FDA Clarifies Benefit/Risk Analysis

When the FDA Ortho Panel meets, the panel is not only asked to advise on safety and effectiveness, but also on whether or not the benefits of a new device outweigh the risks to patients.

On June 16, 2016, the agency issued a draft guidance titled, Factors to Consider Regarding Benefit-Risk in Medical Device Product Availability, Compliance, and Enforcement Decisions.

Draft Guidance

The agency said the purpose of the guidance is to provide a framework for FDA and stakeholders, “that sets forth overarching benefit-risk principles. FDA may consider the types of benefit-risk factors described in this draft guidance—including reliable patient preference information from a representative sample—on a case-by-case basis when determining the appropriate actions to take and to help ensure that informed and science-based decisions are made to the greatest extent practical.”

When finalized, the guidance will “harmonize” the FDA’s approach to “weighing benefits and risks for medical device product availability, compliance, and enforcement decisions with FDA’s benefit-risk framework for assessing medical device marketing and investigational device exemption (IDE) applications.”

The agency says the benefit-risk factors in this draft guidance also support assessment of medical devices with real world evidence. “While the benefit-risk factors in this draft guidance are not identical to the other frameworks, this draft guidance builds upon FDA’s premarket review benefit-risk policy in an effort to improve consistency in our patient centered approach and decision making across the total product life cycle.”

Considerations

According to FDA, when it comes to making compliance and enforcement decisions, the agency will consider any “relevant and reliable” information pertaining to the device in question, including “reliable patient preference information from a representative sample.”

In particular, FDA says it considers the following factors when considering a device’s benefit in light of compliance and enforcement decisions:

• Type of benefit(s)
• Magnitude of benefit(s)
• Likelihood of patients experiencing one or more benefits
• Duration of effects
• Patient preference on benefit
• Benefit factors for healthcare professionals or caregivers
• Medical necessity

For risks, FDA says it considers the following factors:

• Medical device-related deaths and serious injuries
• Medical device-related non-serious adverse events
• Medical device-related events without reported harm
• Duration of harm to patient

Likelihood of risk:

• Likelihood of medical device nonconformity
• Likelihood of a harmful event given exposure to a nonconforming device
• Number of patients exposed
• Nonconforming product risks
• Duration of exposure to population
• False-positive or false-negative result
• Patient tolerance risk
• Risk factors for healthcare professionals or caregivers

Additionally, FDA says it can take other factors into account as well, including:

• Uncertainty
• Mitigations
• Detectability
• Failure mode
• Scope of the device used
• Patient impact
• Preference for availability
• Nature of violations/Nonconforming product
• Firm compliance history

Submitting Comments

Stakeholders should submit comments and suggestions regarding this draft document within 90 days of publication in the Federal Register of the notice announcing the availability of the draft guidance. Submit electronic comments to http://www.regulations.gov. Submit written comments to the Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.

Click here to read the entire 29-page document.