CROSS-COUNTRY COMPARISON OF RARE DISEASE FINANCING MODELS AND ACCESS POLICIES: IMPLICATIONS FOR TURKIYE
Author(s)
Filiz Darici, MSc1, OZNUR SEYHUN, BSc, MFE, MSc2, Guvenc Kockaya, MSc, PhD, MD3, Selin Okcun, MSc4.
1Econix Research, İstanbul, Turkey, 2Econix Research, Tallin, Estonia, 3ec, Tallin, Estonia, 4Health Economist, Econix Research, Samsun, Turkey.
1Econix Research, İstanbul, Turkey, 2Econix Research, Tallin, Estonia, 3ec, Tallin, Estonia, 4Health Economist, Econix Research, Samsun, Turkey.
OBJECTIVES: Access and financing challenges in rare diseases require different policy approaches across countries. To derive strategic policy recommendations for Türkiye by evaluating and comparing orphan drug legislation, dedicated financing mechanisms, and patient access pathways across six selected countries.
METHODS: Rare disease legislation, national strategies, reimbursement and financing mechanisms in France, Australia, Singapore, Russia, Italy, and Türkiye were evaluated through a targeted policy review. Countries were compared in terms of national plans, dedicated funding, orphan drug access, health technology assessment approaches, and patient registry systems.
RESULTS: A national strategy or action plan was identified in five of the six countries, while four had financing mechanisms specific to rare diseases or orphan drugs. France represents one of the most comprehensive models, with three national rare disease plans and a network of reference centers. In Australia, among 124 medicines granted orphan drug designation between 2018 and 2023, 90 were approved and 48 were reimbursed. As of 2024, Singapore’s Rare Disease Fund, supported by 3:1 government co-financing, covers 5 rare diseases and 8 medicines. In Russia, the number of rare diseases covered under the Circle of Kindness Foundation increased from 27 in 2021 to 88 in 2023. In Italy, early access and dedicated funding mechanisms support access to orphan drugs. Overall, effective models commonly include national strategic planning, dedicated financing mechanisms, patient registry systems, and flexible access or reimbursement processes. In Türkiye, the 2023-2027 Rare Diseases Health Strategy Document and Action Plan has strengthened the policy framework; however, orphan drug-specific financing and a comprehensive national patient registry remain key development areas.
CONCLUSIONS: International examples show that different financing models can support patient access. For Türkiye, key priorities include strengthening the financing dimension of the action plan, developing orphan drug-specific assessment processes, and expanding national patient registry infrastructure.
METHODS: Rare disease legislation, national strategies, reimbursement and financing mechanisms in France, Australia, Singapore, Russia, Italy, and Türkiye were evaluated through a targeted policy review. Countries were compared in terms of national plans, dedicated funding, orphan drug access, health technology assessment approaches, and patient registry systems.
RESULTS: A national strategy or action plan was identified in five of the six countries, while four had financing mechanisms specific to rare diseases or orphan drugs. France represents one of the most comprehensive models, with three national rare disease plans and a network of reference centers. In Australia, among 124 medicines granted orphan drug designation between 2018 and 2023, 90 were approved and 48 were reimbursed. As of 2024, Singapore’s Rare Disease Fund, supported by 3:1 government co-financing, covers 5 rare diseases and 8 medicines. In Russia, the number of rare diseases covered under the Circle of Kindness Foundation increased from 27 in 2021 to 88 in 2023. In Italy, early access and dedicated funding mechanisms support access to orphan drugs. Overall, effective models commonly include national strategic planning, dedicated financing mechanisms, patient registry systems, and flexible access or reimbursement processes. In Türkiye, the 2023-2027 Rare Diseases Health Strategy Document and Action Plan has strengthened the policy framework; however, orphan drug-specific financing and a comprehensive national patient registry remain key development areas.
CONCLUSIONS: International examples show that different financing models can support patient access. For Türkiye, key priorities include strengthening the financing dimension of the action plan, developing orphan drug-specific assessment processes, and expanding national patient registry infrastructure.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HPR75
Topic
Health Policy & Regulatory
Topic Subcategory
Pricing Policy & Schemes
Disease
Rare & Orphan Diseases