Gaps in the evidence underpinning high-risk medical devices in Europe at market entry, and potential solutions.

AIM: To determine the level of evidence for innovative high-risk medical devices at market entry. METHODS: We reviewed all Belgian healthcare payer (RIZIV-INAMI) assessor reports on novel implants or invasive medical devices (n = 18, Class IIb-III) available between 2018 to mid-2019 on applications submitted for inclusion on their reimbursement list. We also conducted a review of the literature on evidence gaps and an analysis of relevant legal and ethical frameworks within the European context. FINDINGS: Conformity assessment of medical devices is based on performance, safety, and an acceptable risk-benefit balance. Information submitted for obtaining CE marking is confidential and legally protected, limiting access to clinical evidence. Seven out of the 18 RIZIV-INAMI assessor reports (39%) included a randomized controlled trial (RCT) using the novel device, whilst 2 applications (11%) referred to an RCT that used a different device. The population included was inappropriate or unclear for 3 devices (17%). Only half of the applications presented evidence on quality of life or functioning and 2 (11%) presented overall survival data. Four applications (22%) included no data beyond twelve months. The findings from the literature demonstrated similar problems with the study design and the clinical evidence. DISCUSSION AND CONCLUSIONS: CE marking does not indicate that a device is effective, only that it complies with the law. The lack of transparency hampers evidence-based decision making. Despite greater emphasis on clinical benefit for the patient, the provisions of the European Medical Device Regulation (MDR) are not yet fully aligned with international ethical standards for clinical research. The MDR fails to address key issues, such as the lack of access to data submitted for CE marking and a failure to require evidence of clinical effectiveness. Indeed, a first report shows no improvement in the clinical evidence for implantable devices generated under the MDR. Thus, patients may continue to be exposed to ineffective or unsafe novel devices. The Health Technology Assessment Regulation plans for Joint Scientific Consultations for specific high-risk devices before companies begin their pivotal clinical investigations. The demanded comparative evidence should facilitate payer decisions. Nevertheless, there is also a need for legislation requiring comparative RCTs assessing patient-relevant outcomes for high-risk devices to ensure implementation, including development and implementation of common specifications for study designs.

Introduction of high-risk medical devices: national measures that can be taken under the current European legislation to put the patient interest central.

INTRODUCTION: High-risk medical devices may not always provide a therapeutic added value to patients. In Europe, no proof of efficacy is required to receive a CE label, making it difficult for policymakers to decide on reimbursement of (often expensive) high-risk medical devices. We explore, within the framework of the European legislation, the possibilities at a national level for a guided introduction of such devices. Areas covered: HTA and legal experts worked in close collaboration with medical specialists and government representatives making a legal analysis of what is possible under the (revised) European and national legislation. Expert commentary: At national level, measures for a better evidence-based introduction can be taken that are not in contradiction with the European regulation. From a legal point of view, all restrictive measures must be justified, necessary and proportional. Several measures are possible, a.o. making use of reference centres, applying the IDEAL framework or the 6-step plan set up by the Dutch Order of Medical Specialists. In conclusion, within the framework of the (revised) European legislation, measures at national level can be taken to temporarily restrict and follow up the use of high-risk medical devices with a greater focus on the therapeutic added value for the patients.