Abstract
Purpose
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.
Methods
Web survey to authorities and representatives of third party payers in the Pharmaceutical Pricing and Reimbursement Information (PPRI) network in April 2016.
Results
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.
Conclusions
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.
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References
European Commission Regulation (EC) No. 141/2000. http://ec.europa.eu/health/documents/eudralex/vol-1_en
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
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/
Wästfelt M, Fadeel B, Henter J-I (2006) A journey of hope: lessons learned from studies on rare diseases and orphan drugs. J Intern Med 260(1):1–10. https://doi.org/10.1111/j.1365-2796.2006.01666.x
Kaplan W Priority medicines for Europe and the World 2013 Update. http://www.who.int/medicines/areas/priority_medicines/MasterDocJune28_FINAL_Web.pdf
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
WHO (2018) WHO collaborating centre for pharmaceutical pricing and reimbursement policies. Preferred terms. Available online: http://whocc.goeg.at/Glossary/PreferredTerms/Access
Melnikova I (2012) Rare diseases and orphan drugs. Nat Rev Drug Discov 11(4):267–268. https://doi.org/10.1038/nrd3654
European Medicines Agency (EMA) Orphan designation 2017. http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000029.jsp
European Medicines Agency (EMA). Orphan Medicines Figures 2000–2017. http://www.ema.europa.eu/docs/en_GB/document_library/Other/2015/04/WC500185766.pdf
Gammie T, Lu CY, Babar ZU-D (2015) Access to orphan drugs: a comprehensive review on legislations, regulations and policies in 35 countries. PLoS One 10(10):e0140002. https://doi.org/10.1371/journal.pone.0140002
Schey C, Milanova T, Hutchings A (2011) Estimating the budget impact of orphan medicines in Europe. Orphanet J Rare Dis 6(62):1–10. https://doi.org/10.1186/1750-1172-6-62
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
Gong S, Wang Y, Pan X, Zhang L, Huang R, Chen X, Hu J, Xu Y, Jin S (2016) The availability and affordability of orphan drugs for rare diseases in China. Orphanet J Rare Dis 11(20):1–12. https://doi.org/10.1186/s13023-016-0392-4
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):15–32
Logviss K, Kreivins D, Purvina S (2014) Rare diseases and orphan drugs: Latvian story. Orphanet J Rare Dis 9(147):1–13. https://doi.org/10.1186/s13023-014-0147-z
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
Barak A, Nandi JS (2011) Orphan drugs: pricing, reimbursement and patient access. Int J Pharm Healthc Mark 5(4):299–317. https://doi.org/10.1108/17506121111190121
Zelei T, Molnár MJ, Szegedi M, Kaló Z (2016) Systematic review on the evaluation criteria of orphan medicines in Central and Eastern European countries. Orphanet J Rare Dis 11(72):1–11. https://doi.org/10.1186/s13023-016-0455-6
Denis A, Mergaert L, Fostier C, Cleemput I, Simoens S (2010) A comparative study of European rare disease and orphan drug markets. Health Pol 97:173–179. https://doi.org/10.1016/j.healthpol.2010.05.017
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
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
Roll K, Stargardt T, Schreyögg J (2011) Authorization and reimbursement of orphan drugs in an international comparison. Gesundheitswesen 73(8–9):504–514. https://doi.org/10.1055/s-0030-1262864
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
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
WHO (2015) WHO collaborating centre for pharmaceutical pricing and reimbursement. PPRI: pharmaceutical pricing and reimbursement information. Available online: http://whocc.goeg.at/About/PPRI
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
Finnish Advisory Board on Research Integrity Ethical review in human sciences 2014. http://www.tenk.fi/en/ethical-review-in-human-sciences
University of Eastern Finland (2017) Research ethics. Available online: https://www.uef.fi/in/research/instructions-and-forms
Icelandic Medicines Agency (2017) Marketing authorisations (MA). Available online: https://www.ima.is/licences/marketing_authorisations/nr/2881
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/
Mallkuçi I (2017) Regulatory framework of orphan medicines in Albania. Pharm Regul Aff 6(1):1–4. https://doi.org/10.4172/2167-7689.1000190
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
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
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
Picavet E, Annemans L, Cleemput I, Cassiman D, Simoens S (2012) Market uptake of orphan drugs—a European analysis. J Clin Pharm Ther 37:664–667. https://doi.org/10.1111/j.1365-2710.2012.01364.x
OECD (2008) OECD health policy studies: pharmaceutical pricing policies in the global market. http://apps.who.int/medicinedocs/documents/s19834en/s19834en.pdf
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
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
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
Cote S, Gaudig M, Nielsen SK, Shields GE, Britton JA (2015) Challenges for assessing the economic value of orphan drugs—a literature review of current and alternative approaches. Value Health 18:A681
Michel M, Toumi M (2012) Access to orphan drugs in Europe: current and future issues. Exp Rev Pharmacoecon Outcomes Res 12(1):23–29. https://doi.org/10.1586/erp.11.95
Acknowledgements
We are grateful to the PPRI network for their participation and co-operation in this study.
Funding
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).
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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.
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The authors (KS, RA, JT) were funded by a research grant from the Social Insurance Institution of Finland (Kela) (grant number: 12/26/2015).
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This article does not contain any studies with human participants or animals performed by any of the authors.
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Sarnola, K., Ahonen, R., Martikainen, J.E. et al. Policies and availability of orphan medicines in outpatient care in 24 European countries. Eur J Clin Pharmacol 74, 895–902 (2018). https://doi.org/10.1007/s00228-018-2457-x
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DOI: https://doi.org/10.1007/s00228-018-2457-x