Jenny Berg

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

Costs and quality of life of multiple sclerosis in Germany

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.

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

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.

Costs and quality of life in multiple sclerosis in Europe: method of assessment and analysis.

Background: The current focus in multiple sclerosis (MS) is on early diagnosis and drug intervention, with a view to modifying disease progression. Consequently, healthcare costs have shifted from inpatient care and rehabilitation to outpatient care. Objectives: This European burden of illness study provides data that can be combined with other evidence to assess whether management approaches provide value to society. Methods: A cross-sectional study was conducted in 16 countries. Patients reported on their disease, health-related quality of life (HRQoL) and resource consumption. Descriptive analyses were performed by disease severity. Costs are reported from a societal perspective in 2015€ PPP (adjusted for purchasing power parity). Results: The 16,808 participants had a mean age of 51.5 years, and 52% had relapsing–remitting multiple sclerosis (RRMS). Work capacity declined from 82% to 8%, and utility declined from normal population values to less than zero with advancing disease. Mean costs were 22,800€ PPP in mild, 37,100€ PPP in moderate and 57,500€ PPP in severe disease; healthcare accounted for 68%, 47% and 26%, respectively. Fatigue and cognitive difficulties were reported by 95% and 71% of participants, respectively; both had a significant independent effect on utility. Conclusion: Costs and utility were highly correlated with disease severity, but resource consumption was heavily influenced by healthcare systems organisation and availability of services.

Modeling the cost-effectiveness of a new treatment for MS (natalizumab) compared with current standard practice in Sweden

The objective of this observational study in nine European countries was to establish the current cost of MS at different levels of disease severity. The study used a prevalence-based bottom-up to estimate costs from a societal perspective. Patients were recruited via patient organizations and neurology centres and asked by mail to complete a detailed questionnaire concerning their resource consumption, work capacity, quality of life and current disease status. Resources included all health-care and social services, personal expenditures, informal care and temporary or definitive work absence. Quality of life was collected as utility and disease information collected related to functional capacity, relapses and fatigue. The data were analyzed separately for each country, using local unit costs but a fully standardized methodology. A total of 13 186 patients at all levels of disease severity participated in the survey, with an average response rate of around 40% (range 20–75%).This paper describes the methodology used for the study, while results are presented in the individual country reports.

Costs and quality of life of multiple sclerosis in Switzerland

Objective To estimate the cost-effectiveness of a new treatment (natalizumab) for multiple sclerosis (MS) compared with current standard therapy with disease-modifying drugs (DMDs) in Sweden. Methods A Markov model was constructed to illustrate disease progression based on functional disability (the Expanded Disability Status Scale (EDSS)). The effectiveness of natalizumab was based on a 2-year clinical trial in 942 patients (AFFIRM). The effectiveness of current DMDs was estimated from a matched sample of 512 patients in the Stockholm MS registry. Patients withdrawing from treatment were assumed to follow the disease course of 824 patients with relapsing–remitting disease at onset in the Ontario natural history cohort. Costs and utilities are based on a recent observational study in 1339 patients. All data sets were available at the patient level. Main results are presented from the societal perspective, over a 20-year time frame, in 2005 Euros (€1 = 9.25 SEK). Results In the base case, treatment with natalizumab was less expensive and more effective than treatment with current DMDs. When only healthcare costs were considered, the cost per quality-adjusted life year gained with natalizumab was €38 145. Results are sensitive only to the time horizon of the analysis and assumptions about effectiveness of natalizumab beyond the trial. Conclusions This cost-effectiveness analysis used registry data, cohort and observational studies to extrapolate the efficacy findings of natalizumab from the AFFIRM clinical trial to measure effectiveness in clinical practice. The analysis results suggest that for the population considered, natalizumab provides an additional health benefit at a similar cost to current DMDs from a societal perspective.

An economic evaluation of NIOX MINO airway inflammation monitor in the United Kingdom

This cost-of-illness analysis, based on information from 1101 patients in Switzerland, 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 Swiss MS patient organization were asked to participate in a mail survey and 44% 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, informal care and work capacity (sick leave and early retirement). In addition, patients were asked about their current QOL (measured as utility) and the level of fatigue. The mean age of respondents was 53 years, and 21% were 65 years or older. Almost one-quarter of patients in the sample (23%) had severe disease (Expanded Disability Status Scale [EDSS] score ≥7), while 36% had moderate disease (EDSS score 4–6.5) and 38% had mild disease (EDSS score 0–3). The median EDSS score was 5.0, with a mean of 4.5. Mean utility was 0.53 (range, 0 = death to 1 = full health), while mean fatigue was rated as 5.4 (range, 1 = not tired to 10 = extremely tired). Utility and fatigue were worse for patients with a recent relapse. Costs and utility are highly correlated with disease severity. Workforce participation decreases from greater than 80% in early disease to less than 10% in the very late stages, while total costs increase more than fivefold between an EDSS score of 0–1 and an EDSS of 7, and by more than 40% from an EDSS score of 7 to worse states. Most cost types increase steadily until patients reach an EDSS score of 7, after which there is a sharp increase due to home care, home services and informal care. The mean annual cost per patient in the sample (65.000 Swiss francs, € 42.000) corresponds to costs for a patient at the median EDSS score of 5. Utility decreases from 0.89 to 0.1 as the disease becomes severe, but the loss compared to the age- and gender-matched general population can be observed at all levels of the disease (∼0.1 at an EDSS score of 2 to ∼0.3 at an EDSS score of 5-6), leading to an estimated annual loss of 0.3 quality-adjusted life-years (QALY) per patient in the sample. Relapses for patients with an EDSS score <5 were associated with a utility loss of 0.08 and an incremental cost of 5500 Swiss francs (€ 3.500) during the quarter in which they occurred. Health-care costs are to a large extent covered by insurances, and a substantial amount of services such as home care and home help are available to patients with severe disease. These services represent approximately 15% of total societal costs and are largely covered, resulting in almost 30% of total costs to different payers. Despite this, a large amount of costs falls on families providing care, in particular for patients with severe disease.

The burden of multiple sclerosis 2015: Methods of data collection, assessment and analysis of costs, quality of life and symptoms:

Background:  Fractional exhaled nitric oxide (FENO), a marker of eosinophilic airway inflammation, is easily measured by noninvasive means. The objective of this study was to determine the cost‐effectiveness of FENO measurement using a hand‐held monitor (NIOX MINO), at a reimbursement price of £23, for asthma diagnosis and management in the UK.

Cost-effectiveness of clopidogrel treatment in percutaneous coronary intervention: a European model based on a meta-analysis of the PCI-CURE, CREDO and PCI-CLARITY trials.

Introduction: This article describes the methods used to perform this large European-wide burden-of-illness study on multiple sclerosis (MS) using individual patient data. Methods: The study collected all MS-related resource consumption, workforce participation, prevalent disease symptoms and health-related quality of life (HRQoL). Patients were recruited by national patient associations and, after informed consent, completed a specific questionnaire either on-line or on paper. Analyses were performed by country as well as for the study overall. Costs were estimated from the societal perspective, using publicly available unit costs and reported in national currencies and in EUR 2015 adjusted for purchasing power parity. The results are reported by disease severity groups according to self-assessed Expanded Disability Status Scale (EDSS) (mild, moderate, severe) and by EDSS point to highlight the development of costs as disability progresses. Results: A total of 16,808 patients in 16 countries participated in the study: Austria, Belgium, Denmark, Czech Republic, France, Germany, Hungary, Italy, the Netherlands, Poland, Portugal, Russia, Spain, Sweden, Switzerland and the United Kingdom. Conclusion: This study, endorsed by the European Platform of MS Societies, provides up-to-date information on costs and expands the previously available information on HRQoL and symptoms.