{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2023,9,25]],"date-time":"2023-09-25T17:07:11Z","timestamp":1695661631922},"reference-count":12,"publisher":"Akademiai Kiado Zrt.","issue":"44","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Orvosi Hetilap"],"published-print":{"date-parts":[[2014,11]]},"abstract":"Focusing on the benefits of patients with rare disease the authors analysed the aspects of orphan medicines financed in the frame of the Hungarian social insurance system in 2012 in order to make the consumption more rational, transparent and predictable. Most of the orphan drugs were financed in the frame of compassionate use by the reimbursement system. Consequently, a great deal of crucial problems occurred in relation to the unconventional subsidized method, especially in the case of the highest cost enzyme replacement therapies. On the base of the findings, proposals of the authors are presented for access to orphan drugs, fitting to the specific professional, economical and ethical aspects of this unique field of the health care system. The primary goal is to provide a suitable subsidized method for the treatment of rare disease patients with unmet medical needs. The financial modification of orphans became indispensible in Hungary. Professionals from numerous fields dealing with rare disease patients\u2019 care expressed agreement on the issue. Transforming the orphan medicines\u2019 financial structure has been initiated according to internationally shared principles. Orv. Hetil., 2014, 155(44), 1735\u20131741.<\/jats:p>","DOI":"10.1556\/oh.2014.30031","type":"journal-article","created":{"date-parts":[[2014,10,26]],"date-time":"2014-10-26T01:00:49Z","timestamp":1414285249000},"page":"1735-1741","source":"Crossref","is-referenced-by-count":4,"title":["Focus shifts in the Hungarian reimbursement system. Funding of orphan medicinal products for rare disease patients in Hungary: Financing of orphan medicines"],"prefix":"10.1556","volume":"155","author":[{"given":"M\u00e1rta","family":"Szegedi","sequence":"first","affiliation":[{"name":"Emberi Er\u0151forr\u00e1sok Miniszt\u00e9riuma Eg\u00e9szs\u00e9g\u00fcgy\u00e9rt Felel\u0151s \u00c1llamtitk\u00e1rs\u00e1g Budapest Arany J\u00e1nos u. 6\u20138. 1051"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"M\u00e1ria Judit","family":"Moln\u00e1r","sequence":"additional","affiliation":[{"name":"Semmelweis Egyetem, \u00c1ltal\u00e1nos Orvostudom\u00e1nyi Kar Genomikai Medicina \u00e9s Ritka Betegs\u00e9gek Int\u00e9zete Budapest"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Imre","family":"Boncz","sequence":"additional","affiliation":[{"name":"P\u00e9csi Tudom\u00e1nyegyetem, Eg\u00e9szs\u00e9gtudom\u00e1nyi Kar Eg\u00e9szs\u00e9gbiztos\u00edt\u00e1si Int\u00e9zet P\u00e9cs"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gy\u00f6rgy","family":"Kosztol\u00e1nyi","sequence":"additional","affiliation":[{"name":"P\u00e9csi Tudom\u00e1nyegyetem, Eg\u00e9szs\u00e9gtudom\u00e1nyi Kar Orvosi Genetikai Int\u00e9zet P\u00e9cs"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"425","reference":[{"key":"1","unstructured":"Hungarian National Health Insurance Fund Administration: Statistical Pocketbook 2012. [OEP Statisztikai Zsebk\u00f6nyv, 2012.] Duna-Mix Kft., Budapest, 2013. [Hungarian]"},{"key":"2","unstructured":"European Commission: Public Health:<\/i> Rare diseases \u2013 What are they? http:\/\/ec.europa.eu\/health-eu\/health_problems\/rare_diseases\/index_en.htm<\/a>"},{"key":"3","unstructured":"European Medicines Agency:<\/i> Orphan designation. http:\/\/www.emea.europa.eu\/ema\/index.jsp?curl=pages\/regulation\/general\/general_content_000029.jsp&mid=WC0b01ac05800240ce<\/a>"},{"key":"4","unstructured":"EUROPLAN Project:<\/i> The European Project for Rare Diseases National Plans Development. http:\/\/www.europlanproject.eu\/_newsite_986989\/project.html<\/a>"},{"key":"5","unstructured":"Orphanet Portal:<\/i> List of orphan drugs with marketing authorizations. http:\/\/www.orpha.net\/consor\/cgi-bin\/Drugs_ListOrphanDrugs_List.php?lng=EN&TAG=A<\/a>"},{"key":"6","unstructured":"European Medicines Agency:<\/i> European public assessment reports. http:\/\/www.ema.europa.eu\/ema\/index.jsp?curl=pages\/medicines\/landing\/epar_search.jsp&mid=WC0b01ac058001d124<\/a>"},{"key":"7","unstructured":"Hungarian National Health Insurance Fund Administration: Public druglist (PUPHA). [OEP Publikus Gy\u00f3gyszert\u00f6rzs] http:\/\/www.oep.hu\/portal\/page?_pageid=35,45948635&_dad=portal&_schema=PORTAL<\/a> [Hungarian]"},{"key":"8","unstructured":"Hungarian National Health Insurance Fund Administration database. [OEP-adatb\u00e1zis] [Hungarian]"},{"key":"9","unstructured":"Van Weely, S., Leufkens, H. G.:<\/i> Priority medicines for Europe and the world \u201eA public health approach to innovation\u201d. Background Paper 6.19 Rare Dis., 2013, 6, 19\u20136\u201319\u20138."},{"key":"10","unstructured":"European Commission: Enterprise and industry. Platform on access to medicines in Europe. Mechanism of coordinated access to orphan medicinal products. Key conclusions and recommendations. http:\/\/ec.europa.eu\/enterprise\/sectors\/healthcare\/competitiveness\/process_on_corporate_responsibility\/platform_access\/index_en.htm<\/a>"},{"key":"11","doi-asserted-by":"crossref","unstructured":"F\u00f6ldv\u00e1ri, A., Szy, I., S\u00e1ndor, J., et al.:<\/i> Diagnostic delay of rare diseases in Europe and in Hungary. [A ritka betegs\u00e9gek diagnosztik\u00e1j\u00e1nak k\u00e9sedelme Eur\u00f3p\u00e1ban \u00e9s Magyarorsz\u00e1gon.] Orv. Hetil., 2012, 153<\/i>(30), 1185\u20131190. [Hungarian]","DOI":"10.1556\/OH.2012.29418"},{"key":"12","doi-asserted-by":"crossref","unstructured":"Kosztol\u00e1nyi, Gy.:<\/i> Hungarian National Plan and Strategy for Rare Diseases. [Ritka Betegs\u00e9gek Nemzeti Terve.] Orv. Hetil., 2014, 155<\/i>(9), 325\u2013328. [Hungarian]","DOI":"10.1556\/OH.2014.29820"}],"container-title":["Orvosi Hetilap"],"original-title":[],"language":"hu","link":[{"URL":"https:\/\/www.akademiai.com\/doi\/pdf\/10.1556\/OH.2014.30031","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2020,5,22]],"date-time":"2020-05-22T09:56:18Z","timestamp":1590141378000},"score":1,"resource":{"primary":{"URL":"https:\/\/akjournals.com\/doi\/10.1556\/oh.2014.30031"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2014,11]]},"references-count":12,"journal-issue":{"issue":"44","published-print":{"date-parts":[[2014,11]]}},"alternative-id":["10.1556\/OH.2014.30031"],"URL":"https:\/\/doi.org\/10.1556\/oh.2014.30031","relation":{},"ISSN":["0030-6002","1788-6120"],"issn-type":[{"value":"0030-6002","type":"print"},{"value":"1788-6120","type":"electronic"}],"subject":[],"published":{"date-parts":[[2014,11]]}}}