The FDA recently issued a final Guidance titled, "Factors
to Consider When Making Benefit-Risk Determinations in Medical
Device Premarket Approval and De Novo Classifications." The
purpose of the Guidance is "to provide greater clarity for FDA
reviewers and industry regarding the principal factors FDA
considers when making benefit-risk determinations during the
premarket review process for certain medical devices." The
Guidance applies to diagnostic and therapeutic devices undergoing
premarket approval (PMA) or de novo classification; that is, it
does not apply to the 510(k) process for determining
"substantial equivalence" to an existing and lawfully
Section 513(a) of the Federal Food, Drug & Cosmetic Act
requires the FDA to determine whether PMA applications provide a
"reasonable assurance of safety and effectiveness" by
"weighing any probable benefit to health from the use of the
device against any probable risk of injury or illness from such
use," among other relevant factors. This Guidance summarizes
the factors the FDA considers when making the risk-benefit
determination and also contains several hypothetical examples to
illustrate the process. Appendices to the Guidance include the
worksheet the FDA uses during the review to summarize its analysis
and sample worksheets based on the hypothetical examples. The
Guidance, including the examples, seeks "to improve the
predictability, consistency, and transparency of the review process
for applicable devices" "[b]y providing greater clarity
about FDA's decision-making process."
The Guidance identifies the following factors the FDA considers
when assessing the extent of the probable benefit(s) offered by a
The type of benefit(s)
The magnitude of the benefit(s)
The probability a patient will experience the benefit(s)
The duration of the benefit(s)
To assess the extent of the probable risks, the FDA considers
the following factors:
Severity, types, number and rates of harmful events associated
with the use of the device, including:
Device-related serious adverse events
Device-related non-serious adverse events
Complications from procedures
Probability of a harmful event
Duration of a harmful event
Risk of false-positive or false-negative results for
The Guidance also mentions the following additional factors that
the FDA takes into account in this process:
The degree of certainty of the benefits and risks of a
Characterization of the disease
Patient tolerance for risk and perspective on benefit
Availability of alternative treatments or diagnostics
Novel technology addressing unmet medical need
Each of the risk, benefit, and additional factors is accompanied
by additional explanation. The Guidance also includes discussion of
the types of scientific evidence FDA will accept when weighting
Those interested in more details are advised to consult the
Guidance, a pdf copy of which is available here.
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guide to the subject matter. Specialist advice should be sought
about your specific circumstances.
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