Background Reimbursement policies influence access of patients to orphan drugs in the European countries. Objectives To provide a comprehensive description of orphan drug reimbursement policies and to assess reimbursement decision-making process in the EU-CEE countries as well as the impact of the type of approval and disease on reimbursement decisions. Methods For each drug, the information regarding conditional approval or approval under exceptional circumstances was obtained from the EMA website. The reimbursement status for analyzed drugs was collected in a questionnaire survey performed in a group of experts in reimbursement policy. The agreement between countries was assessed using the κ coefficient, nominal variables tests were compared using the χ2 test or the Fisher exact test. The impact of the EMA’s conditional approval and approval under exceptional circumstances was assessed using logistic regression and presented as an odds ratio (OR). Results The analysis revealed that most orphan drugs were authorized for the treatment of oncological or metabolic diseases [36 drugs (38%) and 22 drugs (23%), respectively]. The shares of reimbursed orphan drugs varied significantly ( p = 0.0031) from 6.3% in Latvia to 27.4% in Poland. No correlation ( r = 0.02; p = 0.9583) with GDP per capita was observed. The highest agreement in reimbursement decisions was observed between Estonia and Lithuania, and the lowest – between Estonia and Latvia, with kappa of 0.69 and 0.11, respectively. Significant impact of the type of approval and reimbursement status was observed for Czechia, Lithuania and Slovakia where conditional approval and exceptional circumstances negatively influenced reimbursement decision. Type of disease has significant influence on reimbursement decision in 4 out of 10 analyzed countries with significant outweigh of positive decisions for oncological diseases. Conclusion In considered countries specific regulations on reimbursement of orphan drugs are valid but in Lithuania and Romania no formal HTA process was employed; in case of some countries higher ICER values for orphans are used. The share of reimbursed orphan drugs varied significantly across the countries, but it was not associated with GDP per capita.
IntroductionThe importance of health-related quality of life (HRQoL) is increasing and many healthcare authorities recommend the use of measures that account for both mortality and morbidity. This study will determine, for the first time in Romania, value sets for EuroQoL-five-dimensions-3-level (EQ-5D-3L) and EQ-5D-5L questionnaires and their population norms (study 1). It will also compare the HRQoL (measured with EQ-5D-5L) of Roma communities in Romania with that of the general population (study 2).Methods and analysisCross-sectional studies of face-to-face interviews conducted in representative samples of the Romanian general population and Romanian Roma communities. 1614 non-institutionalised adults older than 18 years will be interviewed using a computer-assisted interview for study 1. Participants will complete EQ-5D-3L and 5L, 13 composite time trade-off tasks (cTTO), 7 discrete choice experiment questions (DCE) and sociodemographic questions. For study 2, 606 non-institutionalised self-identified Roma people older than 18 years will be interviewed using a pencil-and-paper interview. Participants will complete EQ-5D-5L and the same sociodemographic questions as for study 1. The 3L value set will be estimated using econometric models and the cTTO data. cTTO and DCE data will be used for the 5L value set. Population norms will be reported by age and gender. The ORs for reporting different levels of problems and the most common health states in the population will be estimated. For study 2, t-tests and analysis of variance will be used to explore differences between groups in HRQoL and for each EQ-5D.Ethics and disseminationEthics approval was given by the National Bioethics Committee of Medicines and Medical Devices Romania and Newcastle University’s Research Ethics Committee. Results will be published in peer-reviewed journals, presented at scientific conferences and on the project’s website. The EQ-5D-5L anonymised datasets will be deposited in a centralised repository. Two public workshops with local authorities, physicians and patients’ associations will be held.
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 ~214 million and 180 million EUR, respectively in the observed period from 2014 to 2016. High proportion of the pharmaceutical expenditure on the reimbursed biotechnological medicinal products for RDs for the observed period 2014–2016 is presented in Bulgaria and Slovakia.Conclusions: The non-European Union CEECs face a significant delay in the legal implementation of pharmacoeconomic guideline for assessment of BOMPs. The access to BOMPs is similar among the observed CEECs and the countries with the best access are Hungary and Greece. The influence of BOMP expenditures on the budget in the individual countries is significant.
Objective: To provide health-related quality of life (HRQoL) data to support health technology assessment (HTA) and reimbursement decisions in Romania, by developing a country-specific value set for the EQ-5D-3L questionnaire. Methods: We used the cTTO method to elicit health state values using a computer-assisted personal interviewing approach. Interviews were standardized following the most recent version of the EQ-VT protocol developed by the EuroQoL Foundation. Thirty EQ-5D-3L health states were randomly assigned to respondents in blocks of three. Econometric modeling was used to estimate values for all 243 states described by the EQ-5D-3L. Results: Data from 1556 non-institutionalized adults aged 18 years and older, selected from a national representative sample, were used to build the value set. All tested models were logically consistent; the final model chosen to generate the value set was an interval regression model. The predicted EQ-5D-3L values ranged from 0.969 to 0.399, and the relative importance of EQ-5D-3L dimensions was in the following order: mobility, pain/discomfort, self-care, anxiety/depression, and usual activities. Conclusions: These results can support reimbursement decisions and allow regional cross-country comparisons between health technologies. This study lays a stepping stone in the development of a health technology assessment process more driven by locally relevant data in Romania.
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