Evidence map›Paper›PMID 41608382›Full record

ArticleHealth science reports2026

Transferability of Real World Evidence to Support HTA Recommendations in Lower Income European Countries.

Zoltán Kaló, Guenka Petrova, Bertalan Németh, Petra Došenović Bonča, Diana Delnoij, Alastair Bennett, Konstantin Tachkov, Yingying Zhang, Noemi Kreif, Saskia Knies and 16 more

Abstract read
In one paragraph

Article in Health science reports, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

3 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
  3. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

26 authors.

Zoltán KalóCenter for Health Technology Assessment Semmelweis University Budapest Hungary.ORCID https://orcid.org/0000-0001-7762-2607
Guenka PetrovaDepartment of Organization and Economics of Pharmacy, Faculty of Pharmacy Medical University of Sofia Sofia Bulgaria.ORCID https://orcid.org/0000-0001-8116-5138
Bertalan NémethSyreon Research Institute Budapest Hungary.
Petra Došenović BončaSchool of Economics and Business University of Ljubljana Ljubljana Slovenia.ORCID https://orcid.org/0000-0002-0356-1204
Diana DelnoijNational Health Care Institute Diemen The Netherlands.
Alastair BennettCentre for Health Economics University of York York UK.
Konstantin TachkovDepartment of Organization and Economics of Pharmacy, Faculty of Pharmacy Medical University of Sofia Sofia Bulgaria.
Yingying ZhangCentre for Health Economics University of York York UK.
Noemi KreifCentre for Health Economics University of York York UK.
Saskia KniesNational Health Care Institute Diemen The Netherlands.
Iga LipskaAcademy for Medical and Social Applied Sciences Gdańsk Poland.
Tomas TesarDepartment of Organisation and Management in Pharmacy, Faculty of Pharmacy Comenius University Bratislava Slovakia.
Zornitsa MitkovaDepartment of Organization and Economics of Pharmacy, Faculty of Pharmacy Medical University of Sofia Sofia Bulgaria.
Johan PontenThe Dental and Pharmaceutical Benefits Agency (TLV) Stockholm Sweden.
Anders VibergThe Dental and Pharmaceutical Benefits Agency (TLV) Stockholm Sweden.
Marian-Sorin PaveliuDepartment of Pharmacology Titu Maiorescu University Bucharest Romania.
Oresta PiniazkhoHTA Department of State Expert Centre Ministry of Health Kyiv Ukraine.
Adina Turcu-StiolicaDepartment of Pharmacoeconomics University of Medicine and Pharmacy of Craiova Craiova Romania.
Tomáš DoležalDepartment of Pharmacology, Faculty of Medicine Masaryk University Brno Czechia.
Michal StaňákNational Institute for Value and Technologies in Healthcare Bratislava Slovakia.
Alima AlmadiyevaSalidat Kairbekova National Research Center for Health Development Astana Kazakhstan.
Rok HrenSyreon Research Institute Budapest Hungary.
Dinko VitezićFaculty of Medicine University of Rijeka and University Hospital Centre Rijeka Rijeka Croatia.
Zsuzsanna PetykóCenter for Health Technology Assessment Semmelweis University Budapest Hungary.
Wim GoettschNational Health Care Institute Diemen The Netherlands.ORCID https://orcid.org/0000-0002-8022-7496
Andrea MancaCentre for Health Economics University of York York UK.ORCID https://orcid.org/0000-0001-8342-8421

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

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.

Indexed as

health technology assessmentlower income European countriesreal world evidencetarget trial emulationtransferability

Identifiers

PMID41608382
PMCPMC12836872

What OpenQuestion holds

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LicenceCC BY-NC-ND
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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.