The European challenges of funding orphan medicinal products.
ABSTRACT: 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.
Project description:OBJECTIVES:The primary objective of this study was to compare the availability and access of orphan medicinal products (OMPs) in the devolved nations in the United Kingdom (UK), France, Germany, Italy and Spain. Availability is defined as the possibility to prescribe OMPs. Access refers to their full or partial reimbursement by the public health service. METHODS:Data were collated on: marketing authorisations, Health Technology Assessment (HTA) decisions, commissioning, and reimbursement decisions, and respective dates of these events for all the OMPs centrally authorised. Indicators of availability of and access to OMPs were calculated in each country and compared. RESULTS:We found that since the implementation of the OMPs Regulation in 2000 to end of May 2016, 143 OMPs obtained a marketing authorisation in the European Union. These OMPs are most widely accessible in Germany and France. In the other countries between 30 and 60% of OMPs are reimbursed. In particular in England, less than 50% of centrally authorised OMPs are routinely funded by the NHS, with one-third of these recommended by NICE. In Germany reimbursement is automatically granted to all medicines which receive a marketing authorisation, immediately after authorisation - but since 2011, there is an evaluation and potentially a pricing negotiation between companies and sickness funds (third party payers). In the other countries, the shortest time from authorisation to a reimbursement decision is observed in Italy and France where it takes 18.6 and 19.5 months respectively on average. CONCLUSIONS:Marketing authorisation granted to OMPs is only the first step, as medicines reach patients when reimbursement decisions are implemented by national health systems (this applies to non-OMPs too). We found that more than a half of centrally authorised OMPs were available in the five selected countries, but that access to patients was further restricted by different national reimbursement policies, especially in the UK, Italy and Spain.
Project description:BACKGROUND:Public health care payer organizations face increasing pressures to make transparent and sustainable coverage decisions about ever more expensive prescription drugs, suggesting a need for public engagement in coverage decisions. However, little is known about countries' approaches to integrating public preferences in existing funding decisions. The aim of this study was to describe how Belgium and New Zealand used deliberative processes to engage the public and to identify lessons learned from these countries' approaches. METHODS:To describe two countries' deliberative processes, we first reviewed key country policy documents and then conducted semi-structured interviews with five leaders of the processes from Belgium and New Zealand. We assessed each country's rationales for and approaches to engaging the public in pharmaceutical coverage decisions and identified lessons learned. We used qualitative content analysis of the interviews to describe key themes and subthemes. RESULTS:In both countries, the national public payer organization initiated and led the process of integrating public preferences into national coverage decision making. Reimbursement criteria considered outdated and changing societal expectations prompted the change. Both countries chose a deliberative process of public engagement with a multi-year commitment of many stakeholders to develop new reimbursement processes. Both countries' new reimbursement processes put a stronger emphasis on quality of life, the separation of individual versus societal perspectives, and the importance of final reimbursement decisions being taken in context rather than based largely on cost-effectiveness thresholds. CONCLUSIONS:To face the growing financial pressure of sustainable funding of medicines, Belgium's and New Zealand's public payers have developed processes to engage the public in defining the reimbursement system's priorities. Although these countries differ in context and geographic location, they came up with overlapping lessons learnt which include the need for 1) political commitment to initiate change, 2) broad involvement of all stakeholders, and 3) commitment of all to engage in a long-term process. To evaluate these changes, further research is required to understand how coverage decisions in systems with and without public engagement differ.
Project description:BACKGROUND: In view of the current economic crisis and the resulting austerity measures being implemented by governments across Europe, public expenditure for substance abuse treatment has increasingly become a subject of discussion. An EU cross-country comparison would allow an estimation of the total amount of public resources spent on substance abuse treatment, compare various substance abuse treatment funding options, and evaluate the division of expenditures between alcohol and illegal drugs. The purpose of this study is to estimate the public spending of EU countries for alcohol and illegal drug abuse treatment in hospitals. METHODS: Our study uses a uniform methodology in order to enable valid cross-national comparisons. Our data are drawn from the Eurostat database, which provides anno 2010 data on government spending for the treatment of illegal drug and alcohol abuse in 21 EU member states. The cross-country comparison is restricted to hospitals, since data were unavailable for other types of treatment providers. The systematic registration of in- and outpatient data is essential to monitoring public expenditures on substance abuse treatment using international databases. RESULTS: Total public spending for hospital-based treatment of illegal drug and alcohol abuse in the 21 EU member states studied is estimated to be 7.6 billion euros. Per capita expenditures for treatment of illegal drug abuse vary, ranging from 0.1 euros in Romania to 13 euros in Sweden. For alcohol abuse, that figure varied from 0.9 euros in Bulgaria to 24 euros in Austria. These results confirm other studies indicating that public expenditures for alcohol treatment exceed that for illegal drug treatment. CONCLUSIONS: Multiple factors may influence the number of hospital days for alcohol or illegal substance abuse treatment, and expenditures fluctuate accordingly. In this respect, we found a strong correlation between gross domestic product (GDP) per capita and public expenditures per hospital day. The prevalence of problematic (illegal or legal) drug use in a country did not correlate significantly with the number of hospital days. Other factors must be included in the analysis of public expenditures for the treatment of substance abuse, such as the drug policy in a given country and the social norms regarding alcohol consumption.
Project description:In this study, we analyzed healthcare provision and health expenditure across six Mediterranean countries that adopt the National Health System (Beveridge model) and that form part of the European Union (EU) with the main aim being that of analyzing and comparing out-of-pocket health spending in countries with a European Mediterranean connection. To this end, we considered various economic indicators and statistics to derive commonalities and differences across these countries and also compared trends in these indicators to those observed across the rest of the EU. We then analyzed these findings in light of other data related to the quality of healthcare delivery and the infrastructure of the health system and discussed recent developments in healthcare within each country and the main challenges faced by the respective health systems. The results show that on average, Mediterranean countries spend less on total healthcare expenditure (THE) than the EU average, both as a proportion of GDP, as well as in per capita terms. This is primarily driven by lower-than-EU-average public funding of healthcare. The 2008/2009 macro-economic and financial crisis had a significant impact on the countries under review, and explains the persistent reductions in public health spending as part of the austerity measures put in force across sectors. On the flipside, Mediterranean countries have a higher presence of private health providers in total funding, thereby explaining the higher Out-of-Pocket (OOPs) health expenditures in these countries relative to the EU-average. With regard to the overall health infrastructure in these countries, we observed that although the supply of physicians is largely in line with the rest of the EU, there is under-supply when it comes to hospital beds. This may be symptomatic of lower government funding. Nonetheless, all countries score highly in the evaluation of the quality of health services, as recorded by international rankings like the WHO's 2000 metric, whereas health system performance indicators, namely mortality rates and life expectancy reveal favorable health outcomes in the Mediterranean EU countries. The findings in this paper may be seen in light of the Mediterranean region's lifestyle in terms of diet, health behavior, health beliefs and shared culture. In particular, the higher out-of-pocket expenditure may reflect the tendency for one-to-one relationships with private clinicians and the pursuit of person-centered care (1). Additionally, the Mediterranean people may not be as disciplined as their European counterparts in accessing and using the public health sector. The lower THE also reflects the fact that the Mediterranean countries are less wealthy than the more economically-advanced European countries.
Project description:HIGHLIGHTS Since the geopolitical developments of 1989, former centrally planned economies of Eastern Europe followed distinctively different pathways in national pharmaceutical expenditure evolution as compared to their free market Western European counterparts.Long term spending on pharmaceuticals expressed as percentage of total health expenditure was falling in free market economies as of 1989. Back in early 1990s it was at higher levels in transitional Eastern European countries and actually continued to grow further.Public financing share of total pharmaceutical expenditure was steadily falling in most Central and Eastern European countries over the recent few decades. Opposed scenario were EU-15 countries which successfully increased their public funding of prescription medicines for the sake of their citizens.Pace of annual increase in per capita spending on medicines in PPP terms, was at least 20% faster in Eastern Europe compared to their Western counterparts. During the same years, CEE region was expanding their pharmaceuticals share of health spending in eight fold faster annual rate compared to the EU 15.Private and out-of-pocket expenditure became dominant in former socialist countries. Affordability issues coupled with growing income inequality in transitional economies will present a serious challenge to equitable provision and sustainable financing of pharmaceuticals in the long run.
Project description:The Central European Cooperative Oncology Group (CECOG) and 'ESMO Open-Cancer Horizons' roundtable discussion brought together stakeholders from several European Union (EU) countries involved in drug development, drug authorisation and reimbursement or otherwise affected by delayed and unequal access to innovative anticancer drugs. The approval process of drugs is well established and access delays can be caused directly or indirectly by national or regional decision-making processes on reimbursement. The two key aspects for those involved in reimbursement decisions are first the level of evidence required to decide and second pricing, which can be challenging for some innovative oncology compounds, especially in Eastern and South-Eastern European countries. Other important factors include: available healthcare budget; the structure and sophistication of healthcare authorities and health technology assessment processes; societal context and political will. From the point of view of the pharmaceutical industry, better alignment between stakeholders in the process and adaptive pathway initiatives is desirable. Key aspects for patients are improved access to clinical trials, preapproval availability and reports on real-world evidence. Restricted access limits oncologists' daily work in Eastern and South-Eastern EU countries. The roundtable discussion suggested considering the sequencing of regulatory approval and reimbursement decisions together with more flexible contracting as a possible way forward. The panel concluded that early and regular dialogue between all stakeholders including regulators, payers, patient stakeholders and industry is required to improve the situation.
Project description:Increasing health costs in developed countries are a major concern for decision makers. A variety of cost containment tools are used to control this trend, including maximum price regulation and reimbursement methods for health technologies. Information regarding expenditure-related outcomes of these tools is not available.To evaluate the association between different cost-regulating mechanisms and national health expenditures in selected countries.Price-regulating and reimbursement mechanisms for prescription drugs among OECD countries were reviewed. National health expenditure indices for 2008-2012 were extracted from OECD statistical sources. Possible associations between characteristics of different systems for regulation of drug prices and reimbursement and health expenditures were examined.In most countries, reimbursement mechanisms are part of publicly financed plans. Maximum price regulation is composed of reference-pricing, either of the same drug in other countries, or of therapeutic alternatives within the country, as well as value-based pricing (VBP). No association was found between price regulation or reimbursement mechanisms and healthcare costs. However, VBP may present a more effective mechanism, leading to reduced costs in the long term.Maximum price and reimbursement mechanism regulations were not found to be associated with cost containment of national health expenditures. VBP may have the potential to do so over the long term.
Project description:We analysed the Horizon 2020 project database, currently the European Union’s (EU) largest framework programme for research and innovation—nearly 80 billion euros available over 7 years (2014–2020), to estimate the amount and type of EU-supported biomedical and health research and funding distribution among EU member states and non-European countries. Out of 20,877 projects as of 14th January 2019, a total of 4865 projects were classified as human health related. Ninety-four countries/territories worldwide participated in at least one biomedical project. The EU-15 original member states showed the highest participation as project leaders/partners and for acquired funding. Strong unequal funding distribution and participation between EU-15 and the 13 newest members—with EU-15 receiving about 87% of funding and EU-13 only 3%—have been evidenced. For both EU-15 and EU-13 we detected about 20% of projects involving the public and private sectors, according to Horizon 2020 guidelines. The largest percentage of projects was in the areas of biotechnological research (28.28%) and “basic research” (26.95%); these two sectors together accounted for 46.99% of the total funding assigned (7.9 billion euros). Research in neurosciences and neurological diseases appeared to be an increasing study area. Neurological and mental diseases covered about 21% of projects. Epidemiological studies accounted for about 5% of the total projects and for 14% of funding. Strong correlations were shown by indicators of financial and scientific capacity to identify success rates in obtaining EU funding, making the gap between countries with strong and weak research infrastructures difficult to overcome. Electronic supplementary material The online version of this article (10.1007/s10654-020-00690-9) contains supplementary material, which is available to authorized users.
Project description:The sustainability of long-term care (LTC) is a prominent policy priority in many Western countries. LTC is one of the most pressing fiscal issues for the growing population of elderly people in the European Union (EU) Member States. Country recommendations regarding LTC are prominent under the EU's European Semester.This paper examines challenges related to the financial- and organizational sustainability of LTC systems in the EU. We combined a targeted literature review and a descriptive selected country analysis of: (1) public- and private funding; (2) informal care and externalities; and (3) the possible role of technology in increasing productivity. Countries were selected via purposive sampling to establish a cohort of country cases covering the spectrum of differences in LTC systems: public spending, private funding, informal care use, informal care support, and cash benefits.The aging of the population, the increasing gap between availability of informal care and demand for LTC, substantial market failures of private funding for LTC, and fiscal imbalances in some countries, have led to structural reforms and enduring pressures for LTC policy-makers across the EU. Our exploration of national policies illustrates different solutions that attempt to promote fairness while stimulating efficient delivery of services. Important steps must be taken to address the sustainability of LTC. First, countries should look deeper into the possibilities of complementing public- and private funding, as well as at addressing market failures of private funding. Second, informal care externalities with spill-over into neighboring policy areas, the labor force, and formal LTC workers, should be properly addressed. Thirdly, innovations in LTC services should be stimulated to increase productivity through technology and process innovations, and to reduce costs.The analysis shows why it is difficult for EU Member State governments to meet all their goals for sustainable LTC, given the demographic- and fiscal circumstances, and the complexities of LTC systems. It also shows the usefulness to learn from policy design and implementation of LTC policy in other countries, within and outside the EU. Researchers can contribute by studying conditions, under which the strategies explored might deliver solutions for policy-makers.
Project description:"Labelled drug-related public expenditure" is the direct expenditure explicitly labelled as related to illicit drugs by the general government of the state. As part of the reporting exercise corresponding to 2005, the European Monitoring Centre for Drugs and Drug Addiction's network of national focal points set up in the 27 European Union (EU) Member States, Norway, and the candidates countries to the EU, were requested to identify labelled drug-related public expenditure, at the country level. This was reported by 10 countries categorised according to the functions of government, amounting to a total of EUR 2.17 billion. Overall, the highest proportion of this total came within the government functions of Health (66%), and Public Order and Safety (POS) (20%). By country, the average share of GDP was 0.023% for Health, and 0.013% for POS. However, these shares varied considerably across countries, ranging from 0.00033% in Slovakia, up to 0.053% of GDP in Ireland in the case of Health, and from 0.003% in Portugal, to 0.02% in the UK, in the case of POS; almost a 161-fold difference between the highest and the lowest countries for Health, and a 6-fold difference for POS. Why do Ireland and the UK spend so much in Health and POS, or Slovakia and Portugal so little, in GDP terms? To respond to this question and to make a comprehensive assessment of drug-related public expenditure across countries, this study compared Health and POS spending and GDP in the 10 reporting countries. Results found suggest GDP to be a major determinant of the Health and POS drug-related public expenditures of a country. Labelled drug-related public expenditure showed a positive association with the GDP across the countries considered: r = 0.81 in the case of Health, and r = 0.91 for POS. The percentage change in Health and POS expenditures due to a one percent increase in GDP (the income elasticity of demand) was estimated to be 1.78% and 1.23% respectively. Being highly income elastic, Health and POS expenditures can be considered luxury goods; as a nation becomes wealthier it openly spends proportionately more on drug-related health and public order and safety interventions.