Gisela Kobelt

Costs and quality of life of patients with multiple sclerosis in Europe

Objective: To assess overall resource consumption, work capacity and quality of life of patients with multiple sclerosis in nine European countries. Methods: Information on resource consumption related to multiple sclerosis, informal care by relatives, productivity losses and overall quality of life (utility) was collected with a standardised pre-tested questionnaire from 13 186 patients enrolled in national multiple sclerosis societies or followed up in neurology clinics. Information on disease included disease duration, self-assessed disease severity and relapses. Mean annual costs per patient (€, 2005) were estimated from the societal perspective. Results: The mean age ranged from 45.1 to 53.4 years, and all levels of disease severity were represented. Between 16% and 29% of patients reported experiencing a relapse in the 3 months preceding data collection. The proportion of patients in early retirement because of multiple sclerosis ranged from 33% to 45%. The use of direct medical resources (eg, hospitalisation, consultations and drugs) varied considerably across countries, whereas the use of non-medical resources (eg, walking sticks, wheel chairs, modifications to house and car) and services (eg, home care and transportation) was comparable. Informal care use was highly correlated with disease severity, but was further influenced by healthcare systems and family structure. All types of costs increased with worsening disease. The total mean annual costs per patient (adjusted for gross domestic product purchasing power) were estimated at €18 000 for mild disease (Expanded Disability Status Scale (EDSS) <4.0), €36 500 for moderate disease (EDSS 4.0–6.5) and €62 000 for severe disease (EDSS >7.0). Utility was similar across countries at around 0.70 for a patient with an EDSS of 2.0 and around 0.45 for a patient with an EDSS of 6.5. Intangible costs were estimated at around €13 000 per patient.

Costs and quality of life in multiple sclerosis A cross-sectional study in the United States

Objective: To estimate current costs and quality of life (utility) of patients treated with disease modifying drugs (DMDs) in the United States, and to investigate the effect of disease severity on costs and utility. Methods: Data on demographics, disease information, resource utilization (including work capacity), and utility were collected directly from patients in a cross-sectional postal survey. Patients were selected randomly among participants in the North American Committee on Multiple Sclerosis Patient Registry (NARCOMS) receiving DMDs. Mean annual costs per patient (2004 USD) and mean utilities for the sample and for different levels of functional disability are estimated from the societal perspective. Results: The target answer rate of 50% was reached and 1,909 patients were included in the analysis. The mean age was 49 years and time since diagnosis was 13 years. A total of 10.5% of patients had primary progressive, 47.6% relapsing-remitting, and 33.3% secondary progressive disease. A total of 28.8% of patients indicated to have experienced a relapse during the past 3 months. Total average costs are estimated at

The burden of rheumatoid arthritis and access to treatment: health burden and costs.

47,215 per patient and year. Of these, 53% were for direct medical and non-medical costs, 37% for production losses, and 10% for informal care. Mean utility in the sample was 0.698. Conclusions: Consistent with other studies, costs and utilities were significantly correlated with functional capacity. The proportion of costs represented by disease modifying drugs is considerably lower than estimated in other studies, as all costs related to the disease are included.

Costs and quality of life of multiple sclerosis in Germany

As part of the study “The burden of rheumatoid arthritis and patient access to treatment”, this paper reviews evidence on the health burden of rheumatoid arthritis (RA) in terms of morbidity (DALYs), mortality (% of deaths attributable to RA) and quality of life (utility and loss of QALYs), as well as the economic impact on society. Based on available literature on the prevalence and the cost of RA, combined with economic indicators, the annual cost per patient as well as the total national cost is estimated for Europe and North America (Canada and the United States), as well as Australia, Turkey, the Russian Federation and South Africa. Total costs to society were estimated at €45.3 billion in Europe and at €41.6 billion in the United States. Utility scores were found to be amongst the lowest compared to other diseases.

Cost effectiveness of etanercept (Enbrel) in combination with methotrexate in the treatment of active rheumatoid arthritis based on the TEMPO trial

This cost-of-illness analysis based on information from 921 patients in Italy is part of a Europe-wide cost-of-illness study in multiple sclerosis (MS). The objective was to analyze the costs and quality of life (QOL) related to the level of disease severity and progression. Patients registered with the Italian MS patient organization were asked to participate in a mail survey, and 31% responded. The questionnaire asked for details on the disease (type of disease, relapses, level of functional disability), information on all medical and non-medical resource consumption, and informal care and work capacity (sick leave and early retirement). In addition, patients were asked about their current QOL (in the form of utility) and the level of fatigue. The mean age of respondents was 46 years, and 8.5% were 65 years or older. As many as 20% of patients had severe disease (Expanded Disability Status Scale [EDSS] score of ≥7), 47% had moderate disease (EDSS score of 4–6.5) and only 31% had mild disease (EDSS score of 0–3). Thus, the mean EDSS score in the sample was 4.6 (median 5.0), with a utility of 0.53 (range: 0 = death to 1 = full health) and a fatigue level of 6.4 (range: 1 = not tired to 10 = extremely tired). Costs and utility are highly correlated with disease severity. Workforce participation decreases from approximately 80% in early disease to less than 10% in the very late stages. Total costs increase fivefold between an EDSS score of 0–1 and a score of 7. Health-care costs, however, show a limited increase with worsening disease – hospitalization increases from € 800 per patient to € 3200, and ambulatory care increases from € 900 to € 1500. Productivity losses, on the other hand, increase by a factor of 12, while informal care increases from € 500 at an EDSS score of 0–1 to nearly € 25 000 at an EDSS score of 7, and € 39 000 at an EDSS score of 8–9. Hence, total mean costs per patient are determined essentially by the distribution of the severity levels in the sample, increasing from € 12 000 at an EDSS score of 0–1 to € 57 000 at an EDSS score of 7, and € 71 000 at an EDSS score of 8–9. The same is true for utility, which decreases from 0.80 to 0.06 as the disease becomes severe. However, the utility loss compared to the age- and gender-matched general population is high at all levels of the disease, leading to an estimated annual loss of 0.3 quality-adjusted life year (QALY) per patient. Relapses for patients with an EDSS score of <5 are associated with a cost of approximately € 4000 and a utility loss of 0.18 during the quarter in which they occur.

The burden of rheumatoid arthritis and access to treatment: uptake of new therapies

Objective: To estimate the cost effectiveness of combination treatment with etanercept plus methotrexate in comparison with monotherapies in patients with active rheumatoid arthritis (RA) using a new model that incorporates both functional status and disease activity. Methods: Effectiveness data were based on a 2 year trial in 682 patients with active RA (TEMPO). Data on resource consumption and utility related to function and disease activity were obtained from a survey of 616 patients in Sweden. A Markov model was constructed with five states according to functional status (Health Assessment Questionnaire (HAQ)) subdivided into high and low disease activity. The cost for each quality adjusted life year (QALY) gained was estimated by Monte Carlo simulation. Results: Disease activity had a highly significant effect on utilities, independently of HAQ. For resource consumption, only HAQ was a significant predictor, with the exception of sick leave. Compared with methotrexate alone, etanercept plus methotrexate over 2 years increased total costs by €14 221 and led to a QALY gain of 0.38. When treatment was continued for 10 years, incremental costs were €42 148 for a QALY gain of 0.91. The cost per QALY gained was €37 331 and €46 494, respectively. The probability that the cost effectiveness ratio is below a threshold of €50 000/QALY is 88%. Conclusion: Incorporating the influence of disease activity into this new model allows better assessment of the effects of anti-tumour necrosis factor treatment on patients’ general wellbeing. In this analysis, the cost per QALY gained with combination treatment with etanercept plus methotrexate compared with methotrexate alone falls within the acceptable range.

Costs and quality of life in multiple sclerosis

This paper presents data on international differences in use of TNF inhibitors. It is part of a study on burden and cost of RA, access to new therapies and the role of HTA in determining access and cost-effectiveness. United States has the fastest most extensive use of the new drugs, about three times the average in the western European countries and Canada. Eastern and central European countries as well as Australia, South Africa and Turkey lag far behind. However, some smaller European countries, most notably Norway and Sweden have use of the new drugs not far behind the United States. While the income level of the country, and thus the health care expenditures per capita is a major factor for determining use in low and middle income countries, there are still considerable differences among countries with similar high total health care expenditures. Differences in prices are considerable between the US and Europe due to the changes in exchange rates between the US dollar and the Euro, but high and low use is not systematically related to differences in price.

Disease status, costs and quality of life of patients with rheumatoid arthritis in France: The ECO-PR Study.

We performed a cross-sectional, “bottom-up” observational study of resource use, costs, and quality of life in patients with multiple sclerosis (MS) in Germany. Six centers participated in the study. Patients were asked to complete a questionnaire, and a total of 737 patients returned it (response rate 66%). The questionnaire provided information on all resource consumption, medical, and nonmedical, work absence, informal care related to their MS, and quality of life (EuroQol). Simultaneously, medical charts were also abstracted for a subsample of 202 patients for comparison between answers in the questionnaires and registered data. Levels of disability were assessed using the Expanded Disability Status Scale. The mean age of the cohort was 41.9±14.1 years (disease onset 33.4), mean EDSS score 4.4 (range 1.0–9.5), and mean utility measured by EQ-5D 0.552±0.331). Mean total cost per patient and year was 65,400 DM, adjusted for use of interferons, which was higher in this sample than the current average use in Germany. When this cost is extrapolated to an estimated patient population of 120,000, total costs to society are estimated at 7.85 billion DM. Direct costs represented 57.5%, informal care accounted for 12.1% and indirect costs amounted to 42.5%. Public payers pay for an estimated 24,800 DM per patient or 38% of total costs. All types of costs (direct, informal care, indirect) increased with increasing disability, while utilities decreased.

Estimation of the cost of MS in Europe : Extrapolations from a multinational cost study

OBJECTIVE To investigate resource consumption and quality of life (utility) in a sample of patients covering the full spectrum of the disease, modalities of patient management and geographic areas. METHODS Information on demographics, disease parameters, work capacity and resource consumption (in the past 1, 3 or 12 months depending on the resource) was collected in an anonymous mail survey from all members of a national patient association (ANDAR). Results are presented for the sample and by level of functional capacity, in 2005 euro. RESULTS 1487 patients were included in the analysis (response rate 49%). Mean age was 62.7 years and 83.5% of respondents were female. Mean disease duration was 18 years; mean HAQ was 1.42; fatigue and pain ranked 5.6 and 4.8 on a scale between 0 and 10, respectively. Of patients below 60 years, 34% had taken early retirement due to RA, and only 15% of patients with a HAQ of 2 or higher were working. Productivity losses were estimated at 5076 euro, of which indemnity payments covered 1944 euro. Direct health care costs were 11,757 euro in the societal perspective and 9216 euro in the perspective of the national health insurance. Direct non-medical costs (including informal care) were 4857 euro and 136 euro respectively. Costs to society increased from 9400 euro in mild disease to 40,700 euro in severe disease, and to public payers from 6000 euro to 19,000 euro. Utility decreased simultaneously from 0.80 to 0.06. CONCLUSION The study confirms overall findings in other studies in other countries, and provides the first estimate of all costs by disease severity in France.

New insights into the burden and costs of multiple sclerosis in Europe

The present study aims at estimating the total cost of MS in Europe based on actual cost data from nine countries and published epidemiological evidence. The epidemiological data are reported as 12 months prevalence estimates and cost data calculated as annual cost per patient at given levels of disease severity. Cost data are extrapolated to the rest of Europe based on a model, using economic indexes adjusting for price level differences in different sectors between countries. The aggregated annual cost estimates are presented in Euro for 2005. In 28 European countries with a population of 466 million, an estimated 380 000 individuals are affected by MS. The total annual cost of MS in Europe is estimated at 12.5 billion in year 2005, corresponding to a cost of 27 per European inhabitant. Direct costs represent slightly more than half of the total cost (6.0 billion). Informal care is estimated at 3.2 billion, and indirect costs due to morbidity at 3.2 billion. Thus, the largest component of costs is found outside the formal health care sector. Although our model appears to predict costs reasonably well, when comparing to previous national studies not included in the estimates, there are considerable uncertainties when extrapolating cost data across countries even within Europe. These weaknesses can only be overcome by collecting primary data. Multiple Sclerosis 2007; 13: 1054—1064. http://msj.sagepub.com