Registration and Access to Orphan Medicines in Türkiye: A Comparative Study With Poland and Germany
Author(s)
Kagan Atikeler, MSc1, Aukje Mantel-Teeuwisse, Prof. Dr.1, Nurseda Akgül, BSc2, Tina Wang, PhD3, Wim Goettsch, MSc, PhD1.
1Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sc, Utrecht University, Utrecht, Netherlands, 2Econix Research, Samsun, Turkey, 3CIRS, London, United Kingdom.
1Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sc, Utrecht University, Utrecht, Netherlands, 2Econix Research, Samsun, Turkey, 3CIRS, London, United Kingdom.
OBJECTIVES: Access to orphan medicinal products (OMPs) varies significantly between European countries due to differences in regulatory, reimbursement, and policy frameworks. This study aims to assess the approval and licensing timelines and reimbursement conditions of OMPs recommended for approval by the European Medicines Agency (EMA) in Türkiye and to make comparisons regarding HTA and reimbursement recommendations with Germany and Poland.
METHODS: OMP data from 2016-2024 in Türkiye, corresponding to EMA approvals from 2016-2022, were reviewed using records from the Turkish Medicines and Medical Devices Agency (TİTCK) and the Social Security Institution (SGK). Information from Germany and Poland was gathered through national HTA and reimbursement sources. Licensing delays and reimbursement timelines were analysed, and reimbursement criteria were compared across the three countries.
RESULTS: Among the 97 orphan drugs approved by the EMA between 2016 and 2022, 24 were licensed in Turkey. 11 of these medications were included in reimbursement, and 13 were excluded. The most frequently represented ATC group among the licensed orphan drugs in Türkiye was "L—Antineoplastic and Immunomodulatory Agents." The average delay between EMA approval and Türkiye’s licensing was 1,152 days (median: 990 days), and approval took an average of 537 days. The comparative analysis of HTA and reimbursement recommendations for OMPs revealed that Germany offers the broadest access with minimal restrictions, Türkiye applies the most restrictive reimbursement criteria, and Poland generally limits access to narrow subpopulations.
CONCLUSIONS: Our study suggests a demand for further process optimisation and resource allocation for MA approvals in Türkiye. As expected, we demonstrated that reimbursement processes for OMPs differ across the three studied countries. In summary, these findings highlight the need for a deeper understanding of the observed differences and an exploration of the potential for harmonising reimbursement policies for OMPs across similar settings in Europe.
METHODS: OMP data from 2016-2024 in Türkiye, corresponding to EMA approvals from 2016-2022, were reviewed using records from the Turkish Medicines and Medical Devices Agency (TİTCK) and the Social Security Institution (SGK). Information from Germany and Poland was gathered through national HTA and reimbursement sources. Licensing delays and reimbursement timelines were analysed, and reimbursement criteria were compared across the three countries.
RESULTS: Among the 97 orphan drugs approved by the EMA between 2016 and 2022, 24 were licensed in Turkey. 11 of these medications were included in reimbursement, and 13 were excluded. The most frequently represented ATC group among the licensed orphan drugs in Türkiye was "L—Antineoplastic and Immunomodulatory Agents." The average delay between EMA approval and Türkiye’s licensing was 1,152 days (median: 990 days), and approval took an average of 537 days. The comparative analysis of HTA and reimbursement recommendations for OMPs revealed that Germany offers the broadest access with minimal restrictions, Türkiye applies the most restrictive reimbursement criteria, and Poland generally limits access to narrow subpopulations.
CONCLUSIONS: Our study suggests a demand for further process optimisation and resource allocation for MA approvals in Türkiye. As expected, we demonstrated that reimbursement processes for OMPs differ across the three studied countries. In summary, these findings highlight the need for a deeper understanding of the observed differences and an exploration of the potential for harmonising reimbursement policies for OMPs across similar settings in Europe.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HPR172
Topic
Health Policy & Regulatory
Topic Subcategory
Reimbursement & Access Policy
Disease
Rare & Orphan Diseases