Budgetary Impact of Medicinal Therapies for Rare Diseases in Bulgaria.

BACKGROUND: Rare diseases have been continually outlined as one of the causes for the National Health Insurance Fund's (NHIF) deficit spending in Bulgaria. AIM: To estimate the budgetary impact of rare disease medicinal therapies from NHIF perspective for 2014 and 2016. MATERIALS AND METHODS: Budgetary impact of rare diseases is calculated as a percentage of NHIF total pharmaceutical spending. Total expenditure per ICD-10 code, mean annual number of patients reimbursed and mean annual cost per patient are analysed. RESULTS: Budgetary impact of rare diseases reached a plateau of about 9% of NHIF total pharmaceutical spending for 2014-2016. Mean number of patients reimbursed and mean annual cost per patient increased by median rates of 4.27% and 2.54%, respectively. Glycogen storage disease, neuropathic heredofamilial amyloidosis and C1 esterase inhibitor deficiency stood out, as they had the second, fourth and fifth most expensive medicinal treatment cost. While accounting for only 92 patients in 2016, these three conditions contributed for 22.89% of NHIF total expenditure on rare disease medicinal therapies. For comparison, coagulation defects, with the biggest total cost per indication, had a similar budgetary impact - 24.88%, but for 277 patients reimbursed. CONCLUSIONS: Our study does not support the concerns about uncontrolled growth of expenditures for rare disease medicinal therapies. Nevertheless, there is a need for enhanced post-marketing surveillance and performance-based payment of these treatments. Development, collection and analysis of local real-world data have been increasingly applied as a tool to advance these health policy goals.

Economic Burden And Health-Related Quality Of Life Of Patients With Cystic Fibrosis In Bulgaria.

OBJECTIVE: 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 cystic fibrosis (CF) in Bulgaria. MATERIALS AND METHODS: We conducted a cross-sectional study of 33 patients with CF and 17 caregivers from Bulgaria. Data on socio-demographic characteristics, health resource utilisation, informal care, labor productivity losses and HRQOL were collected from questionnaires completed by patients or their caregivers. HRQOL was evaluated with the EuroQol 5-domain (EQ-5D-3L) questionnaire. RESULTS: Median annual costs of CF in Bulgaria were € 24 152 per patient in 2012 as a reference year. Median annual costs for children were found to be significantly higher than those for adults - € 31 945 vs. € 15 714 (p = 0.012). This outcome came from statistically significant differences in costs for main informal carer (p < 0.001) and costs for other informal carers (p = 0.022). As a single cost item, drugs had the biggest monetary impact. Median annual drug costs were € 13 059. Bulgarian CF patients showed low HRQOL results - 50 median VAS score and 0.592 median health utilities. A quarter of patients even rated their health state as worse than death. CONCLUSION: CF patients from Eastern Europe remain a vulnerable population with risk factors for worse health outcomes. Our study provided a state-of-the art analysis that facilitates the elaboration, adoption and application of targeted public health policies to tackle CF-related problems at national and European level.

Insight into reimbursement decision-making criteria in Bulgaria: implications for orphan drugs.

OBJECTIVE: This article's objective is to critically assess the Bulgarian legislation on health technology assessment (HTA). It analyses how innovative therapies and orphan drugs in particular would respond to the regulators' decision-making criteria for reimbursement. MATERIALS AND METHODS: The study features critical analysis of current decision-making criteria for drug reimbursement in Bulgaria, as well as hypothetical scenario planning for orphan medicinal products. RESULTS: The approval for inclusion into the Positive Drug List (PDL) (which is a must for reimbursement) has been reorganised into an assessment scoring system with decision-making criteria (presence of therapeutic alternative, clinical effectiveness, safety, pharmacoeconomics and societal value) divided into weighted indicators. An explicit threshold has been set--a medicinal product must score 60 points at least to be included in PDL. Under the currently defined reimbursement decision-making criteria a hypothetical middle-of-the-road scenario planning shows that an orphan drug would score 20 points for therapeutic alternative, 28 for clinical effectiveness and 12 for safety. It would take no points for pharmacoeconomics and societal value. This leaves the orphan drugs with a total score of 60 points, making the final outcome of real-life assessment and decision-making heavily dependent on small fluctuations. CONCLUSIONS: The current reimbursement decision-making framework in Bulgaria seems to be generalised and not sufficiently transparent. It is unable to precisely assess innovative health technologies. The availability of a therapeutic alternative emerges as a key reimbursement decision-making criterion for orphan drugs, as these innovative products nominally provide the first medicinal therapy alternative to rare diseases.