Collecting real-world data (RWD) and generating real-world evidence (RWE) from it are becoming more important for making better decisions in healthcare. Central and Eastern European (CEE) countries are often late adopters of novel health technologies, therefore, their HTA systems could benefit from transferring data and evidence including RWD, RWE, and methods from other countries, mainly from Western Europe. But in practice, this rarely happens. Against the backdrop of this, the ISPOR CEE Consortium hosted the webinar titled “Transferability of Data and Methods to Central and Eastern Europe” on September 19, 2024. Dr. Oresta Piniazhko, Chair of ISPOR CEE Consortium moderated the webinar. The session focused on discussing the following questions: 1) What main barriers prevent transferring data and methods in CEE, originally generated in Western Europe? 2) What are the proposed solutions to overcome these barriers and opportunities
The key messages from the speakers are summarized as below
Dr. Boryana Ivanova, MPharm, Head, Health Technology Assessment, National Council on Prices and Reimbursement of Medicinal Products (NCPR), Sofia, Bulgaria presented on the process of collecting RWD and generating RWE from it in Bulgaria and cases on using RWE for HTA recommendations and decision-making in Bulgaria.
National Council on Prices and Reimbursement of Medicinal Products (NCPR) is the Bulgarian national competent body on HTA responsible for monitoring the effect of drug therapy – the process of collecting and analyzing RWD and RWE.
Monitoring the effect of drug therapy is conducted in healthcare establishments, which regularly provide data on each specific medicinal product from the hospital information system to the NCPR central server. Using a special Platform - a Big Data and AI system that integrates and processes real-world data from electronic health records, registers, etc., we evaluate the efficacy of high-cost innovative medicinal products in real medical practice. NCPR has an annual obligation to prepare a report on the collected and analyzed RWD and RWE of the medicinal products designated for monitoring.
The process of monitoring the effect of drug therapy allowed for gaining valuable experience in data processing and collecting sufficiently meaningful and qualitative data on the treatment of Bulgarian patients. This resulted in the first publication with RWD concerning the effectiveness of ribociclib in patients with breast cancer treated in routine Bulgarian clinical practice. This analysis found that patients with comparable characteristics use ribociclib in the real world similarly to what has been investigated in the RCTs. The analysis involved utilizing deep learning natural language processing algorithms (NLPs) to extract and normalize clinical data from electronic health records (EHRs) of breast cancer patients who were undergoing ribociclib treatment. In addition, an abstract for real-world PFS outcomes of Bulgarian patients with melanoma has recently been accepted to be presented at ISPOR Congress Europe 2024. These publications serve as a basis for the development of further publications in the field of oncology and rare diseases.
The process of collecting and analyzing RWD and RWE and conducting HTA is among NCPR’s main responsibilities, which allows paying institutions to draw informed conclusions. Evidence-based decision-making enables the responsible authorities to choose the best therapeutic treatment for patients and society.
Bertalan Nemeth, PhD, Principal Health Economist, Syreon Research Institute, Budapest, Hungary presented the key findings from the European Union’s Horizon 2020 funded HTx research project on next generation HTA, during which these questions were investigated.
Dr. Nemeth 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 that prevent Central and Eastern European (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.
Dr. Nemeth 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.
Oresta Piniazhko, PhD, Director of HTA Department of SEC of Ministry of Health, Kyiv, Ukraine and Chair of ISPOR CEE Consortium, shared her experience in applying transferability for methodological guidelines development in Ukraine from the HTA body perspective to optimize the assessment procedures and act in time.
Dr. Piniazhko presented an overview of the current HTA roadmap in Ukraine: legislation, processes and methods, aim of national HTA according to legal framework Decree by the Cabinet of Ministers of Ukraine №1300 “On the approval of the procedure for the state HTA” on December 23, 2020, with amendments in 2024. In particular highlighted new updated of HTA guideline for medicines in 2023, amendments approved by the Order of MOH № 1741 dated 06.10.2023, which was updated based on adaptation of best international practice to local settings, and recommendation for cost-effectiveness threshold ICER and budget impact test in Ukraine.
Oresta noted the results of over 100 HTA conclusions with recommendations performed for the Ministry of Health of Ukraine, the introduction of managed entry agreements (MEA) in Ukraine 2022-2024. As of September 2024, among 108 publicly available HTA conclusions, 38% had recommended considering a MEA. The first 12 MEAs concluded in 2022. A new service was introduced in 2024- scientific advice before HTA submission, also patient engagement and public communication of project of HTA conclusions. Also a case study conducted in 2022 on RWE used in the HTA submissions and conclusions in Ukraine showed 19% of HTA recommendations based on company submissions included RWE.
RWE was consistent with the results of randomized clinical trials (RCT) of medicines in oncology and the medicines used to prevent cytomegalovirus (CMV) reactivation and disease in CMV-seropositive recipients of allogeneic hematopoietic stem cell transplantation. There were recommendations to collect RWD in the local settings among the published HTA conclusions. For example, RWE for antibiotics plays a crucial role in filling gaps in evidence that is not addressed by traditional approaches such as RCT, especially for reserve groups of antibiotics that are indicated for the treatment of pan-resistant microorganisms.
The use of RWE in HTA recommendations in Ukraine showed a prospective trend as a part of the additional information support of medicines’ assessment, which requires further analysis of transferability opportunities and evidence generation in local settings.