FROM MOLECULAR DIAGNOSIS TO MARKET ACCESS: AVAILABILITY, REIMBURSEMENT TIMING, AND BUDGET IMPACT OF LYSOSOMAL STORAGE DISEASE THERAPIES IN TURKIYE
Author(s)
Ceren Simsek, MSc1, Fatmahan Atalar, PhD2, Altug Ozkosar, MSc1.
1Department of Rare Diseases, Istanbul University, Institution of Health Sciences, Istanbul, Turkey, 2Rare Diseases Research Laboratory, Istanbul University, Faculty of Medicine, Istanbul, Turkey.
1Department of Rare Diseases, Istanbul University, Institution of Health Sciences, Istanbul, Turkey, 2Rare Diseases Research Laboratory, Istanbul University, Faculty of Medicine, Istanbul, Turkey.
OBJECTIVES: Molecular diagnosis is increasingly central to treatment selection in lysosomal storage diseases (LSDs), but disease-specific therapy does not necessarily translate into patient access. This study aimed to characterize selected LSD therapies in Türkiye and evaluate marketing authorization(MA), market availability, reimbursement and budget impact to Turkish Social Security Institution (SGK).
METHODS: A structured review identified 15 prevalent LSDs. MPS VI and VII were additionally included because of treatment availability and access relevance in Türkiye. ICD-10 and ATC-5 codes were mapped; disease-specific medicines were identified through Orphanet. EMA/ FDA approvals, Turkish MA, market availability, reimbursement (Annex 4/A (İGÖK/AGÖK)), Annex 4/C foreign-medicine list, and early access program were assessed using official sources. SGK budget impact was estimated using IQVIA pack sales and public prices. Projected 2026 SGK pharmaceutical expenditure was derived from historical SGK data and used to estimate the budget share of LSD therapies.
RESULTS: Twenty disease-medicine indication records were identified, mainly enzyme replacement therapies. Sixteen records had Turkish MA (80%), but only 10 were both licensed and marketed (50%). Median delays from FDA/EMA approval to Turkish MA were 5.3 and 5.8 years, respectively, median time from Turkish MAto reimbursement was 1.60 years. Among nine reimbursed and marketed records with observable sales and price data, estimated annual SGK burden was approximately USD 205 million, representing 1.72% of projected 2026 SGK pharmaceutical expenditure. Budget impact was driven by Gaucher disease, Fabry disease, MPS II, and MPS I.
CONCLUSIONS: Access to LSD therapies in Türkiye is shaped by authorization, market availability, reimbursement, and budget measurability. Although market access exist, remains heterogeneous, and expenditure is concentrated in a small number of LSDs. Annex 4/C and early-access may represent an important but non-observable budget components because public data are limited. Integrated monitoring of authorization, availability, reimbursement, utilization, and expenditure is needed to support timely, equitable and evidence-based access decisions.
METHODS: A structured review identified 15 prevalent LSDs. MPS VI and VII were additionally included because of treatment availability and access relevance in Türkiye. ICD-10 and ATC-5 codes were mapped; disease-specific medicines were identified through Orphanet. EMA/ FDA approvals, Turkish MA, market availability, reimbursement (Annex 4/A (İGÖK/AGÖK)), Annex 4/C foreign-medicine list, and early access program were assessed using official sources. SGK budget impact was estimated using IQVIA pack sales and public prices. Projected 2026 SGK pharmaceutical expenditure was derived from historical SGK data and used to estimate the budget share of LSD therapies.
RESULTS: Twenty disease-medicine indication records were identified, mainly enzyme replacement therapies. Sixteen records had Turkish MA (80%), but only 10 were both licensed and marketed (50%). Median delays from FDA/EMA approval to Turkish MA were 5.3 and 5.8 years, respectively, median time from Turkish MAto reimbursement was 1.60 years. Among nine reimbursed and marketed records with observable sales and price data, estimated annual SGK burden was approximately USD 205 million, representing 1.72% of projected 2026 SGK pharmaceutical expenditure. Budget impact was driven by Gaucher disease, Fabry disease, MPS II, and MPS I.
CONCLUSIONS: Access to LSD therapies in Türkiye is shaped by authorization, market availability, reimbursement, and budget measurability. Although market access exist, remains heterogeneous, and expenditure is concentrated in a small number of LSDs. Annex 4/C and early-access may represent an important but non-observable budget components because public data are limited. Integrated monitoring of authorization, availability, reimbursement, utilization, and expenditure is needed to support timely, equitable and evidence-based access decisions.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HPR189
Topic
Economic Evaluation, Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Public Spending & National Health Expenditures, Reimbursement & Access Policy
Disease
Biologics & Biosimilars, No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases