title: What type of clinical evidence is needed to assess medical devices?
abstract: 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.
authors: CERGAS, Centre for Research on Social and Healthcare Management, Bocconi University, Milan, Italy Dept of Policy Analysis and Public Management, Bocconi University, Milan, Italy rosanna.tarricone@unibocconi.it.
authors: Rosanna
authors: R
authors: Tarricone
authors: CERGAS, Centre for Research on Social and Healthcare Management, Bocconi University, Milan, Italy.
authors: Paola Roberta
authors: PR
authors: Boscolo
authors: CERGAS, Centre for Research on Social and Healthcare Management, Bocconi University, Milan, Italy.
authors: Patrizio
authors: P
authors: Armeni
mesh_terms: D006328:Cardiac Catheterization / Q000009:adverse effects / Q000191:economics / Q000295:instrumentation*; D003362:Cost-Benefit Analysis; D018795:Device Approval / Q000331:legislation & jurisprudence; D004059:Diffusion of Innovation; D004867:Equipment Design; D004868:Equipment Failure; D004869:Equipment Safety; D004864:Equipment and Supplies / Q000009:adverse effects / Q000191:economics; D019317:Evidence-Based Medicine / Q000191:economics / Q000331:legislation & jurisprudence; D017048:Health Care Costs; D006801:Humans; D062307:Medical Device Legislation; D008944:Mitral Valve Insufficiency / Q000175:diagnosis / Q000191:economics / Q000503:physiopathology / Q000628:therapy*; D011050:Policy Making; D012720:Severity of Illness Index; D016896:Treatment Outcome