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Summary of FDA Draft Guidance: Factors to Consider Regarding Benefit-Risk in Medical Device Product Availability, Compliance, and Enforcement Decisions

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By Daniel S. Wittenberg and Tim Scalo[1]

On June 16, 2016, the Food and Drug Administration (FDA) released a draft guidance entitled Factors to Consider Regarding Benefit-Risk in Medical Device Product Availability, Compliance, and Enforcement Decisions[2].  The non-binding draft guidance provides clarity to FDA staff and the medical device industry by setting out the benefit and risk factors the FDA may consider for decisions concerning product availability, compliance, and enforcement.  Although these decisions are generally fact specific, the FDA hopes that describing the factors will lead to a more consistent and transparent approach that focuses on maximizing patient benefits, reducing patient risks, and improving medical device quality.

Scope of the Draft Guidance

The draft guidance applies to diagnostic and therapeutic medical devices, subject to, and exempt from, premarket review. It does not apply to: 1) devices regulated by the FDA’s Center for Biologics Evaluation and Research, 2) combination products as defined in 21 CFR 3.2(e) for which the Center for Devices and Radiological Health is not the lead center, 3) electronic products that are not devices, and 4) premarket submission review decisions.  The draft guidance is intended to compliment, not supplant, other guidance regarding benefit-risk assessments.

The factors set forth in the draft guidance are applicable to both industry and FDA decisions.  Manufacturers should consider the factors when evaluating appropriate responses to nonconforming product or regulatory noncompliance issues, such as determining whether to limit the availability of a medical device.  The FDA may consider the factors when assessing device shortage situations, evaluating recalls, and selecting the appropriate engagement mechanism following an inspection revealing nonconforming products or regulatory noncompliance.

Patient Focused Benefit-Risk Assessments

The FDA has the ability to limit the availability of violative medical devices through compliance and enforcement actions.  The agency has recognized that, to achieve its ultimate goal of protecting and promoting public health, compliance and enforcement decisions need to be made while focusing on the impact to patients.  Failure to consider patient impact can result in actions with unintended adverse consequences, such as a shortage of medically necessary devices.

When available, the FDA may consider relevant, reliable information regarding patients’ perspectives on what constitutes meaningful benefit, what constitutes risk, and what tradeoffs patients are willing to accept.  The FDA may also consider benefits and risks for non-patient medical device users, such as caregivers and healthcare providers.

Benefit-Risk Factors to Consider

The FDA enumerated three categories of factors to consider in a benefit-risk assessment: 1) benefit factors, 2) risk factors, and 3) additional factors.  When prioritizing compliance and enforcement efforts to maximize medical device quality and patient safety, the FDA considers relevant and reliable evidence and data in accordance with the factors.  The factors should be considered in the aggregate and applied on a case-by-case basis.

Benefit-risk assessments may change over time as more information is learned about the device. For example, once a device is placed on the market, new uses may be found for a device (increasing its benefit), or nonconformities may be identified (increasing its risk).  The FDA may compare current benefit-risk assessments to benefits and risks identified during premarket review to understand whether there has been a change.

Examples of Benefit Factors considered include:

  • 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

All devices have some level of anticipated risk, even in the absence of nonconformities or regulatory noncompliance. The following risk factors take into account considerations related to: 1) nonconforming devices because these devices may directly introduce and increase risk; 2) regulatory noncompliance because noncompliance may be an indicator of a manufacturer’s inability to consistently produce quality devices, even if the device still performs as expected; and 3) harm potentially unrelated to nonconformity or noncompliance because post-market data may show a risk is higher than anticipated, even when the device conforms and the manufacturer complied with applicable statutes and regulations.

Examples of Risk Factors considered include:

  • Risk severity (includes three levels and a duration component)
  • Likelihood of risk (includes three factors related to the number of patients at risk)
  • Nonconforming product risks
  • Duration of exposure to population
  • False-positive or false-negative results
  • Patient tolerance of risk
  • Risk factors for healthcare professionals or caregivers

Beyond the above-mentioned benefit and risk factors, the FDA may also consider the following Additional Factors:

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

How the FDA Considers Benefit-Risk Factors in Patient Focused Decisions

An FDA benefit-risk assessment is triggered by an event, such as a recall, variance petition, or a nonconformity that may lead the FDA to take regulatory action.  First, the FDA will assess the benefit information in accordance with the factors.  The FDA will then evaluate the risk information.  Finally, the FDA will consider information relevant to the additional factors.

Decisions Affecting Medical Device Availability

The FDA plans to use the outcome of benefit-risk assessments to inform decisions related to product availability.  Benefit-risk assessment outcomes may be useful in the following decisions related to product availability:

  • When should a recall include a correction rather than a removal?
  • What actions may the FDA take when continued access to a nonconforming device or a device manufactured by a noncompliant company may be needed during a shortage situation?
  • When might the FDA exercise discretion and not take immediate action?

The draft guidance does not explain how the factors are weighted or evaluated. Generally, however, if a device has a high benefit and a low risk, the FDA will likely conclude that it is appropriate for patients to have continued access to the nonconforming device while long-term corrective action is taken, if alternative treatments are not available.  Conversely, if the device has a low benefit and a high risk, the FDA will likely take action to reduce the product’s availability.

Decisions Not Affecting Medical Device Availability

The FDA may also use benefit-risk assessments to inform compliance and enforcement decisions that will not affect product availability.  Examples of these decisions include:

  • When might the FDA send a Warning Letter or Untitled Letter, as opposed to taking a more informal alternative approach?
  • Is a proposed correction strategy adequate given the assessment and mitigation activities?

In decisions that do not affect availability, the FDA may also consider: 1) whether patients will be impacted by a compliance or enforcement action, 2) the manufacturer’s regulatory history, and 3) steps taken by the manufacturer to address the issue, including mitigation activities and communication with the FDA.

Again, the FDA does not specify how much the benefits must outweigh the risks, but the draft guidance does provide some general instruction.  If a device is assessed to have a high benefit and low risk, the FDA may choose to work with the manufacturer to address the problems without initiating a formal compliance or enforcement action.  Alternatively, if the benefit is low and the risk is high, the FDA will likely take formal action to address the issue.

Examples and Worksheets

The FDA included examples and worksheets in the draft guidance.  The examples discuss hypothetical, or simplified real world, FDA decisions.  While the examples are not predictive, they do demonstrate how the FDA considers the enumerated benefit-risk factors.  The worksheets list the factors and should be referenced during benefit-risk assessments.


The draft guidance outlines benefit-risk factors for the medical device industry and FDA staff to consider when making decisions related to product availability, compliance, and enforcement.  The FDA will consider stakeholder comments regarding the draft guidance that are submitted by September 14, 2016.

Manufacturers should understand the FDA’s process for benefit-risk assessments.  Manufacturers can advocate for favorable FDA decisions by using the enumerated factors to illustrate the high benefits and low risks of the company’s devices.


[1] Dan Wittenberg is a Partner in Snell & Wilmer L.L.P. and Co-Chair of the firm’s Pharmaceutical and Medical Device Industry Group.  Tim Scalo is a summer associate in the firm’s Denver office.

[2] FDA, Draft Guidance for Industry and FDA Staff: Factors to Consider Regarding Benefit-Risk in Medical Device Product Availability, Compliance, and Enforcement Decisions (Jun. 16, 2016).