BackgroundIn case of orphan drugs applicability of the standard health technology assessment (HTA) process is limited due to scarcity of good clinical and health economic evidence. Financing these premium priced drugs is more controversial in the Central and Eastern European (CEE) region where the public funding resources are more restricted, and health economic justification should be an even more important aspect of policy decisions than in higher income European countries.ObjectivesTo explore and summarize the recent scientific evidence on value drivers related to the health technology assessment of ODs with a special focus on the perspective of third party payers in CEE countries. The review aims to list all potentially relevant value drivers in the reimbursement process of orphan drugs.MethodsA systematic literature review was performed; PubMed and Scopus databases were systematically searched for relevant publications until April 2015. Extracted data were summarized along key HTA elements.ResultsFrom the 2664 identified publications, 87 contained relevant information on the evaluation criteria of orphan drugs, but only 5 had direct information from the CEE region. The presentation of good clinical evidence seems to play a key role especially since this should be the basis of cost-effectiveness analyses, which have more importance in resource-constrained economies. Due to external price referencing of pharmaceuticals, the relative budget impact of orphan drugs is expected to be higher in CEE than in Western European (WE) countries unless accessibility of patients remains more limited in poorer European regions. Equity principles based on disease prevalence and non-availability of alternative treatment options may increase the price premium, however, societies must have some control on prices and a rationale based on multiple criteria in reimbursement decisions.ConclusionsThe evaluation of orphan medicines should include multiple criteria to appropriately measure the clinical added value of orphan drugs. The search found only a small number of studies coming from CEE, therefore European policies on orphan drugs may be based largely on experiences in WE countries. More research should be done in the future in CEE because financing high-priced orphan drugs involves a greater burden for these countries.Electronic supplementary materialThe online version of this article (doi:10.1186/s13023-016-0455-6) contains supplementary material, which is available to authorized users.

BackgroundFunding of orphan medicinal products (OMPs) is an increasing challenge in the European Union (EU).ObjectivesTo 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.MethodsInformation 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.ResultsIn 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.ConclusionsStandard 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.Electronic supplementary materialThe online version of this article (10.1186/s13023-018-0927-y) contains supplementary material, which is available to authorized users.

A szerzők a ritka betegségben szenvedők érdekeit szem előtt tartva a gyógyszerár-támogatás és ezáltal a gyógyszer-felhasználás racionalizálása, átláthatóbbá, tervezhetőbbé tétele céljából elemezték az árva gyógyszerek fi nanszírozá-sára vonatkozó 2012. évi hazai egészségbiztosítói adatokat. A legtöbb árva gyógyszer 2012-ben egyedi méltányosság keretében lehetett fi nanszírozott kezelés, amely támogatási lehetőség kapcsán számos, meglehetősen súlyos probléma adódott, különös tekintettel a nagy értékű enzimpótló kezelésekre. A rendkívül nagy költségvonzattal bíró árva gyógyszerek fi nanszírozásának átalakítása Magyarországon sürgetővé vált. A megállapítások alapján a szerzők javaslatot fogalmaznak meg az egészségügyi ellátás szakmai, gazdasági és etikai szempontból sajátságos területe, a nemzetközileg egyre nagyobb fi gyelemmel kísért ritka betegségek érintettjeinek gyógyszerellátására. Elsődleges cél, hogy az enzimpótló orphan gyógyszerek hozzáférését megfelelő támogatási formában lehessen biztosítani azon ritka betegségben szenvedőknek, akik számára kielégítetlen terápiás szükségletet jelent az adott kezelés. A szerzők hangsúlyoz-zák, hogy ebben a szakmai, gazdasági, etikai szempontból rendkívül kényes kérdésben elkerülhetetlen az érintett szakemberek (kezelést végző klinikusok, fi nanszírozási szakemberek, betegszervezeti delegáltak) együttműködése.

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