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Objectives: The aim of the study was to compare the access of patients with rare diseases (RDs) to biotechnological drugs in several Central and Eastern European countries (CEECs). We focused on the legislative pricing and reimbursement requirements, availability of biotechnological orphan medicinal products (BOMPs) for RDs, and reimbursement expenditures. Methods: A questionnaire-based survey was conducted among experts from 10 CEECs: Bulgaria, Croatia, Estonia, Greece, Hungary, Poland, Romania, Slovakia, Serbia, and Macedonia. The legal requirements for reimbursement and pricing of BOMPs were collected. All BOMPs and medicines without prior orphan designations were extracted from the European list of orphan medicinal products, 2017. The reimbursement status of these medicinal products in 2017 in the public coverage of the included CEECs as well as the share of their costs in relation to the total public pharmaceutical spending for the period from 2014 to 2016 were defined. Results: Our survey revealed that some differences in the legal requirements for pricing and reimbursement of BOMPs amongst the countries included in the study. All European Union countries have developed and implemented pharmacoeconomic guidelines with or without some specific reimbursement requirements for orphan medicinal products. Cost-effectiveness analysis, cost-utility analysis, Markov models, meta-analysis, and discount levels of costs and results were required only in Bulgaria, Poland and Hungary. The number of reimbursed BOMPs and biotechnological medicinal products for RDs without prior orphan designation was the highest in Hungary (17 and 40, respectively). Patient-based reimbursement schemes were available only in Hungary for 11 out of 17 BOMPs. Poland and Greece have the highest pharmaceutical expenditure of reimbursed BOMPs with are similar to 214 million and 180 million EUR, respectively in the observed period from 2014 to 2016. High proportion of the pharmaceutical expenditure o

The aim of this study was to explore the cost-effectiveness of short protocols including different types of gonadotropins for controlled ovarian hyperstimulation (COH) for in vitro fertilization (IVF). A retrospective, observational study of the real-life practice in a specialized IVF gynaecology clinic in 2009-2013 was carried out. All women on short COH protocols were recruited into three groups: COH including recombinant follicle stimulating hormone (rFSH) and urinary-FSH (urFSH) (n = 173); including urFSH alone (n = 289); and including rFSH alone (n = 212). The cost-effectiveness of the COH protocols was explored in two different case scenarios for possible outcomes. The first case scenario took into consideration a successful live birth, and the second one, the women to achieve pregnancy with live birth. Decision modelling was done using the TreeAge 2014 Software. According to the results in the first case scenario, the rFSH plus urFSH COH approach showed the highest weighted probability (p = 0.38) of live birth, but the urFSH alternative was cost-effective. The results in the second case scenario demonstrated that the urFSH protocol was again the cost-effective alternative. These results suggest that the strategy with urFSH should be preferred in both cases, but rFSH could also be considered as a cost-effective alternative for successful live birth and achieving pregnancy and delivery, since the incremental cost-effectiveness ratio in comparison with the urFSH protocol is below the gross domestic product per capita. The combined approach of rFSH and urFSH was shown not to be cost-effective in both explored scenarios.

The aim of the present study was to analyze the cost of pharmacotherapy and quality of life of patients with cystic fibrosis (CF), Gaucher disease (GD) and chronic myeloid leukemia (CML) in Bulgaria. Data for the pharmacotherapy cost were compiled retrospectively. The second part of the study included a prospective quality-of-life survey (conducted at the Medical University Hospital "Alexandrovska" in 2010 and 2011). We estimated the average monthly pharmacotherapy costs according to data from the hospital registries. The quality-of-life survey was done through interviews based on the short form of SF-36, which measures 9 health domains. The total number of patients was 18, of which 10 were with CML, 5 with cystic fibrosis and 3 with Gaucher disease. The average age of the male and female patients with CML was 49.1 years and 48.3 years, respectively; 25 and 24 years in the group with GD, respectively; and 29.5 and 35.3 years in CF group, respectively. No statistical difference between the average monthly pharmacotherapy costs for the observed period was revealed In all patients the quality of life was below the maximum possible value, which is equal to 100 points. The lowest values for the different indicators of quality of life were observed in the CF patients, most likely due to the chronic progression of this particular rare disease. The average range of quality of life for this group was 53.75. We found a statistical correlation between the pharmacotherapy costs and the quality of life. The Spearman correlation analysis revealed a correlation between the drug therapy costs and some quality-of-life indicators only in the patients with CIVIL. The correlation between the pharmacotherapy costs and physiological functions in patients with CIVIL was positive, i.e. higher costs improved this indicator The pharmacotherapy costs were high, the average values for the three groups of patients being: BGN 9221.61 for 2010 and BGN 8254.64 for January March 2011 for patients w

The aim of the present study was to analyze the pharmacotherapy cost and quality of life of patents after kidney transplantation compared to those with chronic kidney disease in Bulgaria. This retrospective pharmacotherapy cost study and prospective health related quality of life (QoL) study was performed during 2010 - 2011 at the biggest Sofia hospital serving all transplant patients. The cost of pharmacotherapy was analyzed after reviewing patients' records. The health related quality of life was evaluated with the multi-attributable questionnaire SF-36, which measures 9 health domains. Forty-two percent of the patients with chronic kidney disease, and 26% of those with kidney transplant were female. The average age of the transplanted patients was 39.7 for male patients and 42.2 for female ones, while in the group suffering from chronic kidney disease the average male age was 48.3 and that of female patients, 51.2. No statistically significant difference was observed among the mean monthly cost of therapy per patient during the two-year period. The QoL was with lower values in the role limitations domain for patients with chronic kidney diseases and this difference was statistically significant. We found a correlation among the mean cost of pharmacotherapy and mean QoL in both groups of patients. In the group of patients with kidney transplantation the place of living and general health state were positively correlated. The cost of pharmacotherapy and the health state in comparison with the previous year were also positively correlated. This is the first Bulgarian study of the quality of life of kidney transplant patients. It shows that the SF-36 could be successfully used in this group of patients as well as for comparison with the QoL of patients with chronic kidney disease.