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

Orphanet Journal of Rare Diseases

Position Statement

Frank Hulstaert¹  Célia Primus-de Jong¹  Mattias Neyt¹  Céline Pouppez¹  Kathleen Harkin² 

[1] Administrative Centre Botanique, Belgian Health Care Knowledge Centre (KCE), Doorbuilding (10th floor), Boulevard du Jardin Botanique 55, B-1000, Brussels, Belgium;Centre for Health Policy and Management, Trinity College Dublin, 3-4 Foster Place, Room 0.18, Dublin, Ireland;

关键词: Medical devices;    Evidence gaps;    CE marking;    Reimbursement;    Declaration of Helsinki;    Comparator;    Study design;    Comparative evidence;   

DOI  :  10.1186/s13023-023-02801-7

 received in 2022-12-14, accepted in 2023-07-05,  发布年份 2023

来源: Springer

【 摘 要 】

AimTo determine the level of evidence for innovative high-risk medical devices at market entry.MethodsWe 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.FindingsConformity 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 conclusionsCE 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.

【 授权许可】

CC BY   
© Institut National de la Santé et de la Recherche Médicale (INSERM) 2023