Ewa Orlewska

Access to biologic treatment for rheumatoid arthritis in Central and Eastern European (CEE) countries

Summary Background The aim of this study was to assess and compare patients’ access to biologic anti-RA drugs in selected Central and Eastern European (CEE) countries and to analyze the determinants of differences between countries. Material/Methods This is a multi-country survey study, based on a combination of desk research and direct contact with national RA stakeholders. Data was collected using a pre-defined questionnaire. Affordability was measured using an affordability index, calculated comparing the index of health care expenditures to the price index, using Poland as an index of 1. Results The percentage of patients on biologic treatment in 2009 was highest in Hungary (5% RA patients on biologic treatment), followed by Slovenia (4.5%), Slovakia (3.5%), Czech Republic (2.92%), Romania (2.2%), Estonia (1.8%), and Croatia, Serbia, Poland (below 1.5%). Infliximab, etanercept, adalimumab and rituximab were included in the reimbursement system in all countries, but abatacept and tocilizumab were included only in Slovakia. In Slovenia, public payer covered 75% of the price, and 25% is covered by supplementary health insurance; in Bulgaria public payer covered 50% of etanercept and adalimumab costs, and 75% of rituximab cost. In other countries, biologic drugs are reimbursed at 100%. Affordability index for biologic drugs was the lowest in Slovenia (0.4). In each country national guidelines define which patients are eligible for biologic treatment. Disease Activity Score (DAS28) of over 5.1 and failure of 2 or more disease-modifying anti-RA drugs, including methotrexate, are commonly used criteria. Conclusions The most important factors limiting access to biologic anti-RA treatment in the CEE region are macroeconomic conditions and restrictive treatment guidelines.

Adherence to biologic DMARD therapies in rheumatoid arthritis

Importance of the field: The efficacy of the biologic disease-modifying antirheumatic drugs (DMARDs) shown in clinical trials may be jeopardized due to prevalent poor patient adherence. Areas covered in this review: Patient adherence including compliance and persistence with biologic DMARDs in rheumatoid arthritis. What the reader will gain: This is a comprehensive review of the literature. The various definitions and methodologies of measurement used in adherence research are reviewed and data are presented by separating compliance and persistence. Differences in compliance rates were mainly based on numerical trends. There was evidence for and against greater persistence with infliximab versus adalimumab and etanercept. There was a trend in favour of greater compliance and lower persistence with TNF-α inhibitor monotherapy versus in combination therapy with methotrexate. Take home message: The evidence suggests that adherence to biologic DMARDs is suboptimal. When further research is applied in the field, agreed definitions and methodology need to be used to allow for cross-study comparisons. In addition, adherence should be assessed in conjunction with clinical outcomes and not on its own so that it can be better understood what levels of adherence provide the required clinical outcomes.

Budget-Impact Analyses: A Critical Review of Published Studies

AbstractThis article reviews budget-impact analyses (BIAs) published to date in peer-reviewed bio-medical journals with reference to current best practice, and discusses where future research needs to be directed.Published BIAs were identified by conducting a computerized search on PubMed using the search term ‘budget impact analysis’. The years covered by the search included January 2000 through November 2008. Only studies (i) named by authors as BIAs and (ii) predicting financial consequences of adoption and diffusion of a new health intervention(s) within a specific healthcare setting were included. Relevant studies were evaluated according to the checklist that focuses on issues unique to BIA, highlighting areas of agreement or dissent between published studies and methodological guidelines.A total of 34 studies met the inclusion criteria, the majority published in 2007–8. Of these, 41% were from the US, 54% were prepared for pharmaceuticals and 65% had BIA as their main aim. The published BIAs were heterogeneous in respect of methods for deriving budget-impact estimates, time horizon and population. There is fairly good agreement between published studies and methodological guidelines within the scope of perspective, comparator, cost included and data sources. Specific issues that need to be addressed and/or improved are reporting format, sensitivity analysis and discounting.The results indicate that, recently, BIAs have appeared more frequently in peer-reviewed journals, providing stimulus to development, validation and dissemination of methods. Many published studies fail to reach the desired quality, but this situation should change with good research practice principles that will help codify and clarify important issues and promote standardization and transparency. Future research needs to be directed to quality assurance of published BIAs and investment in data collection for parameters specific to BIAs.

Health economics and health technology assessment in Central and Eastern Europe: a dose of reality

It is an opinion widely shared, at least among health economics (HE) and health technology assessment (HTA) experts, that the need for HE and HTA is increasing due to two main factors. One is the more and more serious budget constraints of the recession, the other increasingly demanding policy makers and funders who require greater evidence for new and existing therapies. But is this really the case? Are policy makers and funders aware of the potentials and limitations of HE and HTA? If they are convinced and committed to use HE and HTA results, is there sufficient capacity at national level to provide the required HE and HTA evaluations both in terms of quantity and quality? Are these methodologies and results recognised or valued during the decision making process? More than 20 years ago, in the early 1990s when the political systems in Central and Eastern European countries changed from socialism to democracy, many of us believed that HE and HTA would spread quickly around the region. We thought HE and HTA institutions and university departments would be established and that this development would be boosted further by the pure fact of EU enlargement incorporating all new member states. We had a dream that more and more resource allocation decisions related to drug reimbursement and public health would be based on results, provided nationally, from HTA and HE. At that time we had no doubt that, due to sustainable economic growth, fresh money would be allocated to different parts of health care systems under the title of ‘‘resource allocation’’. Our methodology was set to assist decisions under this economic condition. Various projects funded by the European Commission, and worldwide (INAHTA) and European networks (EunetHTA) pushed things further. However, this dream is still sluggish in coming to fruition. Still today, the role of HE and HTA is very limited in the new member states. Only two countries, Poland and Hungary, have established HTA offices and some university departments of HE. In the following, we will see what has been achieved so far, look at possible explanations for this slow development, and discuss the main issues still waiting to be resolved.

Project of Polish guidelines for conducting pharmacoeconomic evaluations in comparison to international health economic guidelines

We present the project of Polish guidelines for conducting economic evaluation and compare them with international health economic guidelines, highlighting areas of agreement and dissent. The Polish guidelines are very similar to those in Australia, Canada, and some European Union countries. Nevertheless a number of implementation and attitude problems seem to have delayed the introduction of pharmacoeconomic evaluation as a prerequisite for reimbursement of new pharmaceutical products. A transitional phase is required for collecting reliable cost data and familiarizing and training personnel both in public authorities and private industry

Economic burden of multiple sclerosis: what can we learn from cost-of-illness studies?

The purpose of this article is to provide a critical evaluation of the available literature describing the economic burden of multiple sclerosis and to assess the gaps in information. The studies included in this review describe costs in patients categorized into severity groups according to the Expanded Disability Status Scale levels. Despite the differences in methodology, as well as differences between countries in absolute and relative prices and organization of healthcare systems, all cost-of-illness studies demonstrate that multiple sclerosis represents a major financial burden on the individual, family, health services and society in all countries studied, and that a positive relationship exists between costs (both direct and indirect) and level of disability measured by Expanded Disability Status Scale level. The results of the cost-of-illness studies in the field of multiple sclerosis can be used as an input into future economic evaluation, but provide no useful information about future resource allocation on their own. The current focus of future research efforts should be in refining economic methods, specifically for indirect costs, improving interpretation and communication of study findings and conducting cost–effectiveness assessments of interventions based on results of cost-of-illness studies. Further research should be targeted to patients treated with new disease-modifying drugs.

Cost-Effectiveness Analysis of Enoxaparin versus Unfractionated Heparin in Patients with Acute Coronary Syndrome in Poland: Modelling Study from the Hospital Perspective

AbstractAim and perspective: To estimate the cost effectiveness of enoxaparin versus unfractionated heparin (UFH) in patients with acute coronary syndrome (ACS) from a Polish hospital perspective. This was intended to facilitate the decision-making process in selecting the most cost-effective treatment for ACS. Method: A decision model was used to quantify costs and effectiveness of alternative treatments. Published results from the Efficacy and Safety of Subcutaneous Enoxaparin in Non-Q-Wave Coronary Events (ESSENCE) study were used to estimate the probability for clinical endpoints (death, myocardial infarction or recurrent angina) at 30 days. Probabilities of patients undergoing revascularisation procedures were obtained from the Global Registry of Acute Coronary Events (GRACE) which included data from 961 patients at six centres in Poland. The analysis assessed only direct medical costs, determined from actual resource consumption on a patient-specific basis (6-month observational study) and estimated using Polish data on unit costs. One- and two-way sensitivity analyses and threshold analysis were performed. Results: At 30 days, 19.8% of patients receiving enoxaparin compared with 23.3% of those receiving UFH reached a composite endpoint consisting of death, myocardial infarction and recurrent angina (p = 0.02). The average costs (in zloty [Zl];

Costs and Effects of Secondary Prevention with Perindopril in Stable Coronary Heart Disease in Poland : An Analysis of the EUROPA Study Including 1251 Polish Patients

US1 = Z14 [2000 values]) were 1085 per patient receiving enoxaparin compared with 1097 per patient receiving UFH. Therefore, for every 29 patients treated, not only would enoxaparin therapy avoid one event of the composite endpoint, it would also save Z1348. The threshold analysis suggests that enoxaparin would lose dominance when the cost of enoxaparin increased by 10%, the cost of monitoring UFH therapy decreased by 12%, the probability of reaching the composite endpoint in the enoxaparin arm increased to 0.22 or decreased to 0.21 in the UFH arm. Conclusion: According to our model enoxaparin was more effective at a lower cost than UFH, therefore this treatment was shown to be dominant for patients with ACS in Poland.

Cost-effectiveness of pegylated IFN-α2b and -2a and ribavirin for chronic hepatitis C treatment

AbstractObjectives: To estimate the long-term impact of treatment with perindopril on costs and health effects in patients with stable coronary artery disease in Poland. Methods: The cost-effectiveness analysis was based on data from a randomized double-blind, placebo-controlled trial. A decision-tree analysis was employed, including Monte Carlo and bootstrapping techniques. This study was a sub-study of the EUROPA (European Trial on Reduction of Cardiac Events with Perindopril in Stable Coronary Artery Disease) trial (n = 12 218; mean follow-up 4.2 years). Resource use was based on data from Polish EUROPA study patients (n = 1251), while effectiveness was based on the whole EUROPA study. The health gain of perindopril in life-years was based on overall EUROPA study results, and the adapted Polish life expectancy of patients not dying during the trial. Costs were calculated in new Polish zloty (PLN), year 2003 values; €1 = PLN4.053. Only direct healthcare costs related to cardiovascular events and medication use were studied. Results: When observed mortality was combined with life expectancy beyond the end of the study, perindopril use showed a gain in life expectancy of 0.182 lifeyears (SD ± 0.129) at a cost of PLN1983 (SD ± 103) with discounting of 5% per annum on costs and no discounting on effects. This resulted in an incremental cost-effectiveness ratio (ICER) of PLN10 896 per life-year gained. The probability that the ICER for perindopril was below the threshold of PLN60 000 was 88%. The overall results were insensitive to discount rates for costs and life-years. Conclusions: Perindopril leads to a reduction in the risk of coronary events among patients with stable heart disease. When the expected improvement in life expectancy is combined with associated medical costs, there is a high probability that perindopril is cost effective, given the threshold of PLN60 000 per life-year gained.

Principles of Good Practice for Budget Impact Analysis: Report of the ISPOR Task Force on Good Research Practices—Budget Impact Analysis

Pegylated interferon-α2a (Pegasys®, Roche) and pegylated interferon-α2b (Peg-Intron®, Schering-Plough) with ribavirin (Copegus®, Roche; Rebetol®, Schering-Plough) is now acknowledged as the therapy of choice for chronic hepatitis C. This article reviews the existing evidence concerning the cost-effectiveness of pegylated interferon and ribavirin versus nonpegylated interferon and ribavirin for the treatment of chronic hepatitis C and highlights key issues for future research. Recently published cost-effectiveness analyses suggest that use of pegylated interferon and ribavirin should reduce the cumulative incidence of liver complications, increase life expectancy, improve quality of life, and be cost-effective in comparison with nonpegylated interferon and ribavirin. The cost and benefits of the treatment depend on factors such as patient age, sex, viral load, histological severity and viral genotype. Studies of the natural history of chronic hepatitis C and randomized clinical trials of pegylated interferon and ribavirin versus other therapies need to be performed in special populations of patients to provide valuable data that could be incorporated into future cost-effectiveness analyses. The direct comparison of two marketed pegylated interferons in terms of cost-effectiveness is needed. The cost-effectiveness analyses of the two pegylated combination products should be preceded by a head-to-head clinical trial that would provide evidence of comparative efficacy.