Plovdiv, Bulgaria
Plovdiv, Bulgaria

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Iskrov G.,Medical university-Plovdiv | Hristova E.,Medical university-Plovdiv | Stefanov R.,Medical university-Plovdiv | Stefanov R.,Institute of Rare Diseases
General Medicine | Year: 2015

Over the last decade, rare diseases have been gradually established as a priority area for the EU public health policy. The specifics of these conditions - a great number of distinct nosologies with low prevalence, severe clinical course and prognosis, limited information and expertise, lack of therapies - impose initiating cooperation and sharing experience in order to use in optimal way the available resources for these diseases. In Bulgaria, Ordinance No 16 on the conditions and procedures for registration of rare diseases and designation of centers of expertise and reference networks for rare diseases was adopted in 2014. This act is expected to improve significantly the prevention, diagnosis, treatment, follow-up and rehabilitation of rare diseases in the country. The practical implementation of these objectives, however, largely depends on the awareness and motivation of rare disease stakeholders to initiate and take part in such activities. Health authorities could eliminate this risk by adopting policy measures, including financial incentives for the future centers of expertise and reference networks for rare diseases in Bulgaria.


PubMed | Institute of Rare Diseases Research, Corvinus University of Budapest, Instituto Superiore Of Sanita Iss, Instituto Of Ricerche Farmacologiche Mario Negri and 8 more.
Type: | Journal: The European journal of health economics : HEPAC : health economics in prevention and care | Year: 2016

The aim of this study was to determine the social/economic costs and health-related quality of life (HRQOL) of patients with epidermolysis bullosa (EB) in eight EU member states.We conducted a cross-sectional study of patients with EB from Bulgaria, France, Germany, Hungary, Italy, Spain, Sweden and the United Kingdom. Data on demographic characteristics, health resource utilisation, informal care, labour productivity losses, and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire.A total of 204 patients completed the questionnaire. Average annual costs varied from country to country, and ranged from 9509 to 49,233 (reference year 2012). Estimated direct healthcare costs ranged from 419 to 10,688; direct non-healthcare costs ranged from 7449 to 37,451 and labour productivity losses ranged from 0 to 7259. The average annual cost per patient across all countries was estimated at 31,390, out of which 5646 accounted for direct health costs (18.0%), 23,483 accounted for direct non-healthcare costs (74.8%), and 2261 accounted for indirect costs (7.2%). Costs were shown to vary across patients with different disability but also between children and adults. The mean EQ-5D score for adult EB patients was estimated at between 0.49 and 0.71 and the mean EQ-5D visual analogue scale score was estimated at between 62 and 77.In addition to its negative impact on patient HRQOL, our study indicates the substantial social/economic burden of EB in Europe, attributable mostly to high direct non-healthcare costs.


PubMed | Institute of Rare Diseases Research, Bocconi University, Corvinus University of Budapest, Instituto Of Ricerche Farmacologiche Mario Negri and 9 more.
Type: | Journal: The European journal of health economics : HEPAC : health economics in prevention and care | Year: 2016

The aim of this study was to determine the economic burden from a societal perspective and the health-related quality of life (HRQOL) of patients with juvenile idiopathic arthritis (JIA) in Europe.We conducted a cross-sectional study of patients with JIA from Germany, Italy, Spain, France, the United Kingdom, Bulgaria, and Sweden. Data on demographic characteristics, healthcare resource utilization, informal care, labor productivity losses, and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D-5L) questionnaire.A total of 162 patients (67 Germany, 34 Sweden, 33 Italy, 23 United Kingdom, 4 France, and 1 Bulgaria) completed the questionnaire. Excluding Bulgarian results, due to small sample size, country-specific annual health care costs ranged from 18,913 to 36,396 (reference year: 2012). Estimated direct healthcare costs ranged from 11,068 to 22,138; direct non-healthcare costs ranged from 7837 to 14,155 and labor productivity losses ranged from 0 to 8715. Costs are also shown to differ between children and adults. The mean EQ-5D index score for JIA patients was estimated at between 0.44 and 0.88, and the mean EQ-5D visual analogue scale score was estimated at between 62 and 79.JIA patients incur considerable societal costs and experience substantial deterioration in HRQOL in some countries. Compared with previous studies, our results show a remarkable increase in annual healthcare costs for JIA patients. Reasons for the increase are the inclusion of non-professional caregiver costs, a wider use of biologics, and longer hospital stays.


PubMed | Institute of Rare Diseases Research, Corvinus University of Budapest, Instituto Superiore Of Sanita Iss, Mario Negri Institute for Pharmacological Research and 8 more.
Type: | Journal: The European journal of health economics : HEPAC : health economics in prevention and care | Year: 2016

The aim of this study was to determine the economic burden from a societal perspective and the health-related quality of life (HRQOL) of patients with Duchenne muscular dystrophy (DMD) in Europe.We conducted a cross-sectional study of patients with DMD from Bulgaria, France, Germany, Hungary, Italy, Spain, Sweden, and the UK. Data on demographic characteristics, healthcare resource utilization, informal care, labor productivity losses, and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire. Costs have been estimated from a societal perspective adopting a bottom-up approach.A total of 422 questionnaires were included in the study; 268 of which were collected from patients with DMD and 154 from caregivers. The average annual cost per person in 2012 ranged from 7657 in Hungary to 58,704 in France. Direct non-healthcare costs are the main component of whole costs and informal care is the main driver of non-healthcare costs. Costs are also shown to differ between children and adults. With regard to HRQOL of adult patients, the EQ-5D VAS score and EQ-5D index scores were 50.5 and 0.24, respectively. The corresponding EQ-5D VAS and EQ-5D index scores for caregivers were 74.7 and 0.71, respectively.We have estimated the average annual cost per patient with DMD in eight European countries adopting a social perspective, and to our knowledge this is the first study with such a wide perspective. The results on costs show a considerable gap between Eastern and Western European countries. Non-healthcare costs range from 64 to 89% of overall costs and informal care is to a great extent the main driver of this cost category. The HRQOL of people with DMD is much lower than that of the general population.


PubMed | Institute Investigacion en Enfermedades Raras IIER, Corvinus University of Budapest, Instituto Superiore Of Sanita Iss, Instituto Of Ricerche Farmacologiche Mario Negri and 9 more.
Type: | Journal: The European journal of health economics : HEPAC : health economics in prevention and care | Year: 2016

The aim of this study was to determine the economic burden from a societal perspective and the health-related quality of life (HRQOL) of patients with systemic sclerosis (SSc; scleroderma) in Europe.We conducted a cross-sectional study of patients with SSc (involving both localised and systemic sclerosis) from Germany, Italy, Spain, France, the UK, Hungary and Sweden. Data on demographic characteristics, healthcare resource utilisation, informal care, labour productivity losses and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire.A total of 589 patients completed the questionnaire. The rate of patients with localised scleroderma, limited cutan and diffuse cutan SSc were 28, 68 and 4%, respectively. Average annual costs varied from country to country and ranged from 4607 to 30,797 (reference year: 2012). Estimated direct healthcare costs ranged from 1413 to 17,300; direct non-healthcare costs ranged from 1875 to 4684 and labour productivity losses ranged from 1701 to 14,444. The mean EQ-5D index score for adult SSc patients varied from 0.49 to 0.75 and the mean EQ-5D visual analogue scale score was between 58.72 and 65.86.The main strengths of this study lie in our bottom-up approach to costing and our evaluation of SSs patients from a broad societal perspective. This type of analysis is very unusual in the international literature on rare diseases in comparison with other illnesses. We concluded that SSc patients incur considerable societal costs and experience substantial deterioration in HRQOL.


PubMed | Institute of Rare Diseases Research IIER, Corvinus University of Budapest, Instituto Superiore Of Sanita Iss, Instituto Of Ricerche Farmacologiche Mario Negri and 9 more.
Type: | Journal: The European journal of health economics : HEPAC : health economics in prevention and care | Year: 2016

The aim of this study was to determine the economic burden from a societal perspective and health-related quality of life (HRQOL) of patients with Prader-Willi syndrome (PWS) in Europe.We conducted a cross-sectional study of patients with PWS from Spain, Bulgaria, Hungary, Germany, Italy, the UK, Sweden and France. Data on demographic characteristics, healthcare resource utilisation, informal care, labour productivity losses and HRQOL were collected from questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire.A total of 261 patients completed the questionnaire. The average annual costs ranged from 3937 to 67,484 between countries; the reference year for unit prices was 2012. Direct healthcare costs ranged from 311 to 18,760, direct non-healthcare costs ranged from 1269 to 44,035, and loss of labour productivity ranged from 0 to 2255. Costs were also shown to differ between children and adults. The mean EQ-5D index score for adult PWS patients ranged between 0.40 and 0.81 and the mean EQ-5D visual analogue scale score ranged between 51.25 and 90.00.The main strengths of this study lie in our bottom-up approach to costing and in the evaluation of PWS patients from a broad societal perspective. This type of analysis is very scarce in the international literature on rare diseases in comparison with other illnesses. We conclude that PWS patients incur considerable societal costs and experience substantial deterioration in HRQOL.


PubMed | Institute of Rare Diseases Research, Corvinus University of Budapest, URC Eco Ile de France, Instituto Superiore Of Sanita Iss and 8 more.
Type: | Journal: The European journal of health economics : HEPAC : health economics in prevention and care | Year: 2016

Our goal was to provide data on the economic burden and health-related quality of life (HRQOL) of patients with cystic fibrosis (CF) and their caregivers in Europe.A cross-sectional study was carried out on adults and children with CF in eight European countries. Patients completed an anonymous questionnaire regarding their socio-demographic characteristics, use of healthcare services and presence of a caregiver. Costs were calculated with a bottom-up approach using unit costs from each participating country, and HRQOL was assessed using EQ-5D. The principal caregiver also answered a questionnaire on their characteristics, HRQOL and burden.A total of 905 patients with CF was included (399 adults and 506 children). The total average annual cost per patient varied from 21,144 in Bulgaria to 53,256 in Germany. Adults had higher direct healthcare costs than children, but children had much higher informal care costs (P<0.0001). Total costs increased with patients level of dependence. In adults, mean utility fell between 0.640 and 0.870, and the visual analogue scale ranged from 46.0 to 69.7. There was no difference in caregiver HRQOL regardless of whether they cared for an adult or a child. However, caregivers who looked after a child had a significantly higher burden (P=0.0013).Our study highlights the burden of CF in terms of costs and decreased HRQOL for both patients and their caregivers throughout Europe.


PubMed | Institute of Rare Diseases Research, Corvinus University of Budapest, Instituto Superiore Of Sanita Iss, Mario Negri Institute for Pharmacological Research and 8 more.
Type: | Journal: The European journal of health economics : HEPAC : health economics in prevention and care | Year: 2016

The aim of this study was to determine the economic burden from a societal perspective and the health-related quality of life (HRQOL) of patients with haemophilia in Europe.We conducted a cross-sectional study of patients with haemophilia from Bulgaria, France, Germany, Hungary, Italy, Spain Sweden and the UK. Data on demographic characteristics, health resource utilisation, informal care, loss of labour productivity and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire. The costs have been estimated from a societal perspective adopting a bottom-up approach.A total of 401 questionnaires were included in the study, of which 339 were collected from patients with haemophilia and 62 from caregivers. The lowest average annual cost per person was reported in Bulgaria (6,660) and the highest in Germany (194,490). Our results demonstrate both a large difference from country to country in the average annual cost per patient in 2012 and the driving role of drugs in costs. Drugs represent nearly 90% of direct healthcare costs in a majority of the countries analysed (Hungary, Italy, Spain and Germany). In Bulgaria, France and Sweden, however, healthcare services (visits, tests and hospitalisations) prevail. Costs are also shown to differ between children and adults. The mean EQ-5D index score for adult patients was 0.69 and mean EQ-5D VAS was 66.6. The mean EQ-5D index score for carers was 0.87 and mean EQ-5D VAS was 75.5. In the disability score, 60% showed no disability and measuring caregiver burden with the Zarit Index produced an overall mean score of 25.3.We have shown that haemophilia is associated with a substantial economic burden and impaired HRQOL. Studies on cost of illness and HRQOL are important for haemophilia as the future of this disease is likely to change with the development of new innovative treatments. The introduction of these treatments will most likely impact future costs related to haemophilia.


PubMed | Institute of Rare Diseases Research, Corvinus University of Budapest, URC Eco Ile de France, Instituto Superiore Of Sanita Iss and 8 more.
Type: | Journal: The European journal of health economics : HEPAC : health economics in prevention and care | Year: 2016

To estimate the social/economic costs of fragile X syndrome (FXS) in Europe and to assess the health-related quality of life (HRQOL) of patients and caregivers.A cross-sectional study was conducted in a sample of European countries. Patients were recruited through patients associations. Data on their resource use and absence from the labour market were retrospectively obtained from an online questionnaire. Costs were estimated by a bottom-up approach and the EuroQol-5 Domain (EQ-5D) questionnaire was used to measure patients and caregivers HRQOL.Five countries were included in the analysis. The mean annual cost of FXS per patient varied from 4951 in Hungary to 58,862 in Sweden. Direct non-healthcare costs represented the majority of costs in all countries but there were differences in the share incurred by formal and informal care among those costs. Costs were also shown to differ between children and adults. Mean EQ-5D utility score for adult patients varied from 0.52 in France (n = 42) to 0.73 in Hungary (n = 2), while for caregivers this score was consistently inferior to 0.87.Our findings underline that, although its prevalence is low, FXS is costly from a societal perspective. They support the development of tailored policies to reduce the consequences of FXS on both patients and their relatives.


PubMed | Institute of Rare Diseases Research, Corvinus University of Budapest, Hungarian Federation of People with Rare and Congenital Diseases RIROSZ, Instituto Superiore Of Sanita Iss and 10 more.
Type: | Journal: The European journal of health economics : HEPAC : health economics in prevention and care | Year: 2016

To assess the health-related quality of life (HRQOL) of patients with mucopolysaccharidosis (MPS) and their caregivers and to quantify the disease-related costs from a societal perspective.In the context of a multi-country study of rare diseases (BURQOL-RD project), a cross-sectional survey was performed among MPS patients in seven European countries. Data on demographic characteristics, health resource utilization, informal care, and loss of labor productivity were collected. The EQ-5D, Barthel index (BI), and Zarit burden interview (ZBI) questionnaires were used to assess patients and their informal caregivers quality of life, patients functional ability, and caregivers burden, respectively.Altogether, 120 patients (children 62%, females 40%) and 66 caregivers completed the questionnaire. Patients mean age was 16.5years and median age at diagnosis was 3years. Adult patients average EQ-5D and EQ VAS scores varied across countries from 0.13 to 0.43 and 30.0 to 62.2, respectively, mean BI was 46.7, and ZBI was 32.7. Mean informal care time was 51.3h/week. The mean total annual cost per patient (reference year 2012) was 24,520 in Hungary, 25,993 in France, 84,921 in Italy, 94,384 in Spain, and 209,420 in Germany. Costs are also shown to differ between children and adults. Direct costs accounted for most of the costs in all five countries (80, 100, 99, 98, and 93%, respectively).MPS patients experience substantial loss of HRQOL and their families take a remarkable part in their care. Although utilization of health and social care resources varies significantly across countries, MPS incurs considerable societal costs in all the countries studied.

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