of economic analyses to individual patients. Further, as the physician may be treating a given patient for multiple years, AEDs with the greatest perceived likelihood of resulting in satisfactory treatment and patient satisfaction (rather than those providing acceptable incremental benefits for specified incremental costs) may be selected. Cost-effectiveness generally evaluates costs (and benefits) for longer time periods. Cost-effectiveness analyses may assess the incremental costs and benefits of a new AED over the course of 1 or multiple years, including the potential for reduced hospitalization, surgery, and other expensive medical-resource use at some point in the future. In contrast, physicians are often interested in the optimal treatment for a patient now, one that will lead to satisfactory seizure control as quickly as possible. Institutional factors and the way in which fees and charges are paid may offer little incentive for doctors to consider cost-effectiveness issues when they are making clinical decisions. These factors may lead doctors to believe that “savings in economic studies are anticipated not real”(19). For example, money saved by prescribing a cheaper AED may not be reallocated to different aspects of epilepsy care, such as improved access to an epilepsy nurse specialist or patient information service, and may instead be subsumed into a more general hospital budget, or even the organization's profits. Further, savings in drug costs do not mean actual savings in net long-term medical care costs; use of a less expensive drug may result in increased costs due to increases in other types of medical care use. Finally, the quality of economic studies themselves may be poor. The methods by which economic outcomes are assessed are still being developed (33). There are many clinical situations, such as treatment of the elderly and women of childbearing age, which have not been investigated by any form of economic evaluation. Nevertheless, the number of economic evaluations continues to increase, and understanding of the research methods is likely to continue to increase. In contrast to the uncertain effect that economic information has in the clinical-care context, cost-effectiveness analyses are clearly having an impact through the committee room, where decisions about resource allocation and antiepileptic treatment reimbursement are often made. No study has comprehensively reviewed the role that economic data hve played in decisions about epilepsy resources and program funding. However, several studies have assessed the attitudes of policy decision makers regarding use of economic information. A study in the Netherlands (34) found that all participating health care decision makers had positive attitudes toward economic studies. However, health care regulators and hospital pharmacists had greater knowledge of economic evaluations and used them more often than did physicians. In a study of five U.S. hospital pharmaceutical and therapeutics (P&T) committees, Segal and Pathak (35) reported that impact on hospital costs was the second most important factor in selecting medications for hospital formularies (the first factor was the effects of the potential formulary addition on the quality of drug treatments available in the hospital). Similarly, U.S. managed-care pharmacy directors indicated that cost-effectiveness analyses were the second most important factor in decision making, behind assessment of clinical effectiveness (36). Steiner et al. (37) also reported that economic information, particularly cost-effectiveness analyses, was the main factor in coverage decisions for new medication technologies. In the U.K., health care decision makers were willing to consider higher cost drugs for which better outcomes could be demonstrated (38). Throughout the world, the process by which new drugs are approved and licensed differs, and the role of cost-effectiveness data in this process varies markedly. A number of countries, including Australia, Canada, and the United Kingdom, have instituted statutory bodies that consider the cost-effectiveness of treatments alongside any clinical benefits that are offered by a drug. In other countries, similar processes of drug evaluation exist, although these are limited to less formal authorities. The role of economic considerations in this process can be considered by reference to oxcarbazepine (OCBZ), which has been licensed in a number of countries for a range of clinical indications similar to those for CBZ, a cheaper drug that has been used in treatment of epilepsy for 30 years. There is evidence to suggest that the OCBZ has similar efficacy to CBZ in terms of seizure control, but that it has a lower incidence of skin rash and fewer cognitive side effects (39). In Australia, OCBZ failed to be approved by the Pharmaceutical Benefits Scheme (PBS), which is a statutory government body that considers all new pharmaceuticals before they are included on a nationally approved formulary. The process of approval demands that the pharmaceutical companies provide evidence that new drugs are cost-effective and clinically efficacious. The PBS determined that OCBZ be compared with CBZ in a cost-minimization analysis, and found that the extra cost of OCBZ precluded approval. Drugs that are not approved for the PBS are not reimbursed by the Australian Health Service, and although this does not preclude their being prescribed by the treating physician, they do not attract government subsidy and hence may often prove prohibitively expensive for the patient. A similar system of drug approval operates in Canada. Drugs are licensed for use on clinical grounds, but through a separate process, they also are evaluated by each province in terms of their cost-effectiveness. Thus, in Canada, OCBZ was licensed for use but failed to be approved for government reimbursement. As with Australia, without government reimbursement, many patients are unlikely to be prescribed this drug. In the U.K., the government has recently created a special health authority, the National Institute for Clinical Excellence (NICE), whose stated aims include the objective of “promoting clinical and cost effectiveness through guidance and audit.” Since its inception, NICE has approved the use of a number of neurologic treatments including β-interferon for multiple sclerosis, riluzole for motor neuron disease, and donepexil (Aricept) for Alzheimer disease. The Authority does not consider all treatments. New technologies are evaluated only if they are thought to result in a significant (a) health across the if given to all relevant (b) impact on other government and impact on National Health resources if given to all relevant patients. OCBZ was licensed for use in the without reference to and neurologists and general are free to prescribe it to patients for it is clinically Many of the to the use of economic information in decision making in the committee room are similar to those by doctors in the clinical care context. 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