Skip to main content

Abstract

Aim: Real-world data and real-world evidence (RWE) are becoming more important for healthcare decision making and health technology assessment. We aimed to propose solutions to overcome barriers preventing Central and Eastern European (CEE) countries from using RWE generated in Western Europe. Materials & methods: To achieve this, following a scoping review and a webinar, the most important barriers were selected through a survey. A workshop was held with CEE experts to discuss proposed solutions. Results: Based on survey results, we selected the nine most important barriers. Multiple solutions were proposed, for example, the need for a European consensus, and building trust in using RWE. Conclusion: Through collaboration with regional stakeholders, we proposed a list of solutions to overcome barriers on transferring RWE from Western Europe to CEE countries.

Tweetable abstract

Research investigating ways to overcome barriers preventing Central and Eastern European countries from using Western European real-world evidence for healthcare decision making, using a multi-methods approach to create a list of solutions in collaboration with local stakeholders.

Plain language summary

Collecting real-world data and generating real-world evidence from it is becoming more important for making better decisions in healthcare. We investigated the main barriers which prevent using real-world evidence in Central and Eastern Europe, originally generated in Western Europe. After identifying the nine most important barrier, with the help of local experts we proposed solutions to overcome those barriers. Several possible solutions were proposed, many of them highlighting the need for a European consensus on these matters and building trust in new methods. Our results can hopefully serve as a guidance document to help overcome the barriers.

Formats available

You can view the full content in the following formats:

References

Papers of special note have been highlighted as: • of interest; •• of considerable interest
1.
O'Rourke B, Oortwijn W, Schuller T. The new definition of health technology assessment: a milestone in international collaboration. Int. J. Technol. Assess. Health Care 36(3), 187–190 (2020).
2.
Németh B, Goettsch W, Kristensen FB et al. The transferability of health technology assessment: the European perspective with focus on Central and Eastern European countries. Expert Rev. Pharmacoecon. Outcomes Res. 20(4), 321–330 (2020).
• Paper on the transferability issues in health technology assessment with a Central and Eastern European focus.
3.
Makady A, de Boer A, Hillege H, Klungel O, Goettsch W. What is real-world data? A review of definitions based on literature and stakeholder interviews. Value Health 20, 858–865 (2017).
• Review of definitions connected to the topic of the manuscript.
4.
Haute Autorité de santé (HAS). Real-world studies for the assessment of medicinal products and medical devices. www.has-sante.fr/upload/docs/application/pdf/2021-06/real-world_studies_for_the_assessment_of_medicinal_products_and_medical_devices.pdf
5.
US Department of Health and Human Services, Food and Drug Administration (FDA). Real-world data (RWD) and real-world evidence (RWE) are playing an increasing role in health care decisions. www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence
6.
National Institute for Health and Care Excellence (NICE). NICE real-world evidence framework. www.nice.org.uk/corporate/ecd9/resources/nice-realworld-evidence-framework-pdf-1124020816837
7.
Kamphuis B, Avouac B, Colomer R et al. RWE in Europe Paper V: Policy Challenges around Real World Evidence Adoption in Europe 2018. The London School of Economics and Political Science. (2018). www.lse.ac.uk/business/consulting/assets/documents/rwe-in-europe-paper-v.pdf
8.
Czech M, Jasiński Z, Krupa D. Real-life treatment patterns and medication costs in patients with hypertension treated with ramipril monotherapy or ramipril loose and fixed combinations in Poland. J. Health Pol. Outcomes Res. 2, 31–43 (2015).
9.
Kardas P, Lichwierowicz A, Urbański F et al. The potential to reduce patient co-payment and the public payer spending in Poland through an optimised implementation of the generic substitution: the win-win scenario suggested by the real-world big data analysis. Pharmaceutics 13(8), 1165 (2021).
•• Experience with using real-world evidence (RWE) in Poland, a Central and Eastern European country.
10.
Czech M, Boguslawski S, Smaga A, Filipiak KJ. Use of single pill combinations in the treatment of arterial hypertension in Poland: the current practice and guidelines, the impact on reimbursement spending and patient co-payment. Cardiol. J. 29(3), 405–412 (2022).
11.
Makady A, Ham RT, de Boer A, Hillege H, Klungel O, Goettsch W. GetReal Workpackage. Policies for use of real-world data in health technology assessment (HTA): a comparative study of six HTA agencies. Value Health 20(4), 520–532 (2017).
• A comparative study of RWE use in health technology assessment across Western European countries.
12.
Klonoff DC. The expanding role of real-world evidence trials in health care decision making. J. Diabetes Sci. Technol. 14(1), 174–179 (2020).
13.
Kamusheva M, Németh B, Zemplényi A et al. Using real-world evidence in healthcare from Western to Central and Eastern Europe – a review of existing barriers. J. Comp. Eff. Res. 11(12), 905–913 (2022).
•• An article explaining the previous steps of the current research.
14.
Justo N, Espinoza MA, Ratto B et al. Real-world evidence in healthcare decision making: global trends and case studies from Latin America. Value Health 22(6), 739–749 (2019).
15.
Szkultecka-Debek M, Kawalec P, Jahnz-Rózyk K. Real world evidence (RWE) and orphan drug policies in selected Central and Eastern European (CEE) countries. J. Health Pol. Outcomes Res www.jhpor.com/article/2213-real-world-evidence-rwe-and-orphan-drug-policies-in-selected-central-and-eastern-european-cee-countries
16.
Daubner-Bendes R, Kovács S, Niewada M et al. Quo Vadis HTA for medical devices in Central and Eastern Europe? Recommendations to address methodological challenges. Front. Public Health 8, 994 (2021).
17.
Ciani O, Wilcher B, Blankart CR et al. Health technology assessment of medical devices: a survey of non-European union agencies. Int. J. Technol. Assess. Health Care 31(3), 154–165 (2015).
18.
Lu N, Xu Y, Yue LQ. Good statistical practice in utilizing real-world data in a comparative study for premarket evaluation of medical devices. J. Biopharm. Stat. 29(4), 580–591 (2019).
19.
European Medicines Agency (EMA). Data Analysis and Real World Interrogation Network (DARWIN EU). www.ema.europa.eu/en/about-us/how-we-work/big-data/data-analysis-real-world-interrogation-network-darwin-eu
20.
Kristensen FB. Development of European HTA: from vision to EUnetHTA. Michael 9, 147–156 (2012).
21.
Willemsen A, Ettinger S, Helmink C, Erdos J, Hviding K, Ormstad SS. EUnetHTA relative effectiveness assessments: efforts to increase usability, transparency and inclusiveness. Int. J. Technol. Assess. Health Care 38(1), e41 (2022).
22.
Berger ML, Sox H, Willke RJ et al. Good practices for real-world data studies of treatment and/or comparative effectiveness: recommendations from the joint ISPOR-ISPE Special Task Force on real-world evidence in health care decision making. Value Health 20(8), 1003–1008 (2017).
23.
Wilkinson MD, Dumontier M, Aalbersberg IJ et al. The FAIR Guiding Principles for scientific data management and stewardship. Sci. Data 3(1), 1–9 (2016).
24.
European Health Data Evidence Network (EHDEN). European Health Data & Evidence Network. www.ehden.eu/ (2018).
25.
Kent S, Burn E, Dawoud D et al. Common problems, common data model solutions: evidence generation for health technology assessment. Pharmacoeconomics 39(3), 275–285 (2021).
26.
Hogervorst MA, Pontén J, Vreman RA, Mantel-Teeuwisse AK, Goettsch WG. Real world data in health technology assessment of complex health technologies. Front. Pharmacol. 13, 837302 (2022).
27.
Evans K. Real world evidence: can we really expect it to have much influence? Drugs Real World Outcomes 6(2), 43–45 (2019).
28.
Orsini LS, Berger M, Crown W et al. Improving transparency to build trust in real-world secondary data studies for hypothesis testing-why, what, and how: recommendations and a road map from the Real-World Evidence Transparency Initiative. Value Health 23(9), 1128–1136 (2020).
•• A joint RWE transparency initiative by two influential scientific societies.
29.
Vreman RA, Broekhoff TF, Leufkens HGM, Mantel-Teeuwisse AK, Goettsch WG. Application of managed entry agreements for innovative therapies in different settings and combinations: a feasibility analysis. Int. J. Environ. Res. Public Health 17(22), 8309 (2020).
30.
Kovács S, Kaló Z, Daubner-Bendes R et al. Implementation of coverage with evidence development schemes for medical devices: a decision tool for late technology adopter countries. Health Econ. 31(Suppl. 1), 195–206 (2022).