ArticleOrphanet journal of rare diseases2021
Analysis of patient access to orphan drugs in Turkey.
Article in Orphanet journal of rare diseases, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
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Who cites it
11 citing papers in PubMed.
- Management of rare and undiagnosed diseases: insights from researchers and healthcare professionals in Türkiye.Frontiers in public health · 2024Article
- Pharmacy practice and policy research in Türkiye: a systematic review of literature.Journal of pharmaceutical policy and practice · 2024Review
- Enhancing Equitable Access to Rare Disease Diagnosis and Treatment around the World: A Review of Evidence, Policies, and Challenges.International journal of environmental research and public health · 2023Review
- Literature Review on Health Emigration in Rare Diseases-A Machine Learning Perspective.International journal of environmental research and public health · 2023Review
- Out-of-pocket healthcare expenditures of Turkish households living with rare diseases.Frontiers in public health · 2023Article
- Review
- Obstacles and expectations of rare disease patients and their families in Türkiye: ISTisNA project survey results.Frontiers in public health · 2022Article
- Gene Therapy for Neuromuscular Diseases: Health Economic Challenges and Future Perspectives.Journal of neuromuscular diseases · 2022Review
- The Awareness of Rare Diseases Among Medical Students and Practicing Physicians in the Republic of Kazakhstan. An Exploratory Study.Frontiers in public health · 2022Article
- Financing of Rare Diseases and Orphan Drugs in A Sanctioned Country: A Qualitative Study.Medical journal of the Islamic Republic of Iran · 2022Article
- Article
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Authors and funding
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundRare 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.
methodsThe 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.
resultsBased 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.
conclusionAn 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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