Transferability of real world evidence to support HTA recommendations in lower income European countries

Kaló, Zoltán (author), Došenović Bonča, Petra (author), Hren, Rok (author)

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.

• Language: English
• Keywords: healthcare, health technology assessment, Europe
• Work type: Article
• Typology: 1.01 - Original Scientific Article
• Organization: FMF - Faculty of Mathematics and Physics; EF - School of Economics and Business
• Publication status: Published
• Publication version: Version of Record
• Year: 2026
• Number of pages: 8 str.
• Numbering: Vol. 9, iss. 2, art. no. e71534
• PID: 20.500.12556/RUL-178481
• UDC: 614
• ISSN on article: 2398-8835
• DOI: 10.1002/hsr2.71534
• COBISS.SI-ID: 266509571
• Publication date in RUL: 28.01.2026

Record is a part of a journal

• Title: Health science reports
• Shortened title: Health sci. rep.
• Publisher: J. Wiley and Sons, Inc.
• ISSN: 2398-8835
• COBISS.SI-ID: 529715737

Licences

• License: CC BY-NC-ND 4.0, Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International
• Link: http://creativecommons.org/licenses/by-nc-nd/4.0/
• Description: The most restrictive Creative Commons license. This only allows people to download and share the work for no commercial gain and for no other purposes.

Secondary language

• Language: Slovenian
• Keywords: zdravstvo, vrednotenje zdravstvenih tehnologij, Evropa

Projects

• Funder: EC - European Commission
• Project number: 825162
• Name: Next Generation Health Technology Assessment to support patient-centred, societally oriented, real-time decision-making on access and reimbursement for health technologies throughout Europe
• Acronym: HTx