Patient access to rheumatoid arthritis treatments
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- Jönsson, B. Eur J Health Econ (2008) 8: 35. doi:10.1007/s10198-007-0086-x
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This paper is an introduction to the study “The Burden of Rheumatoid Arthritis and Patient Access to Treatment”. The objective of the study is to compare patient access to new drugs in Europe, North America and a selection of other countries, and to analyse the determinants of differences between countries, as basis for a discussion on how patients’ access to new and effective treatments can be improved. There were few treatments available that could affect the progression of the disease prior to the introduction of the first TNF inhibitors in the late 1990s. Since the cost per patient treated with these biological drugs is high compared to previously available treatments, reimbursement through private and public insurance is an important determinant for access to treatment.
KeywordsRheumatoid arthritisTreatment access
The prevalence of rheumatoid arthritis (RA) is estimated to be 0.5–1.5% worldwide . The progressive nature of the disease and its onset in early or middle life mean that patients often live for 30 years or more with the disease. The social and economic impact of the disease is therefore considerable [2–10]. The cost to society associated with RA is large, as the disease leads rapidly to restricted joint mobility, chronic pain, fatigue and functional disability [11, 12], with about one-third to one-half of patients unable to work within 10 years of disease onset [13, 14]. As a consequence, productivity losses are a significant part of the economic burden of the disease [2, 9, 15, 16]. Direct health care costs are dominated by in-patient and community care, with drug costs representing a small (but increasing) fraction [17, 18]. There are at present several different medicinal treatment options available to patients with RA. These include analgesics such as non-steroidal anti-inflammatory drugs (NSAIDs), disease-modifying anti-rheumatic drugs (DMARDs) and biological agents, especially the tumor necrosis factor inhibitors (TNF inhibitors). The cost effectiveness of RA treatments, in particular the more recent TNF inhibitors, has been assessed in a number of studies [19, 16, 20–29].
Additional new therapies will be launched in the near future. Introduction and market access are a complex process, involving many steps and evaluations by various decision-makers, authorities, institutions, etc. These processes, as well as other mechanisms affecting patient access to new drugs, vary between countries. It is therefore interesting to assess and discuss the introduction and market access of RA treatments in different countries.
The objective of this study is to compare patient access to new drugs in Europe and North America and to analyze the determinants of differences between countries as a basis for discussing how patients’ access to new and effective treatment can be improved.
Access, RCT and HTA
“All effective treatments should be free.” This famous statement by Archie Cochrane was made in connection with an argumentation for increased use of randomized clinical trials (RCTs) for assessing outcome in medicine . He was convinced that if money were not spent on ineffective treatments and unproven indications, there should be enough resources to fund all available effective treatments.
Nearly 40 years later we know that scientific progress has outpaced the growth of economic resources available for health care. Medical research has produced an impressive growth in knowledge that can be used for improving health through prevention, early detection, treatment and rehabilitation. A significant part of this knowledge is embodied in new drugs that—combined with the improved skills of the medical professions—are translated into an ever-increasing number of new effective treatment options.
While the randomized clinical trial has emerged as the gold standard for assessing efficacy and effectiveness, additional tools have been developed to help health care decision-makers with the difficult task of allocating resources to optimize patients’ access to effective treatment. The most important of these is health technology assessment (HTA), which was introduced in the United States in the 1970s to help Congress make better informed decisions about innovation and technology. An HTA assessment includes an assessment of medical effectiveness, as advocated by Archie Cochrane, but also an analysis of wider social implications as well as an assessment of the balance between effectiveness and the costs involved, i.e., the cost effectiveness of the technology. Over time, HTA has gain widespread use as an important tool and instrument for making policy decisions in health care. From the beginning, drugs were not the focus of HTA, since there was a formal procedure for assessing the safety, efficacy and quality of drugs in place in most countries. However, over time, drugs have been targeted for assessment of cost effectiveness as a basis for reimbursement decisions.
Access and reimbursement
Since reimbursement is at the centre of patient access, and patient access to effective treatments is the fundamental goal of private and public “third party” spending on health care, the role of economic factors for patients’ access to treatment is back on the agenda, albeit in a new form. The development of public health care systems in Europe and the Medicare and Medicaid programs in the United States had the explicit goal of reducing economic barriers to care. Focus was on differences in access to care between different parts of the population, particularly for individuals with low incomes such as the elderly or chronically ill. Later research, particularly the trailblazing studies by Wennberg on “small area variations,” revealed that income was not the only factor that could influence access to care . Characteristics of the health care system, in particular incentives for the providers, were also important determinants of access.
Access and the internationalization of health care
With the internationalization of health care, the discussion about patients’ access to health care has reached a new dimension. Innovation and new medical technologies, in particular new drugs and devices, are created through investments in public and private research. The interaction between public investments in basic and translational research and private investments by global commercial companies is increasingly recognized as a driving factor for medical innovation. Medical innovation has also become a significant factor for economic growth and development in many countries. A consequence of the globalization of health-care markets is that prices, and thus costs of medical innovation, in particular drugs and devices, tend to be similar in different countries, despite striking differences in income and wealth. A new drug with a price set for the US market, where the average spending on health care is 6,000 USD per capita, should be accommodated into the Bulgarian health care system that spends about 600 USD per capita on health care. This necessarily creates problems with access, accentuating the fact that patients in Bulgaria are well aware of the opportunities, and those with resources can also travel to have access to treatment.
Innovation in RA
RA is a disease in which the development described above is highly visible. Until the late 1990s innovation in this field was very slow. The drugs available were, with a few exceptions, old and cheap, and the surgical interventions are of benefit only to a minority of patients for well-defined indications. While the need for treatments that could affect the progression of the disease was widely recognized, there were many disappointments until the introduction of the first TNF inhibitor in the late 1990s. Clinical trials showed improved outcome both in terms of disease activity and functional disability. In addition, as is typical of the increased interest in a disease generated by innovation, more aggressive treatment with traditional DMARDs accompanied the introduction of the new treatment options [32, 33]. Management of RA has thus significantly changed over the past decade. New outcome instruments have been developed that can be employed both in clinical trials and in clinical practice  and enable rapid recognition of treatment outcome and adaptation of therapies in search of the optimal treatment strategy for each patient. With the wide array of treatments available, the important question today is hence not to identify the most cost-effective drug or treatment, but the best and most cost-effective treatment strategy that avoids both over- and under-treatment. Patients have therefore benefited in several ways from innovation. But innovation was, as usual, incremental, and thus the need for further innovation persists.
Typically, however, global innovation comes at a price. The treatment costs for the TNF inhibitors are in the magnitude of €10,000–15,000 per patient and year. As a comparison, traditional DMARD treatment would be in the magnitude of €500–1,500. There are two consequences of this. Firstly, at this price, very few patients can pay for the treatment out of their own funds. Reimbursement by private and public health insurers or governments is hence the key to access. Secondly, the total costs for the new class of drugs rapidly went up to over 90% of the total drug bill for this patient group. In addition, within the total drug budget, the TNF inhibitors climbed rapidly to the top of the list of most-sold drugs, replacing statins, proton pump inhibitors and antidepressants. In Sweden, for example, the three TNF inhibitors were numbers 1, 3 and 10 among the ten most-sold drugs in 2006, representing 8.1% of the total drug market.
Payers were not unprepared for the new situation. In many countries patient registries were created, including all patients treated with the new drugs. The main purpose of this was to make sure that the drugs were used for the correct indications. At the level of costs involved, it is important that the drugs first of all be given to patients with the greatest potential to benefit. Further purposes of the registries were to facilitate long-term follow-up, to make sure that benefits from clinical trials were confirmed in clinical practice, and to make sure that potential long-term side effects were identified. These registries have turned out to be very useful both for research and for health care policy decisions, providing another example of how innovation can produce positive externalities and benefits for patients, current as well as future.
To secure patient benefit is most important for patients and the health care system. But cost is also important, since this determines what the payers can afford as well as the reward for the innovator and thus incentives for further innovation. Since price is the major determinant of the intervention cost, the question also arises whether it is “reasonable” or not. In a market some buyers find it reasonable and some not, or rather find it reasonable for some patients or groups of patients (indications) and not for others. Price therefore determines patient access to a large extent, and we are back to the fundamental objective of health care systems to provide access for patients to available treatments, regardless of whether they can pay for them or not. The new situation is that we are assessing this in an international perspective, with different abilities to pay and different priorities in different health care systems and countries.
Introduction of new RA drugs—an exemplary process?
This report discusses the trade-off between cost and outcome, its determinants and its consequences. The focus is on a description of the uptake and use of these drugs in different countries, including an analysis of possible explanations for the variations found. In particular, we are interested in the influence of price, income and systematic policies for influencing usage such as health technology assessment (HTA) and economic evaluation (cost-effectiveness analyses). This is by no means a simple undertaking. There are no easily available comparative data on numbers of patients treated, and the sources of variations can be manifold: variations in the size and composition of the patient population, health care system characteristics including the medical profession, regulation of market access and reimbursement and economic factors such as the income level and health care spending per person. Since we believe that there is a common goal, i.e., that as many patients who need certain therapies should have access to them, we are, in this report, particularly interested in factors that can be influenced by public policy, at the national as well as the international level. The question about “reasonable” prices and optimal allocation of resources is addressed through a review of the extensive literature on the cost of RA and economic evaluation of treatments.
One of our conclusions is that the introduction of the TNF inhibitors can, in many ways, be seen as an exemplary process, based to a large extent on accumulation of evidence for decision-making regarding market authorization, reimbursement and use in different indications. But even if it can be a model for other innovations, there are still large opportunities for improvement. The importance of the health improvements for patients, as well as the significant private and public resources invested in new treatments for RA, makes continuous investigation of the progress mandatory in order to fully utilize the opportunities created by medical research and innovation.
Conflict of interest
This study has been funded by unrestricted grant from F. Hoffmann-La Roche Ltd., Basel, Switzerland.