BACKGROUND: A core evaluation framework that captures the health care and societal benefits of value added medicines (VAMs, also often called repurposed medicines) was proposed in Report 1, aiming to reduce the heterogeneity in value assessment processes across countries and to create incentives for manufacturers to invest into incremental innovation. However, this can be impactful only if the framework can be adapted to heterogeneous health care financing systems in different jurisdictions, and the cost of evidence generation necessitated by the framework takes into account the anticipated benefits for the health care system and rewards for the developers. AREAS COVERED: The framework could potentially improve the pricing and reimbursement decisions of VAMs by adapting it to different country specific decision-contexts such as deliberative processes, augmented cost-effectiveness frameworks or formal multi-criteria decision analysis (MCDA); alternatively, some of its domains may be added to current general evaluation frameworks of medicines. The proposed evaluation framework may provide a starting point for practices based on which VAMs can be exempted from generic pricing mechanisms or can be integrated into the reimbursement and procurement system, allowing for price differentiation according to their added value. Besides evidence from RCTs, pricing and reimbursement decision processes of VAMs should allow for ex-ante non-RCT evidence for certain domains. Alternatively, relying on ex-post evidence agreements-such as outcome guarantee or coverage with evidence development-can also reduce decision uncertainty. CONCLUSIONS: The core evaluation framework for VAMs could trigger changes in the existing pricing, reimbursement and procurement practices by improving the appraisal of the added value created by incremental innovation.

• Klíčová slova
• Drug repurposing, Evidence, Generic reference pricing, Incremental innovation, Multi-criteria decision analysis, Value assessment framework, Value-added medicines,
• Publikační typ
• časopisecké články MeSH
• přehledy MeSH

Objectives: To analyse the market shares of biosimilars in Slovakia and to calculate the potential cost-savings from the use of biosimilars in Slovakia based on two different data sources. Methods: National reimbursement lists from the Czech Republic, Hungary, Poland and Slovakia were used for analyzing the availability of biosimilars with public funding. In addition, the reimbursement dossiers of biosimilars, the justifications of reimbursement decisions by the Slovak Ministry of Health, and final reimbursement decrees, which are published on the webpage of the Slovak Ministry of Health, were utilized for this study. Reimbursement decisions regarding biosimilars by the Slovak Ministry of Health from 2006 to August 2019 were considered and the detailed utilization of biosimilars in 2018 was analyzed based on data from the State Institute for Drug Control. The study was validated based on data from the Slovak National Health Information Center. Results: Fifty four biosimilars were approved by the European Medicines Agency (EMA) in August 2019. Of the total group of licensed biosimilars on the market, 29 biosimilars (54%) were available in the Czech Republic, 28 biosimilars (52%) were available in Poland, and 27 biosimilars (50%) were available in Hungary and 24 biosimilars (44%) were available in Slovakia. Our analysis, based on the data provided by distributors of medicinal products to the State Institute for Drug Control, revealed that the health fund in Slovakia could have saved 35 to 50 million euros per year if biosimilars with marketing authorisations had been available on the Slovak market. The calculations assumed a 25-35% price decrease against the original biological medical products, and that there would be no increase in the utilization of biosimilars in Slovakia. Conclusions: To achieve significant improvement in patient access to biosimilars in Slovakia, a top-down approach establishing targets and quotas for the procurement of biosimilars should be applied.

• Klíčová slova
• Slovakia, biomedical, decision making, health, health policy, insurance, reimbursement, savings,
• MeSH
• biosimilární léčivé přípravky * terapeutické užití   MeSH
• lidé MeSH
• Check Tag
• lidé MeSH
• Publikační typ
• časopisecké články MeSH
• Geografické názvy
• Česká republika MeSH
• Maďarsko MeSH
• Polsko MeSH
• Slovenská republika MeSH
• Názvy látek
• biosimilární léčivé přípravky * MeSH

INTRODUCTION: Although there is a significant utilization gap of biologic medicines in the EU, many studies estimate equity in patient access to biopharmaceuticals only based on their availability on the national list of reimbursed medicines. Hidden access barriers may facilitate financial sustainability of pharmaceuticals in less affluent EU countries; however, they have rarely been documented in scientific publications. Our objective was to explore these access barriers for tumor necrosis factor (TNF) alpha inhibitors in rheumatoid arthritis (RA) in five Central and Eastern European countries. METHODS: A detailed interview guide was developed based on multi-stakeholder workshops and a targeted literature review. In each participant country 3-3-3-3 interviews with payers, rheumatologists, patients/patient representatives, and industry representatives were conducted. Responses were aggregated at a country level and validated by primary investigators in each country. RESULTS: Limited number of RA centers and consequently significant travelling time and cost for patients in distant geographical areas, uneven budget allocation among centers, limited capacity of nurses, narrowed patient population in national financial protocols compared to international clinical guidelines in initiating or continuing biologics, high administrative burden in prescribing biologics and limited health literacy of patients were the most relevant barriers to timely patient access in at least three participant countries. CONCLUSION: Assessing only the availability of TNF alpha inhibitors on the national list of reimbursed medicines provides limited information about real-world patient access to these medicines. Revealing hidden access barriers may contribute to initiate policy actions which could reduce inequity in patient access.

• Klíčová slova
• Central Eastern Europe, TNF alpha inhibitor, access barriers, biologic, patient access, pharmaceutical policy, rheumatoid arthritis,
• Publikační typ
• časopisecké články MeSH

BACKGROUND: Funding of orphan medicinal products (OMPs) is an increasing challenge in the European Union (EU). OBJECTIVES: To identify the different methods for public funding of OMPs in order to map the availability for rare disease patients, as well as to compare the public expenditures on OMPs in 8 EU member states. METHODS: Information on the reimbursement status of 83 OMPs was collected in 8 countries by distinguishing standard and special reimbursements. In two consecutive years, the total public expenditures on OMPs were calculated by using annual EUR exchange rates. Annual total public expenditures were calculated per capita, and as a proportion of GDP, total public pharmaceutical and healthcare budgets. Differences between countries were compared by calculating the deviations from the average spending of countries. RESULTS: In 2015 29.4-92.8% of the 83 OMPs were available with any kind of public reimbursement in participant countries including special reimbursement on an individual basis. In Austria, Belgium and France more OMPs were accessible for patients with public reimbursement than in Bulgaria, Czech Republic, Hungary and Poland. Standard reimbursement through retail pharmacies and/or hospitals was applied from 0 to 41% of OMPs. The average annual total public expenditure ranged between 1.4-23.5 €/capita in 2013 and 2014. Higher income countries spent more OMPs in absolute terms. Participant countries spent 0.018-0.066% of their GDPs on funding OMPs. Average expenditures on OMPs were ranged between 2.25-6.51% of the public pharmaceutical budget, and 0.44-0.96% of public healthcare expenditures. CONCLUSIONS: Standard and special reimbursement techniques play different roles in participant countries. The number of accessible OMPs indicated an equity gap between Eastern and Western Europe. The spending on OMPs as a proportion of GDP, public pharmaceutical and healthcare expenditure was not higher in lower income countries, which indicates substantial differences in patient access to OMPs in favour of higher-income countries. Equity in access for patients with rare diseases is an important policy objective in each member state of the EU; however, equity in access should be harmonized at the European level.

• Klíčová slova
• Equity, European Union, Funding, Orphan medicinal products, Patient access, Reimbursement,
• MeSH
• Evropská unie MeSH
• lidé MeSH
• náklady na léky * MeSH
• výdaje na zdravotnictví MeSH
• výroba orphan drugs ekonomika   MeSH
• vzácné nemoci MeSH
• Check Tag
• lidé MeSH
• Publikační typ
• časopisecké články MeSH
• Geografické názvy
• Evropa MeSH

This policy research aims to map patient access barriers to biologic treatments, to explore how increased uptake of biosimilars may lower these hurdles and to identify factors limiting the increased utilisation of biosimilars. A policy survey was developed to review these questions in 10 Central and Eastern European (CEE) and Commonwealth of Independent States (CIS) countries. Two experts (one public and one private sector representative) from each country completed the survey. Questions were related to patient access, purchasing, clinical practice, and real-world data collection on both original biologics and biosimilars. Restrictions on the number of patients that can be treated and related waiting lists were reported as key patient access barriers. According to respondents, for both clinicians and payers the primary benefit of switching patients to biosimilars would be to treat more patients. However, concerns with therapeutic equivalence and fear of immunogenicity may reduce utilisation of biosimilars. Similar limitations in patient access to both original biologics and biosimilars raise concerns about the appropriateness and success of current biosimilar policies in CEE and CIS countries. The conceptual framework for additional real-world data collection exists in all countries which may provide a basis for future risk-management activities including vigorous pharmacovigilance data collection.

• MeSH
• biosimilární léčivé přípravky škodlivé účinky   terapeutické užití   MeSH
• farmakovigilance MeSH
• lidé MeSH
• průzkumy a dotazníky MeSH
• Společenství nezávislých států epidemiologie   MeSH
• terapeutická ekvivalence * MeSH
• Check Tag
• lidé MeSH
• Publikační typ
• časopisecké články MeSH
• Geografické názvy
• Společenství nezávislých států epidemiologie   MeSH
• východní Evropa epidemiologie   MeSH
• Názvy látek
• biosimilární léčivé přípravky MeSH