Policies and availability of orphan medicines in outpatient care in 24 European countries
- 347 Downloads
To assess pricing and reimbursement policies specific to orphan medicines and the availability and distribution settings of ten recently authorised medicinal products suitable for outpatient care with orphan status and centralised marketing authorisation in Europe, and whether patients receive these products free of charge or have to pay some or all of the costs themselves.
Web survey to authorities and representatives of third party payers in the Pharmaceutical Pricing and Reimbursement Information (PPRI) network in April 2016.
In most of the 24 countries, special policies were not implemented in the assessment of reimbursement status (22 countries) or in the pricing (20 countries) of orphan medicines. An average of five of the ten recently authorised products per country were available for outpatient care. Products were dispensed from community pharmacies in eight countries and from health care units in five countries. In four countries, both distribution settings were used. When products were dispensed from community pharmacies, patients typically paid some of the price themselves. Products dispensed from health care units were often free of charge for patients.
Most European countries had not implemented pricing and reimbursement policies specific to orphan medicines. The availability of orphan products varied between countries. It is important to discuss whether orphan medicines should be considered as a separate group in the reimbursement regulations in order to secure patient access to these medicines.
KeywordsOrphan medicines Availability Pricing Reimbursement Europe
We are grateful to the PPRI network for their participation and co-operation in this study.
All authors have made a substantial contribution to (1) the conception or design of the work (the acquisition, analysis or interpretation of data for the work), (2) drafting the work or revising it critically for important intellectual content and (3) final approval of the version to be published. All authors agree to be accountable for all aspects of the work in ensuring that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved.
This study was conducted at the School of Pharmacy of the University of Eastern Finland and was granted research funding from the Social Insurance Institution of Finland (Kela).
Compliance with ethical standards
Conflict of interest
The authors (KS, RA, JT) were funded by a research grant from the Social Insurance Institution of Finland (Kela) (grant number: 12/26/2015).
Research involving human participants and/or animals
This article does not contain any studies with human participants or animals performed by any of the authors.
For this type of study, informed consent is not required.
- 1.European Commission Regulation (EC) No. 141/2000. http://ec.europa.eu/health/documents/eudralex/vol-1_en
- 2.Luisetti M, Balfour-Lynn IM, Johnson SR, Miravitlles M, Strange C, Trapnell BC, van Bronswijk H, Vogelmeier C (2012) Perspectives for improving the evaluation and access of therapies for rare lung diseases in Europe. Respir Med 106(6):759–768. https://doi.org/10.1016/j.rmed.2012.02.016 CrossRefPubMedGoogle Scholar
- 3.Global Genes (2015) Rare diseases facts and statictics. Statistics and figures on prevalence of genetic and rare diseases. Available online: https://globalgenes.org/rare-diseases-facts-statistics/
- 5.Kaplan W Priority medicines for Europe and the World 2013 Update. http://www.who.int/medicines/areas/priority_medicines/MasterDocJune28_FINAL_Web.pdf
- 6.Thieren M (2005) World Health Organization Background paper on the concept of universal access. Technical meeting for the development of a framework for universal access to HIV/AIDS prevention, treatment and care in the health sector. WHO, Geneva. Available online: http://www.who.int/hiv/universalaccess2010/UA_definitions_Dec05.pdf
- 7.WHO (2018) WHO collaborating centre for pharmaceutical pricing and reimbursement policies. Preferred terms. Available online: http://whocc.goeg.at/Glossary/PreferredTerms/Access
- 9.European Medicines Agency (EMA) Orphan designation 2017. http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000029.jsp
- 10.European Medicines Agency (EMA). Orphan Medicines Figures 2000–2017. http://www.ema.europa.eu/docs/en_GB/document_library/Other/2015/04/WC500185766.pdf
- 13.Blankart CR, Stargardt T, Schreyögg J (2011) Availability of and access to orphan drugs: an international comparison of pharmaceutical treatments for pulmonary arterial hypertension, Fabry disease, hereditary angioedema and chronic myeloid leukaemia. PharmacoEconomics 29(1):63–82. https://doi.org/10.2165/11539190-000000000-00000 CrossRefPubMedGoogle Scholar
- 15.Menon D, Clark D, Stafinski T (2015) Reimbursement of drugs for rare diseases through the public healthcare system in Canada: where are we now? Health Policy 11(1):15–32Google Scholar
- 17.Saastamoinen LK, Saarelainen L, Autti-Rämö I, Martikainen JE Lääkkeiden ja ravintovalmisteiden käyttö harvinaisten sairauksien hoidossa—Kysely harvinaissairaita hoitaville lääkäreille. Kela (The Social Insurance Institution of Finland), Työpapereita 76/2015. https://helda.helsinki.fi/bitstream/handle/10138/155626/Tyopapereita76.pdf?sequence=10
- 21.Arnold RJG, Bighash L, Bryón Nieto A, Tannus Branco de Araújo G, Gay-Molina JG, Augustovski F (2015) The role of globalization in drug development and access to orphan drugs: orphan drug legislation in the US/EU and in Latin America. F1000 Res 4(57):1–9. https://doi.org/10.12688/f1000research.4268.1 CrossRefGoogle Scholar
- 22.Garau M, Mestre-Ferrandiz J (2009) Access mechanisms for orphan drugs: a comparative study of selected european countries. Office of health economics (OHE) research briefing 52:1-39. Available online: https://papers.ssrn.com/sol3/papers2.cfm?abstract_id=2640099
- 24.Bignami F (2007) EURORDIS rare diseases Europe. EURORDIS survey on orphan drugs availability in Europe. 6th EURORDIS round table of companies workshop. Barcelona. Available online: http://www.eurordis.org/IMG/pdf/2007ODsurvey-eurordis.pdf
- 25.Habl C, Bachner F (2011) EMINET initial investigation to assess the feasibility of a coordinated system to access orphan medicines. The European commission, directorate-general enterprise and industry. Final report Vienna 2011. Available online: http://whocc.goeg.at/Literaturliste/Dokumente/BooksReports/EMINet_Initial%20investigationOMP_updated2011.pdf
- 26.WHO (2015) WHO collaborating centre for pharmaceutical pricing and reimbursement. PPRI: pharmaceutical pricing and reimbursement information. Available online: http://whocc.goeg.at/About/PPRI
- 27.European Medicines Agency (2016) European public assessment reports EPAR. Available online: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/landing/epar_search.jsp&mid=WC0b01ac058001d125
- 28.Finnish Advisory Board on Research Integrity Ethical review in human sciences 2014. http://www.tenk.fi/en/ethical-review-in-human-sciences
- 29.University of Eastern Finland (2017) Research ethics. Available online: https://www.uef.fi/in/research/instructions-and-forms
- 30.Icelandic Medicines Agency (2017) Marketing authorisations (MA). Available online: https://www.ima.is/licences/marketing_authorisations/nr/2881
- 31.Ministry of Health Care Servives (2018) Marketing authorisation of medicinal products. Available online: https://www.regjeringen.no/en/topics/health-and-care/Pharmaceutical-products/innsikt/product-approval/id434890/
- 33.Sorainen & National Agency of Investment and Privatization (2016) Belarusian pharmaceutical market. Business guide. Available online: http://www.investinbelarus.by/upload/pdf/2016_%D0%A4%D0%B0%D1%80%D0%BC%D0%B0%D1%86%D0%B5%D0%B2%D1%82%D0%B8%D0%BA%D0%B0_%D0%BE%D0%B1%D0%BD%D0%BE%D0%B2%D0%BB%D0%B5%D0%BD%D0%BD%D0%B0%D1%8F.pdf
- 34.Tyupa V (2018) Distribution and marketing of drugs in the Russian Federation: overview. Thomson Reuters Practical Law. Available online: https://uk.practicallaw.thomsonreuters.com/5-618-5113?transitionType=Default&contextData=(sc.Default)&firstPage=true&bhcp=1
- 35.Karakulak ÖA, Doğan D, Geçgil TA (2018) Distribution and marketing of drugs in Turkey: overview. Thomson Reuters Practical Law. https://uk.practicallaw.thomsonreuters.com/2-618-7255?transitionType=Default&contextData=(sc.Default)&firstPage=true&bhcp=1
- 37.OECD (2008) OECD health policy studies: pharmaceutical pricing policies in the global market. http://apps.who.int/medicinedocs/documents/s19834en/s19834en.pdf
- 38.Schlander M, Garattini S, Kolominsky-Rabas P, Nord E, Persson U, Postma M, Richardson J, Simoens S, Solà-Morales O, Tolley K, Toumi M (2016) Determining the value of medical technologies to treat ultra-rare disorders: a consensus statement. J Market Access Health Policy 4:33039. https://doi.org/10.3402/jmahp.v4.33039 CrossRefGoogle Scholar
- 39.Kanavos P, Vandoros S, Irwin R, Nicod E, Casson M, Medical Technology Research Group – LSE Health et al (2011) Differences in costs of and access to pharmaceutical products in the EU. European Commission, Directorate general for internal policies, Policy department A: Economic and scientific policy. Brussels. http://www.europarl.europa.eu/RegData/etudes/etudes/join/2011/451481/IPOL-ENVI_ET(2011)451481_EN.pdf
- 40.Dukes MNG, Haaijer-Ruskamp FM, de Joncheere CP, Reitveld AH (eds) (2003) World health organization drugs and money - prices, affordability and cost containment, 7th edn. Available online: http://apps.who.int/medicinedocs/pdf/s4912e/s4912e.pdf