The Use of Real-World Evidence in HTA in Central and Eastern Europe

Published Nov 6, 2023
  • Oresta Piniazhko, PhD, Chair of ISPOR CEE Consortium Executive Committee and Director of HTA Department of SEC of Ministry of Health, Kyiv, Ukraine
  • Bertalan Németh, PhD, Principal Health Economist of Syreon Research Institute, Budapest, Hungary
  • Maria Kamusheva, MPh, PhD, President-Elect of ISPOR Bulgaria Chapter, Head of Bulgarian Association of Pharmacoeconomics, and Professor of Department of Organization and Economics of Pharmacy, Faculty of Pharmacy, Medical University of Sofia, Bulgaria
  • Maciej Niewada, MD, PhD, Professor of Department of Clinical & Experimental Pharmacology Medical University of Warsaw and CEO of HealthQuest, Warsaw, Poland

Collecting real-world data (RWD) and generating real-world evidence (RWE) from it is becoming more important for making better decisions in healthcare. What are the main barriers which prevent using real-world evidence in Central and Eastern Europe (CEE), which was originally generated in Western Europe? What are the proposed solutions to overcome these barriers? To explore these important questions, the ISPOR CEE Consortium hosted a webinar on “The Use of Real-World Evidence in HTA in Central and Eastern Europe” on 13 September 2023.

Dr Oresta Piniazhko moderated the webinar and shared experience in use of RWE from the perspective of HTA national expertise in Ukraine. Dr Bertalan Németh presented on the research conducted on barriers and their solutions. Prof. Maria Kamusheva focused on the experience from a Central and Eastern European university perspective. Dr Maciej Niewada presented his views from an independent researcher perspective. The key messages from the speakers are summarized below.

Dr. Bertalan Németh explained that as part of the European Commission funded HTx H2020 (Next Generation Health Technology Assessment) project, a group of experts investigated the most important barriers, which prevent CEE countries from using RWE generated in Western Europe in their healthcare decision making and health technology assessment. Moreover, together with various stakeholders from the CEE region, they proposed solutions to overcome said barriers.

Bertalan showed that a scoping review was conducted, followed by a webinar, after which the most important barriers were selected through a survey. Finally, an international workshop was held with CEE experts to discuss the proposed solutions to the nine most important barriers.

Multiple solutions were proposed for all barriers. Dr. Németh highlighted three main themes emerging across the various pieces of practical advice: the need for international collaboration, the necessity of improving the knowledge base on RWD and RWE across all stakeholder groups, and finally, the political will that supports the use of RWD and RWE.

Suggested reading: Kamusheva M, Németh B, Zemplényi A, Kaló Z, Elvidge J, Dimitrova M, Pontén J, Tachkov K, Mitkova Z. Using real-world evidence in healthcare from Western to Central and Eastern Europe: a review of existing barriers. Journal of Comparative Effectiveness Research. 2022 Aug;11(12):905-13.

Németh B, Kamusheva M, Mitkova Z, Petykó ZI, Zemplényi A, Dimitrova M, Tachkov K, Balkányi L, Czech M, Dawoud D, Goettsch W. Guidance on using real-world evidence from Western Europe in Central and Eastern European health policy decision making. Journal of Comparative Effectiveness Research. 2023 Apr;12(4):e220157.

Dr. Maria Kamusheva focused on several main aspects related to RWE and RWD: (1) the difference between the two terms, (2) their importance, benefits and application in healthcare decision making and scientific research, (3) the issues recognized considering the specifics of the CEE regions for the use of RWD in pharmacoeconomic evaluations, (4) Bulgaria as a reference country regarding usage of RWD for scientific purposes, and (5) the main steps forward in improving, collecting and implementing RWD and RWE in CEE countries.

Maria emphasized that RWD and RWE provide complimentary evidence about effectiveness and safety of available treatment approaches. Because of the technological development and rapid digitalization processes in the healthcare sector, the volume of RWD is increasing tremendously. Scientists, clinicians, healthcare policy decision makers become more and more aware about RWD and apply it in their everyday work.

Despite its high value, a number of challenges about ensuring reliable RWE for HTA are identified. This may lead to disparities among different stakeholders when discussing RWD and RWE use in decision making. Moreover, a quality control process is required in order to ensure that the RWD collected is accurate, timely, and useful. It is also crucial to involve patients’ associations and societies in the process of collecting and understanding the importance and benefits of RWD.

Maria clarified how useful economic evaluation (EE) studies are and that RWD should be used as a source of data for EE. However, there are many challenges regarding standardization of methodology in EE using RWD. Two separate published systematic reviews identified a limited number of EE based on RWD but the trend is for converting it into an increasingly common practice. 

Dr Kamusheva presented a case with Bulgaria where a specifically designed RWD study to analyze the cost effectiveness of the available treatment approaches for patients with acromegaly was conducted. It was also highlighted that generating RWE is a resource intensive process, and CEE countries usually are short of financial and human resources.

The key take home messages were summarized by Maria: RWD and RWE have an increasing role in scientific research, more efforts are needed to increase the quality of RWD collected and united efforts of CEE courtiers in developing guidance and approaches on how to generate, use and analyze RWD are needed.

Dr Maciej Niewada shared insights into the Polish experience and perspective regarding the collection of RWD and the use of RWE to inform decision-making. He highlighted several key points. Firstly, he emphasized that RWD is highly valued by payers, clinicians, and other stakeholders. These data offer an opportunity to more effectively finance individualized and highly valuable therapies. In the context of having a single-payer in the market and a limited need to consolidate data from various sources, coupled with a relatively constrained private healthcare system, Poland has been able to collect extensive data that provides a comprehensive overview of current healthcare practices.

Maciej also noted the significance of the EU-funded project "Maps for Health Demand" (see, which has resulted in public data analyses and comprehensive findings shared in the public domain. The culture of data analysis is actively promoted by the National Health Fund, with in-house analyses and collaborative projects with universities and other scientific institutions. Furthermore, he highlighted the substantial impact of e-prescriptions, which proved highly beneficial during the Covid-19 pandemic. As of the end of December 2023, e-prescriptions covered a substantial 1.8 billion prescriptions issued. All these sources of RWD are commonly utilized to populate Health Technology Assessment (HTA) dossiers and guide pricing and reimbursement decisions.

It's worth noting that under the Medical Fund regulation, highly innovative technologies identified by the HTA Agency are provided with a fast reimbursement decision pathway and access. Renewal decisions, however, will be influenced by outcomes collected through dedicated clinical registries (market entry arrangements guided by real-world clinical outcomes).

Maciej also mentioned the challenges associated with the analysis of RWD. These challenges include issues like non-systematic access to non-consolidated data, formal limitations, regulatory constraints, and concerns about the quality of the collected data and the standards of analysis. Sharing and promoting best practices are essential steps in addressing these challenges effectively.

To view the full webinar recording, please visit here.
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