A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries
Background: Despite international initiatives on collaboration within the field of rare diseases, patient access to orphan medicinal products (OMPs) and healthcare services differ greatly between countries. This study aimed to create a comprehensive and in-depth overview of rare diseases policies an...
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2020-01-01
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| Series: | Frontiers in Public Health |
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| Online Access: | https://www.frontiersin.org/article/10.3389/fpubh.2019.00416/full |
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DOAJ |
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English |
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Article |
| sources |
DOAJ |
| author |
Marcin Czech Aleksandra Baran-Kooiker Kagan Atikeler Kagan Atikeler Maria Demirtshyan Kamilla Gaitova Malwina Holownia-Voloskova Malwina Holownia-Voloskova Adina Turcu-Stiolica Coen Kooiker Oresta Piniazhko Natella Konstandyan Olha Zalis'ka Jolanta Sykut-Cegielska |
| spellingShingle |
Marcin Czech Aleksandra Baran-Kooiker Kagan Atikeler Kagan Atikeler Maria Demirtshyan Kamilla Gaitova Malwina Holownia-Voloskova Malwina Holownia-Voloskova Adina Turcu-Stiolica Coen Kooiker Oresta Piniazhko Natella Konstandyan Olha Zalis'ka Jolanta Sykut-Cegielska A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries Frontiers in Public Health rare diseases newborn screening national plan patient registries reimbursement policy |
| author_facet |
Marcin Czech Aleksandra Baran-Kooiker Kagan Atikeler Kagan Atikeler Maria Demirtshyan Kamilla Gaitova Malwina Holownia-Voloskova Malwina Holownia-Voloskova Adina Turcu-Stiolica Coen Kooiker Oresta Piniazhko Natella Konstandyan Olha Zalis'ka Jolanta Sykut-Cegielska |
| author_sort |
Marcin Czech |
| title |
A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries |
| title_short |
A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries |
| title_full |
A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries |
| title_fullStr |
A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries |
| title_full_unstemmed |
A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries |
| title_sort |
review of rare disease policies and orphan drug reimbursement systems in 12 eurasian countries |
| publisher |
Frontiers Media S.A. |
| series |
Frontiers in Public Health |
| issn |
2296-2565 |
| publishDate |
2020-01-01 |
| description |
Background: Despite international initiatives on collaboration within the field of rare diseases, patient access to orphan medicinal products (OMPs) and healthcare services differ greatly between countries. This study aimed to create a comprehensive and in-depth overview of rare diseases policies and reimbursement of OMPs in a selection of 12 countries in the Western Eurasian region: Armenia, France, Germany, Kazakhstan, Latvia, The Netherlands, Poland, Romania, Russia, Turkey, Ukraine, and the United Kingdom.Methods: A systematic literature review was performed and an analysis of publicly available legislative and rare disease health policy data was undertaken in five focus areas: rare disease definition, newborn screening, registries, national plans, access to/reimbursement of OMPs.Results: Screening programs are broadly implemented but the number of screened diseases differs significantly (2–35 diseases), either between EU and non-EU countries, between EU member states and sometimes even within a single country. In most countries rare disease registries are operating with regional, national, European or worldwide coverage. The number of rare disease registries is growing, as a result of the National Plans (EU) and increased international scientific cooperation. France, Russia, and Poland have a centrally acting registry. National plans are present in all EU countries but implementation varies and is ongoing. The number of reimbursed OMPs in the selected countries ranges from nearly all available OMPs in the Netherlands, Germany, and France to zero in Armenia. Reimbursement rules differ considerably regionally and a trend is observed of reimbursement conditions getting stricter for expensive (orphan) drugs.Discussion: Inequality in patient access to new OMPs still exists due to variations in national policies, healthcare budgets, health insurance, and reimbursement systems. The observed differences are challenging for rare disease patients, health authorities and manufacturers alike. Progress can be seen, however, and international cooperation and harmonization is slowly but steadily expanding in the rare disease arena. |
| topic |
rare diseases newborn screening national plan patient registries reimbursement policy |
| url |
https://www.frontiersin.org/article/10.3389/fpubh.2019.00416/full |
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doaj-54fbf934650d4dfaa00a5bc3825e34262020-11-25T00:29:13ZengFrontiers Media S.A.Frontiers in Public Health2296-25652020-01-01710.3389/fpubh.2019.00416502840A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian CountriesMarcin Czech0Aleksandra Baran-Kooiker1Kagan Atikeler2Kagan Atikeler3Maria Demirtshyan4Kamilla Gaitova5Malwina Holownia-Voloskova6Malwina Holownia-Voloskova7Adina Turcu-Stiolica8Coen Kooiker9Oresta Piniazhko10Natella Konstandyan11Olha Zalis'ka12Jolanta Sykut-Cegielska13Department of Pharmacoeconomics, The Institute of Mother and Child, Warsaw, PolandDepartment of Pharmacoeconomics, Faculty of Pharmacy, Medical University of Warsaw, Warsaw, PolandDivision of Pharmacoepidemiology and Clinical Pharmacology, Faculty of Science, Utrecht Institute for Pharmaceutical Sciences, Utrecht, NetherlandsUnit of Health Technology Assessments, Turkish Ministry of Health, Turkish Medicines and Medical Devices Agency, Ankara, TurkeyAscent Global Market Solutions (Non-profit), Walnut Creek, CA, United StatesCenter for Economics and Health Technology Assessment, Republican Center for Health Development, Ministry of Health, Nur-Sultan, KazakhstanState Budgetary Institution Research Institute for Healthcare Organization and Medical Management of Moscow Healthcare Department, Moscow, RussiaDepartment of Experimental and Clinical Pharmacology, Medical University of Warsaw, Warsaw, PolandDepartment of Pharmacoeconomics, Faculty of Pharmacy, University of Medicine and Pharmacy of Craiova, Craiova, Romania0Independent Researcher, Warsaw, Poland1Department of Management and Economy of Pharmacy, Medicine Technology and Pharmacoeconomics, Postgraduate Faculty, Danylo Halytsky Lviv National Medical University, Lviv, Ukraine2Republican Center of Medical Genetics, Yerevan State Medical University, Yerevan, Armenia1Department of Management and Economy of Pharmacy, Medicine Technology and Pharmacoeconomics, Postgraduate Faculty, Danylo Halytsky Lviv National Medical University, Lviv, Ukraine3Department of Inborn Errors of Metabolism and Paediatrics, The Institute of Mother and Child, Warsaw, PolandBackground: Despite international initiatives on collaboration within the field of rare diseases, patient access to orphan medicinal products (OMPs) and healthcare services differ greatly between countries. This study aimed to create a comprehensive and in-depth overview of rare diseases policies and reimbursement of OMPs in a selection of 12 countries in the Western Eurasian region: Armenia, France, Germany, Kazakhstan, Latvia, The Netherlands, Poland, Romania, Russia, Turkey, Ukraine, and the United Kingdom.Methods: A systematic literature review was performed and an analysis of publicly available legislative and rare disease health policy data was undertaken in five focus areas: rare disease definition, newborn screening, registries, national plans, access to/reimbursement of OMPs.Results: Screening programs are broadly implemented but the number of screened diseases differs significantly (2–35 diseases), either between EU and non-EU countries, between EU member states and sometimes even within a single country. In most countries rare disease registries are operating with regional, national, European or worldwide coverage. The number of rare disease registries is growing, as a result of the National Plans (EU) and increased international scientific cooperation. France, Russia, and Poland have a centrally acting registry. National plans are present in all EU countries but implementation varies and is ongoing. The number of reimbursed OMPs in the selected countries ranges from nearly all available OMPs in the Netherlands, Germany, and France to zero in Armenia. Reimbursement rules differ considerably regionally and a trend is observed of reimbursement conditions getting stricter for expensive (orphan) drugs.Discussion: Inequality in patient access to new OMPs still exists due to variations in national policies, healthcare budgets, health insurance, and reimbursement systems. The observed differences are challenging for rare disease patients, health authorities and manufacturers alike. Progress can be seen, however, and international cooperation and harmonization is slowly but steadily expanding in the rare disease arena.https://www.frontiersin.org/article/10.3389/fpubh.2019.00416/fullrare diseasesnewborn screeningnational planpatient registriesreimbursementpolicy |