Sevim F., Akyürek Ç. E.
EUROPEAN JOURNAL OF PUBLIC HEALTH, cilt.36, sa.6, ss.1-5, 2026 (SCI-Expanded, SSCI, Scopus)
-
Yayın Türü:
Makale / Tam Makale
-
Cilt numarası:
36
Sayı:
6
-
Basım Tarihi:
2026
-
Doi Numarası:
10.1093/eurpub/ckag204
-
Dergi Adı:
EUROPEAN JOURNAL OF PUBLIC HEALTH
-
Derginin Tarandığı İndeksler:
Academic Search Ultimate (EBSCO), Natural Science Collection (ProQuest), Biomedical Reference Collection: Corporate Edition (EBSCO), Health Research Premium Collection (ProQuest), Scopus, Pharma Collection (ProQuest), Sociology Source Ultimate (EBSCO), Science Citation Index Expanded (SCI-EXPANDED), Social Sciences Citation Index (SSCI), ABI/INFORM, Abstracts in Social Gerontology, CINAHL, Educational research abstracts (ERA), EMBASE, Index Islamicus, MEDLINE, Political Science Complete, Psycinfo, Public Affairs Index, Directory of Open Access Journals, Political Science Abstract (IPSA)
-
Sayfa Sayıları:
ss.1-5
-
Karadeniz Teknik Üniversitesi Adresli:
Evet
Özet
Abstract
Rare diseases are individually uncommon but collectively affect a substantial share of any population. The medicines developed for them combine severe conditions, very small treatable populations, high prices, and evidence that is often immature at launch. Conventional cost-effectiveness analysis handles this poorly, and systems relying on it heavily tend to reimburse fewer orphan medicines than those applying alternative evidence standards. Türkiye offers an instructive current case. Of the 66 orphan medicines granted central marketing authorization in the European Union between 2021 and 2024, 3 were available in Türkiye at the start of 2026, against a European Union average of 28. The comparable figure for all new medicines was 9 of 168, against an average of 76. Restricted access to orphan medicines should therefore not be read solely as an orphan-specific exclusion, but as part of a broader constraint on new medicines, distinguished by one feature: a parallel route in which medicines are authorized for named patients, imported, and reimbursed outside the standard list. That route carries real access that list-based indicators capture only in part, and it makes a patient entitlement depend on which list a medicine occupies. A rule issued in June 2026 restricting reimbursement of spinal muscular atrophy medicines after gene replacement therapy illustrates the limited public availability of the clinical and economic reasoning behind reimbursement restrictions. Broadening the basis of appraisal, rather than reducing reliance on economic evaluation, requires published criteria, instruments for managing uncertainty, and monitoring of the pathway as well as the list.