OBJECTIVES:

Healthcare decision-making for medical devices (MD) faces unique challenges, such as scarce trial data and less standardized clinical outcomes. Although regulatory agencies have attempted to increase the evidence bar for the assessment of MDs, reimbursement bodies fall behind in providing clear guidance. We reviewed assessment guidance documents on MDs with the aim to capture current methods and gaps and provide a critical outlook for the decade ahead.

METHODS:

We conducted a pragmatic review of guidance documents from regulatory agencies and payers around the world (Food and Drug Administration, European Medicines Agency, Health Canada, National Institute for Health and Care Excellence, Federal Joint Committee, Canadian Agency for Drugs and Technologies in Health, Institute for Clinical and Economic Review and others).

RESULTS:

Regulatory bodies have adjusted evidence requirements and criteria are now more stringent in the European Union (EU) for class III MDs. The reimbursement landscape for MDs is different; although decision-makers do not make a clear distinction between assessment criteria for MDs and pharmaceuticals, prior decisions on MDs have considered lower-quality evidence, indirectly recognizing the methodological challenges in generating data for MDs. However, this leads to uncertainty around evidence requirements for different device classes and inexplicit recognition of challenges and potential benefits unique to devices, such as the inability to trial blinding or formal incorporation of other outcomes specific to MDs in decision-making.

CONCLUSIONS:

Given the trend for increased scrutiny of MDs, manufacturers are facing challenges in raising evidence without clear methodological solutions and guidance on evidence standards from reimbursement bodies. Increased attention to real-world evidence may facilitate higher evidence standards by enabling better MD study designs, while respecting differences to pharmaceuticals, such as the possibility for MD evolution. Recognizing these challenges will be vital for efforts like the EU Joint Clinical Assessment to provide acceptable timely access for patients.