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The objective of this mini-review is to discuss the role of real-world studies as a source of clinical evidence when experimental studies, such as randomised controlled trials (RCTs), are not available. Waiting for RCT evidence when the technology is diffusing could be anti-economical, inefficient from the policy perspective and methodologically questionable.We explain how real-world studies could provide relevant evidence to decision makers. Matching techniques are discussed as a viable solution for bias reduction.We describe a case study concerning a cost-effectiveness analysis based on real-world data of a technology already in use: Mitraclip combined with medical therapy versus medical therapy alone in patients with moderate-to-severe mitral regurgitation. The CEA has encountered the scepticism of most reviewers, due not to the statistical methodology but to the fact that the study was observational and not experimental. Editors and reviewers converged in considering real-world economic evaluations premature in the absence of a RCT, even if in the meantime the technology had been implanted >30 000 times. We believe there is a need to acknowledge the importance of real-world studies, and engage the scientific community in the promotion and use of clinical evidence produced through observational studies. @ERSpublications Real-world data are a valid complement and/or alternative to RCTs to support policy decisions on medical devices http://ow.ly/SHsZ300pfCB

Purpose: This paper presents a comparative analysis of value-based frameworks (VBFs) developed in the United States and Europe for both drugs and medical devices. Methods: The authors relied on a comprehensive literature review to identify relevant dimensions of analysis and available VBFs. Compared to previous studies on this topic, this study focused on the roles and strategic objectives of different institutions and organizations currently orchestrating the debate and development of VBFs and value-based initiatives. Findings: Among several projects and initiatives aimed at measuring value in health care, the study identified and analyzed in detail 7 VBFs. Different frameworks not only reflect different conceptions of value, but also different strategic objectives for which value needs to be measured: influencing clinical decision making, informing health care policies, affecting reimbursement, and pricing mechanisms or driving industry developments. Ultimately, all of these objectives represent different levers for fostering value-based treatment choices, but their different routes imply the assignment of key responsibilities to different actors within health care: medical professionals, patients, policymakers, payers, and medical technology and pharmaceutical companies. Implications: The proliferation of frameworks used for measuring the value of different health care objects and processes seems certainly a worthy exercise, but a real paradigm shift toward value-based care could only happen if these tools will be systematically used in practice and if all of these levers are played together, with joint actions and shared awareness of the key stakeholders' groups.

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