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Final Guidance: Factors to Consider Regarding 

Benefit-Risk in Medical Device Product 

Availability, Compliance and Enforcement 

Decisions

Ann Ferriter

Director

Division of Analysis and Program Operations

Office of Compliance (OC)

Center for Devices and Radiological Health (CDRH)

February 9, 2017 CDRH Webinar
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Benefit-Risk

and what you need to know about it.

Focuses on patients

• OC strives to make compliance and enforcement 

decisions that put the patient first and honor CDRH’s 

mission to protect and promote public health. We do this 

by considering the relationship between potential 

benefits and risks.

Promotes Consistency

• Though we already assess benefit and risk, this guidance 

provides us with a framework to consistently and 

systematically apply these principles to our decision 

making, within the Center and in the field. This 

consistency will help improve our work. 
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Benefit-Risk

and what you need to know about it.

Interfaces with Industry

• Benefit-risk allows us to engage firms with a risk-based and 

situational approach. Externally, it facilitates the conversation 

and changes the focus from sole compliance to quality, 

patient benefit, and consideration of patient preferences. 

Encourages New Thinking 

• We will customize approaches to address issues, and use 

creative problem solving. This will require a culture change. 

Aligns CDRH 

• Aligns CDRH with mutual support and a shared understanding 

across divisions, we can maximize patient benefit, reduce 

patient risks, and improve overall medical device quality. 
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Key Points from the

Benefit-Risk in Medical Device Product 

Availability, Compliance and 

Enforcement Decisions Guidance

1. Complements the premarket Benefit-Risk Guidance 

documents 

2. Overarching for a wide variety of decisions 

3. Inform Compliance and Enforcement Decisions to 

Maximize Patient Benefit
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Guidance Contents

I. Introduction

II. Scope

III. Patient Focused Benefit-Risk Assessments 

IV. Description of Factors to Consider 

V. How the FDA Considers Benefit-Risk

VI. Examples 

Appendix A: Intersection with International Organization for 

Standardization (ISO) 14971

Appendices B-D: Work Sheets

http://www.fda.gov/downloads/medicaldevices/deviceregulatio

nandguidance/guidancedocuments/ucm506679.pdf
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I. Introduction 

Why?

• Provide clarity for FDA staff and industry 

regarding the benefit and risk factors 

• Maximize medical device quality and 

patient safety

How will the FDA implement?

• Pilots and other evaluation techniques 

• Harmonize with premarket benefit-risk 

assessment 

• Include a patient focus and use of Real 

World Evidence 
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II. Scope 

The FDA may consider benefit-risk factors during:

• Evaluation of device shortage situations

• Selection of the appropriate regulatory engagement mechanism 

• Evaluation of recalls

• Petitions for variance from sections of Quality Systems (QS) 

regulation (21 CFR part 820) for which there were inspectional 

observations during a Premarket Approval (PMA) inspection. 

This guidance:

• Applies to diagnostic and therapeutic devices 

• Excludes CBER devices, combination products where CDRH is not 

the lead and Electronic Product Radiation Control products and 

other products regulated by FDA
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III. Patient Focused Benefit-Risk 

Assessment

The FDA has the authority to limit the availability 

of violative medical devices and to pursue other 

compliance and enforcement actions related to 

violative medical devices— 

decisions regarding these actions should be 

made while focusing on the impact for 

patients 
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IV. Description of Factors to Consider

• Factors for the Assessment of Medical Device Benefits 

– Type of benefit

– Magnitude of benefit

– Likelihood of patients experiencing one or more benefits

– Duration of effect

– Patient perspective on benefit

– Benefit factors for healthcare professionals or caregivers 

– Medical necessity
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IV. Description of Factors to Consider

• Factors for the Assessment of Medical Device Risks 

– Severity of harm

– Likelihood of risk

– Distribution of nonconforming devices

– Duration of exposure to population

– False-positive or false-negative results

– Patient tolerance on risk

– Risk factors for healthcare professionals or caregivers
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IV. Description of Factors to Consider

• Additional Benefit-Risk Factors

– Uncertainty 

– Mitigation

– Detectability

– Failure mode

– Scope of the device issue

– Patient impact

– Preference for availability

– Nature of violations or nonconforming product 

– Firm compliance history
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V. How the FDA Considers Benefit-Risk 

• A Benefit-Risk evaluation is prompted by events that may 

lead the FDA to take regulatory action

• The FDA evaluates available benefit information from 

various sources

– Manufacturers may provide information through the 

designated FDA point of contact (e.g. recall coordinator)

• The FDA evaluates risk information from various sources

• The FDA complete s a benefit-risk assessment 

considering additional factors

• The FDA uses the outcome of a benefit-risk assessment 

to inform decisions
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High benefit, little risk Low benefit, high risk

Product 

Availability

Ensure patients have 

access to the device

Limit product availability

Compliance 

and 

Enforcement

Work with the 

manufacturer to 

address underlying 

issues

Take formal compliance 

or enforcement action

V. How the FDA Considers Benefit-Risk 

How might the FDA use benefit-risk to determine whether to take 

a regulatory or non-regulatory approach?
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Examples Related to Product 

Availability Decisions

Example 1: Recall and potential shortage of a 

high benefit implantable coated device with 

low additional risk

Example 2: Evaluation of a variance petition 

related to a high benefit drug delivery system 

for which risks posed by QS issues were 

sufficiently mitigated

Example 3: Continued access to 

nonconforming biological indicators with 

high benefit and mitigated additional risk

Example 4: Malfunction of a pregnancy test 

with low benefit and moderate additional risk

Example 5: Recall of a radiation therapy 

device with high benefit and increased risk 

for some patients

Examples Related to Compliance 

and Enforcement Decisions

Example 1: Evaluating whether to send 

a Warning Letter or take an alternative 

approach for a low benefit, low 

additional risk aesthetic device

Example 2: Evaluation of potential 

actions following an inspection with 

observed QS deficiencies regarding a 

high benefit spinal fixation system

VI. Examples 
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Appendices B-D Worksheets

How is a benefit-risk assessment performed?

Benefit

• How does the device 

benefit the patient?

• What is the patient’s 

perspective on 

benefit?

• Is the device 

medically necessary?

Risk

• What is the severity of 

the risk?

• What is the likelihood 

of harm?

• Were nonconforming 

devices distributed?

• Will patients tolerate 

the risks? 

Other factors

• What is the impact to 

the patient?

• What was the nature 

of the violation or 

nonconformity?

• What is the firm 

compliance history?
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Benefit 

• How does the device benefit the patient?

• Using real world data or other available data, what is the medical 

device’s impact on clinical management and patient health?

• Does the marketed product achieve the anticipated benefits?

• Has real world practice led to new benefits?

• Using real world data or other available data, what proportion of 

patients have been observed to benefit from the device?

• What is the patient’s perspective on benefit?

• What is the severity of the disease state?

• Is this a chronic disease?

• If chronic, can the illness be managed with other treatments or 

therapies?

• How long do patients live with the disease?

• Even if the benefit is in a small portion of the population, do those 

patients who would experience the benefit value it?
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Benefit 

• Is the device medically necessary?

• Is the device essential to the survival of patients?

• Are alternative treatments available?

• What other therapies are available for this condition?

• How effective are the alternative treatments?
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Risk

• What is the severity of the risk?

• Do real world data or other available data show that medical device-related 

injuries have occurred at expected severity?

• Are there any unanticipated injuries ?

• Were there any changes in variations in serious adverse events among 

subpopulations?

• Is the duration of harmful events longer than anticipated?

• Is the harmful event reversible?

• Has the type of intervention needed to address the harmful event changed?

• What is the likelihood of harm?

• How frequently does this specific failure mode or defect occur?

• What proportion of patients treated with or diagnosed by the nonconforming 

medical device is harmed?

• How many patients were exposed to nonconforming devices?
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Risk

• Were nonconforming devices distributed?

• What is the number of units on the market and market share?

• Will patients tolerate the risks?

• What level of concern do patients have regarding the risks?

• Even if the risk is in a small portion of the population, do those patients who 

would experience the risk understand it?

• Are patients willing to take the risk of this treatment to achieve the benefit?
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Other Factors

• How much uncertainty exists?

• What information does FDA have to assess benefit and risk?

• What is the quality of the information FDA is using (for example, MDRs, literature, 

registry or clinical trial data, limited case studies, inspectional data etc.)? Is the 

quality of information a reliable source for making an objective and unbiased 

benefit or risk decision?

• What is the impact on the patient?

• What are the risks to patients if the device is not available?

• Are patients better off if the device is available?

• What is the potential impact on patients related to the inspectional observation 

or regulatory non-compliance? 
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Other Factors

• What is the nature of the violation or nonconformity?

• What is the firm's compliance history?

• Has the same or a similar inspectional observation or regulatory 

violation been observed at the manufacturer in the past 2 years? In 

the past 5 years? In the past 10 years?

• Is the regulatory non-compliance significant enough that FDA would 

take regulatory action?

• When did the firm report the harm to the FDA?

• Would providing notice to the firm assist in informing the firm of its 

legal responsibilities?
Questions?

Division of Industry and Consumer Education: 

DICE@fda.hhs.gov

Slide Presentation, Transcript and Webinar Recording 

will be available at:

http://www.fda.gov/training/cdrhlearn

Under the heading: Postmarket Activities; 

Sub-heading: General Policy Heading