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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. 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. 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.

Additional file 3.

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Additional file 1. Survey voting results (charts).

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Outcome-based reimbursement models can effectively reduce the financial risk to health care payers in cases when there is important uncertainty or heterogeneity regarding the clinical value of health technologies. Still, health care payers in lower income countries rely mainly on financial based agreements to manage uncertainties associated with new therapies. We performed a survey, an exploratory literature review and an iterative brainstorming in parallel about potential barriers and solutions to outcome-based agreements in Central and Eastern Europe (CEE) and in the Middle East (ME). A draft list of recommendations deriving from these steps was validated in a follow-up workshop with payer experts from these regions. 20 different barriers were identified in five groups, including transaction costs and administrative burden, measurement issues, information technology and data infrastructure, governance, and perverse policy outcomes. Though implementing outcome-based reimbursement models is challenging, especially in lower income countries, those challenges can be mitigated by conducting pilot agreements and preparing for predictable barriers. Our guidance paper provides an initial step in this process. The generalizability of our recommendations can be improved by monitoring experiences from pilot reimbursement models in CEE and ME countries and continuing the multistakeholder dialogue at national levels.

Additional file 1: Table S1. Example cases for the different repurposing models (based on the IQVIA report) [17]. Table S2. Search syntax for the systematic literature review. Table S3. Illustrative benefits of value added medicine test cases according to the domains of the core evaluation framework as reported by the IQVIA report [17]. Table S4. A non-exhaustive list of value propositions in each value domain.

Interview Guide and Questionnaires of the EQUIPT Stakeholder Survey. (DOCX 86 kb)

The Map for Funding Orphan Medicinal Products in 8 EU Member States in 2015. (DOCX 63 kb)

Table S1. Distribution of stakeholders in each country. Table S2. Perceived availability and importance of tobacco-control interventions across countries. (DOC 278 kb)

Includes the detailed literature search strategy in PudMed and Scopus databases. Furthermore, the document contains the predefined exclusion criteria for Title-Abstract screening of the records. (DOCX 15 kb)

Objectives: Methodological challenges in the evaluation of medical devices (MDs) may be different for early and late technology adopter countries, as well as the potential health technology assessment (HTA) solutions to tackle them. This study aims to provide guidance to Central and Eastern European (CEE) countries on how to address key challenges of HTA for MDs with special focus on the transferability of scientific evidence. Methods: As part of the COMED Horizon 2020 project, a comprehensive list of issues related to MD HTA were identified based on a targeted literature review. Health technology assessment issues which pose a greater challenge or require different solutions in late technology adopter countries were selected. Draught recommendations to address these issues were developed and discussed in a focus group. The recommendations were then validated with a wider group of experts, including HTA and reimbursement decision makers from CEE countries in May and June 2020. Results: A consolidated list of 11 recommendations were developed in 3 major areas: (1) clinical value assessment, focusing on the use of joint EU work, relying on real-world evidence, use of coverage with evidence development schemes, transferring evidence from foreign countries and addressing the challenges of learning curve and centre effect; (2) economic value assessment, covering cost calculation of complex medical devices and transferability of economic evaluations of MDs; (3) HTA processes, related to the frequent product modifications and various indications of MDs. Conclusions: Central and Eastern European countries with limited resources for conducting HTA, can benefit from HTA methods and evidence generated in early technology adopter countries. Considering the appropriate reuse of international HTA materials, late technology adopter countries can still implement HTA, even for MDs, which have a more limited evidence base compared with pharmaceuticals.

SELFIE Interview Protocols. Standardized protocols for the semi-structured interviews in the SELFIE project. (PDF 1666 kb)

SELFIE Interview Protocols. Standardized protocols for the semi-structured interviews in the SELFIE project. (PDF 1666 kb)

European governments employ sophisticated health technology assessment and regulatory procedures to identify which pharmaceuticals to fund publicly. However, there are persisting demands from patients for those drugs excluded from positive reimbursement lists, leading to the emergence of what are here termed “alternative access schemes”. This paper presents a purposive review of these schemes based on available scholarly and grey literature, illustrated with real-world examples from recent practice. It puts forward an original typology of alternative access schemes based on their marketing authorization (regulation) and reimbursement (redistribution) status. We describe the complex, multidimensional policy trade-offs between the principles of patient freedom of choice, clinical autonomy, encouragement of innovation, evidence-informed decisions on safety and quality, access to treatment, and financial sustainability, involved in marketing authorization and reimbursement decisions. We discuss the ways in which alternative access schemes differ and conclude that our typology can illuminate salient policy dilemmas raised by alternative access schemes in national drug reimbursement systems.