The US Food and Drug Administration (FDA) on Tuesday finalized two guidance documents: one on factors to consider when making benefit-risk determinations for premarket approval (PMA) and de novo classifications, and one on the concepts that sponsors and other stakeholders should consider when choosing to collect patient preference information (PPI) that may inform FDA's benefit-risk determinations.
The 62-page guidance on benefit-risk determinations, finalized more than five years after the draft was proposed, looks to provide greater clarity for FDA reviewers and industry on the principal factors FDA considers when making benefit-risk determinations during the premarket review process for certain medical devices.
In assessing a device’s potential benefits, FDA considers the type of benefit, magnitude, probability of a patient experiencing such a benefit and the duration the benefit can be expected to last. In terms of risks, FDA evaluates the severity, type, number and rate of harmful events, the probability and duration of a harmful event and the risk from false-positive or false-negative results from diagnostics.
Additional factors considered include uncertainties (“there is never 100% certainty when determining reasonable assurance of safety and effectiveness of a device”), patient-centric assessments and patient-reported outcomes, characterization of the disease and other patient perspectives, which FDA says “should take into account both the patient’s willingness and unwillingness to use a device or tolerate risk in exchange for probable benefit.”
The guidance also includes four hypothetical examples of benefit-risk determinations, as well as appendices on the intersection of this guidance with ISO 14971, a worksheet for benefit-risk determinations and worksheets for the examples.
Patient Preference Information
The other guidance, a 29-page document finalized on Tuesday from a draft issued in May 2015, explains the concepts that sponsors and other stakeholders should consider when choosing to collect PPI, which may inform FDA's benefit-risk determinations in PMAs, humanitarian device exemption (HDE) applications, and de novo classification requests.
The guidance also discusses FDA's inclusion of PPI in its decision summaries and provides recommendations for the inclusion of such information in device labeling for certain devices.
The guidance is part of FDA’s interest in providing a systematic way to ensure that patients are represented and patient perspectives are considered in the decision making process.
Guidance for Industry and Food and Drug Administration Staff: Factors to Consider When Making Benefit-Risk Determinations in Medical Device Premarket Approval and De Novo Classifications
Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders: Patient Preference Information – Voluntary Submission, Review in Premarket Approval Applications, Humanitarian Device Exemption Applications, and De Novo Requests, and Inclusion in Decision Summaries and Device Labeling