Value Framework to Support Payer Decisions in Hemophilia in Türkiye
Author(s)
Zsuzsa Reka Pozsar, MA1, Saadet Koca, MSc2, Imrahim Yaradiliis, MD3, Hasan Coskun, MD3, AYSEGUL GUNBEY OZTEK, Pharm.4, Ugursel Erol, MSc4, Olgun SENER, MSc5, Sayime Koc, Assoc. Prof.6, Bulent Zulfikar, Prof.6, Caner Dadak, MD6, Haluk Ozsari, Prof.7, Zafer Caliskan, Prof.7, Huseyin Celik, MSc7, Hakki Gursoz, MD8, Ahmet Oğuz Sarıca, BSc, MSc, PhD9, Esra SAFAK YILMAZ, PhD10, Merve Altunba?, MD11, Ceren Simsek, Pharm.11, KAGAN ATIKELER, Pharm.12, Ozgur Pektas, MD12, Eray Oktay Yaşasın, BSc, MSc, PhD13, Guvenc Kockaya, Assoc. Prof.14, Oznur Seyhun, MSc14, Said Al Rashdi, BSc, MSc1, Tamas Agh, MSc, PhD, MD15, Zoltan Kalo, PhD1.
1a) Center for Health Technology Assessment, Semmelweis University, Budapest, Hungary b) Center for Pharmacology and Drug Research & Development, Semmelweis University, Budapest, Hungary c) Syreon Research Institute, Budapest, Hungary, 2Ministry of Treasury and Finance, Ankara, Turkey, 3Turkish Medicine and Medical Devices Agency, Ankara, Turkey, 4Social Security Institute (SGK), Ankara, Turkey, 5Ministry of Health of Republic of Türkiye, Çankaya-Ankara, Turkey, 6Turkish Hemophilia Association, Istanbul, Turkey, 7Value Based Health Association, Istanbul, Turkey, 8FarmaLojik, Ankara, Turkey, 9Presidency of Republic of Türkiye, Ankara, Turkey, 10Novo Nordisk, Ankara, Turkey, 11Novo Nordisk, Istanbul, Turkey, 12Sanofi Turkey, Istanbul, Turkey, 13Sanofi, İstanbul, Turkey, 14ECONIX, Istanbul, Turkey, 15Center for HTA and Pharmacoeconomic Research, University of Pecs & Syreon Research Institute, Budapest, Hungary.
1a) Center for Health Technology Assessment, Semmelweis University, Budapest, Hungary b) Center for Pharmacology and Drug Research & Development, Semmelweis University, Budapest, Hungary c) Syreon Research Institute, Budapest, Hungary, 2Ministry of Treasury and Finance, Ankara, Turkey, 3Turkish Medicine and Medical Devices Agency, Ankara, Turkey, 4Social Security Institute (SGK), Ankara, Turkey, 5Ministry of Health of Republic of Türkiye, Çankaya-Ankara, Turkey, 6Turkish Hemophilia Association, Istanbul, Turkey, 7Value Based Health Association, Istanbul, Turkey, 8FarmaLojik, Ankara, Turkey, 9Presidency of Republic of Türkiye, Ankara, Turkey, 10Novo Nordisk, Ankara, Turkey, 11Novo Nordisk, Istanbul, Turkey, 12Sanofi Turkey, Istanbul, Turkey, 13Sanofi, İstanbul, Turkey, 14ECONIX, Istanbul, Turkey, 15Center for HTA and Pharmacoeconomic Research, University of Pecs & Syreon Research Institute, Budapest, Hungary.
OBJECTIVES: The growing complexity of hemophilia therapies necessitates the reconsideration of value judgment by healthcare payers. This study aimed to develop a value framework (VF) to serve as the basis of a future multi-criteria decision analysis (MCDA) tool that facilitates transparent, structured, and evidence-informed evaluations of hemophilia treatments in Türkiye.
METHODS: A narrative literature review was conducted in January 2025 using Medline to identify value elements (VEs) with potential relevance to reimbursement decisions in hemophilia. The search strategy combined terms related to hemophilia, VFs, and MCDA. The review was supplemented with grey literature sources, including clinical guidelines and clinical trial databases. All identified VEs were critically assessed, deduplicated, and organized into thematic categories, resulting in a preliminary list of 12 VEs. This initial VF was further refined during two structured expert review meetings that engaged national representatives from clinical societies, patient organisations, policymakers, the national payer body, academic HTA experts, and the pharmaceutical industry.
RESULTS: Based on stakeholder consensus and considerations of future applicability, the initial VF was streamlined to a final set of 6 VEs, reflecting traditional, patient-centric, and societal perspectives. These include i) health gain, measured by the reduction in annual bleeding rate, ii) cost-effectiveness, iii) annual treatment cost, iv) local economic contribution metrics, v) patient experience with the treatment regimen, and vi) health technology-related health literacy of patients and caregivers.
CONCLUSIONS: Active engagement with a diverse group of stakeholders was critical to ensure the transparency, validity, and acceptability of the VF. The ranking and weighting of the selected VEs, along with the finalization of the scoring functions and preparation for future application, will be carried out to transform the VF into a functional MCDA tool that supports transparent, consistent, and value-based prioritization of hemophilia therapies within the Turkish healthcare system.
METHODS: A narrative literature review was conducted in January 2025 using Medline to identify value elements (VEs) with potential relevance to reimbursement decisions in hemophilia. The search strategy combined terms related to hemophilia, VFs, and MCDA. The review was supplemented with grey literature sources, including clinical guidelines and clinical trial databases. All identified VEs were critically assessed, deduplicated, and organized into thematic categories, resulting in a preliminary list of 12 VEs. This initial VF was further refined during two structured expert review meetings that engaged national representatives from clinical societies, patient organisations, policymakers, the national payer body, academic HTA experts, and the pharmaceutical industry.
RESULTS: Based on stakeholder consensus and considerations of future applicability, the initial VF was streamlined to a final set of 6 VEs, reflecting traditional, patient-centric, and societal perspectives. These include i) health gain, measured by the reduction in annual bleeding rate, ii) cost-effectiveness, iii) annual treatment cost, iv) local economic contribution metrics, v) patient experience with the treatment regimen, and vi) health technology-related health literacy of patients and caregivers.
CONCLUSIONS: Active engagement with a diverse group of stakeholders was critical to ensure the transparency, validity, and acceptability of the VF. The ranking and weighting of the selected VEs, along with the finalization of the scoring functions and preparation for future application, will be carried out to transform the VF into a functional MCDA tool that supports transparent, consistent, and value-based prioritization of hemophilia therapies within the Turkish healthcare system.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA355
Topic
Economic Evaluation, Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Value Frameworks & Dossier Format
Disease
Oncology, Rare & Orphan Diseases