Transferability of Real World Evidence to Support HTA Recommendations in Lower Income European Countries
Kaló Z. Petrova G. Németh B. Bonča P.D. Delnoij D. Bennett A. Tachkov K. Zhang Y. Kreif N. Knies S. Lipska I. Tesar T. Mitkova Z. Ponten J. Viberg A. Paveliu M.-S. Piniazkho O. Turcu-Stiolica A. Doležal T. Staňák M. Almadiyeva A. Hren R. Vitezić D. Petykó Z. Goettsch W. Manca A.
February 2026John Wiley and Sons Inc
Health Science Reports
2026#9Issue 2
Background and Aims: Lower income European countries (LIECs) have more limited financial resources to cover high-cost technologies in rare diseases than higher income European countries (HIECs). Our study explores how treatment recommendations in myelodysplastic syndrome (MDS) can be supported in LIECS by transferring real-world evidence (RWE) generated by target trial emulation (TTE) method in HIECs. Method: In the HTx project transferability aspects of the MDS case study were considered upfront. HTA agency consortium partners set expectations for the MDS case study team on how to integrate the new TTE methodology into the routine work of HTA bodies. In consecutive workshops consortium members and external HTA experts identified the main challenges of transferring evidence generated by TTE method to LIECs and made conclusions on how to overcome these challenges. Results: The lack of local real-world data before making reimbursement decisions is an important challenge to apply the TTE method to LIECs. Differences in patient pathways and comparator technologies, limited expertise and resources for adapting international HTA methods are significant barriers of transferring RWE from other countries. Still, transferring RWE to LIECs from other countries based on the TTE methodology represents an improvement to the current standard HTA methods, especially if joint clinical assessment provides the unbiased judgement on the relative effectiveness of orphan medicines. The TTE approach also provides an opportunity to LIECs to judge the value of high-cost technologies for different patient subgroups. However, HTA professionals in LIECs need training about advanced methodologies. Conclusion: This is the first study to explore how RWE generated by the TTE method can be transferred to optimize treatment decisions of patients with a rare disease in countries with limited HTA capacities. Five general concluding statements were made on the novelty of the TTE method and on how to overcome main challenges of transferring TTE results to HTA systems in LIECs.
health technology assessment , lower income European countries , real world evidence , target trial emulation , transferability
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Center for Health Technology Assessment, Semmelweis University, Budapest, Hungary
Center for Pharmacology and Drug Research & Development, Semmelweis University, Budapest, Hungary
Syreon Research Institute, Budapest, Hungary
Department of Organization and Economics of Pharmacy, Faculty of Pharmacy, Medical University of Sofia, Sofia, Bulgaria
School of Economics and Business, University of Ljubljana, Ljubljana, Slovenia
National Health Care Institute, Diemen, Netherlands
Erasmus School of Health Policy & Management, Erasmus University Rotterdam, Rotterdam, Netherlands
Centre for Health Economics, University of York, York, United Kingdom
Academy for Medical and Social Applied Sciences, Gdańsk, Poland
Health Policy Institute, Gdańsk, Poland
Department of Organisation and Management in Pharmacy, Faculty of Pharmacy, Comenius University, Bratislava, Slovakia
The Dental and Pharmaceutical Benefits Agency (TLV), Stockholm, Sweden
Department of Pharmacology, Titu Maiorescu University, Bucharest, Romania
HTA Department of State Expert Centre, Ministry of Health, Kyiv, Ukraine
Department of Pharmacoeconomics, University of Medicine and Pharmacy of Craiova, Craiova, Romania
Department of Pharmacology, Faculty of Medicine, Masaryk University, Brno, Czech Republic
Value Outcomes, Prague, Czech Republic
Cogvio, Prague, Czech Republic
National Institute for Value and Technologies in Healthcare, Bratislava, Slovakia
Salidat Kairbekova National Research Center for Health Development, Astana, Kazakhstan
Institute of Mathematics Physics and Mechanics, Ljubljana, Slovenia
Faculty of Mathematics and Physics, University of Ljubljana, Ljubljana, Slovenia
Faculty of Medicine University of Rijeka and University Hospital Centre Rijeka, Rijeka, Croatia
Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences (UIPS), Utrecht University, Utrecht, Netherlands
Center for Health Technology Assessment
Center for Pharmacology and Drug Research & Development
Syreon Research Institute
Department of Organization and Economics of Pharmacy
School of Economics and Business
National Health Care Institute
Erasmus School of Health Policy & Management
Centre for Health Economics
Academy for Medical and Social Applied Sciences
Health Policy Institute
Department of Organisation and Management in Pharmacy
The Dental and Pharmaceutical Benefits Agency (TLV)
Department of Pharmacology
HTA Department of State Expert Centre
Department of Pharmacoeconomics
Department of Pharmacology
Value Outcomes
Cogvio
National Institute for Value and Technologies in Healthcare
Salidat Kairbekova National Research Center for Health Development
Institute of Mathematics Physics and Mechanics
Faculty of Mathematics and Physics
Faculty of Medicine University of Rijeka and University Hospital Centre Rijeka
Division of Pharmacoepidemiology and Clinical Pharmacology
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