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Analysis of patient access to orphan drugs in Turkey

BACKGROUND: Rare diseases are life-threatening, serious, and chronic conditions that require complex care and have a low prevalence. An estimated one in 15 people worldwide are affected by rare diseases. This study aims to analyze the accessibility, reimbursement status, licensed status, and Anatomi...

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Autores principales: Koçkaya, Güvenç, Atalay, Sibel, Oğuzhan, Gülpembe, Kurnaz, Mustafa, Ökçün, Selin, Sar Gedik, Çiğdem, Şaylan, Mete, Şencan, Nazlı
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7868010/
https://www.ncbi.nlm.nih.gov/pubmed/33549137
http://dx.doi.org/10.1186/s13023-021-01718-3
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author Koçkaya, Güvenç
Atalay, Sibel
Oğuzhan, Gülpembe
Kurnaz, Mustafa
Ökçün, Selin
Sar Gedik, Çiğdem
Şaylan, Mete
Şencan, Nazlı
author_facet Koçkaya, Güvenç
Atalay, Sibel
Oğuzhan, Gülpembe
Kurnaz, Mustafa
Ökçün, Selin
Sar Gedik, Çiğdem
Şaylan, Mete
Şencan, Nazlı
author_sort Koçkaya, Güvenç
collection PubMed
description BACKGROUND: Rare diseases are life-threatening, serious, and chronic conditions that require complex care and have a low prevalence. An estimated one in 15 people worldwide are affected by rare diseases. This study aims to analyze the accessibility, reimbursement status, licensed status, and Anatomical Therapeutic Chemical (ATC) codes of drugs that the European Medicines Agency (EMA) in Turkey considers to be “orphan” pharmaceuticals. METHODS: The drugs included in this analysis were obtained from the list of orphan drugs published by the EMA. Orphan drugs’ accessibility and licensing status in Turkey were obtained from the Health Implementation Communiqué published by the Social Security Institution (SGK) and the List of Abroad Active Substance and List of Licensed Products published by the Turkey Pharmaceuticals and Medical Devices Agency (TİTCK). Descriptive analysis was applied to determine the accessibility status of orphan drugs identified by the EMA in Turkey. RESULTS: Based on the EMA, 105 pharmaceuticals were approved with “orphan drug” status except for drugs that have lost orphan drug status, decommissioned in the European Union and withdrawn from the European Community Register by January 2020. Of the 105 rare drugs on the EMA list, 34 were inaccessible in Turkey. Of the 71 available drugs, 23 (32%) were licensed and 48 (68%) were unlicensed in Turkey. 17 (74%) of licensed products and 17 (35%) of unlicensed products were covered by reimbursement. When orphan drugs’ ATC codes were examined, the most common ATC group was found to be “L—Antineoplastic and Immunomodulatory” agents. CONCLUSION: An orphan drug incentive policy is very important to ensure early access to the drugs used to treat rare diseases. Considering the capacity and prices for orphan drugs in Turkey, it can be said that many patients with rare diseases have difficulty in their treatment. It is obvious that such a policy must prepare for the regulation of orphan drugs in Turkey.
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spelling pubmed-78680102021-02-08 Analysis of patient access to orphan drugs in Turkey Koçkaya, Güvenç Atalay, Sibel Oğuzhan, Gülpembe Kurnaz, Mustafa Ökçün, Selin Sar Gedik, Çiğdem Şaylan, Mete Şencan, Nazlı Orphanet J Rare Dis Research BACKGROUND: Rare diseases are life-threatening, serious, and chronic conditions that require complex care and have a low prevalence. An estimated one in 15 people worldwide are affected by rare diseases. This study aims to analyze the accessibility, reimbursement status, licensed status, and Anatomical Therapeutic Chemical (ATC) codes of drugs that the European Medicines Agency (EMA) in Turkey considers to be “orphan” pharmaceuticals. METHODS: The drugs included in this analysis were obtained from the list of orphan drugs published by the EMA. Orphan drugs’ accessibility and licensing status in Turkey were obtained from the Health Implementation Communiqué published by the Social Security Institution (SGK) and the List of Abroad Active Substance and List of Licensed Products published by the Turkey Pharmaceuticals and Medical Devices Agency (TİTCK). Descriptive analysis was applied to determine the accessibility status of orphan drugs identified by the EMA in Turkey. RESULTS: Based on the EMA, 105 pharmaceuticals were approved with “orphan drug” status except for drugs that have lost orphan drug status, decommissioned in the European Union and withdrawn from the European Community Register by January 2020. Of the 105 rare drugs on the EMA list, 34 were inaccessible in Turkey. Of the 71 available drugs, 23 (32%) were licensed and 48 (68%) were unlicensed in Turkey. 17 (74%) of licensed products and 17 (35%) of unlicensed products were covered by reimbursement. When orphan drugs’ ATC codes were examined, the most common ATC group was found to be “L—Antineoplastic and Immunomodulatory” agents. CONCLUSION: An orphan drug incentive policy is very important to ensure early access to the drugs used to treat rare diseases. Considering the capacity and prices for orphan drugs in Turkey, it can be said that many patients with rare diseases have difficulty in their treatment. It is obvious that such a policy must prepare for the regulation of orphan drugs in Turkey. BioMed Central 2021-02-06 /pmc/articles/PMC7868010/ /pubmed/33549137 http://dx.doi.org/10.1186/s13023-021-01718-3 Text en © The Author(s) 2021 Open AccessThis article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated in a credit line to the data.
spellingShingle Research
Koçkaya, Güvenç
Atalay, Sibel
Oğuzhan, Gülpembe
Kurnaz, Mustafa
Ökçün, Selin
Sar Gedik, Çiğdem
Şaylan, Mete
Şencan, Nazlı
Analysis of patient access to orphan drugs in Turkey
title Analysis of patient access to orphan drugs in Turkey
title_full Analysis of patient access to orphan drugs in Turkey
title_fullStr Analysis of patient access to orphan drugs in Turkey
title_full_unstemmed Analysis of patient access to orphan drugs in Turkey
title_short Analysis of patient access to orphan drugs in Turkey
title_sort analysis of patient access to orphan drugs in turkey
topic Research
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7868010/
https://www.ncbi.nlm.nih.gov/pubmed/33549137
http://dx.doi.org/10.1186/s13023-021-01718-3
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