Patient Preference Information (PPI) in Medical Device Decision Making

Update: September 5, 2024 - The FDA issued draft guidance: Incorporating Voluntary Patient Preference Information over the Total Product Life Cycle. This guidance, when finalized, is intended to provide recommendations on how patient preferences can be collected and shared with the FDA and be considered in the decision-making process. 

Submit comments under docket number FDA-2015-D-1580 at www.regulations.gov by December 5, 2024 to ensure the FDA considers comments on this draft guidance before it begins work on the final version of the guidance.

The Center for Devices and Radiological Health (CDRH) recognizes that it takes many people to evaluate and communicate the benefits and risks of medical devices. However, only patients and their caregivers who have lived with their medical conditions and use medical devices understand the factors that impact their health care choices and the patients' perspectives are critical to understanding the impact of medical devices on their daily lives.

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On this page:

• About Patient Preference Information
• Patient Preference Information to Help Understand Medical Device Benefits and Risks
• Patient Preference Information Resources
• Conducting a Patient Preference Information Study
• Contact Information

Related pages:

• CDRH Patient Science and Engagement Program
• Patient Preference-Sensitive Areas: Using Patient Preference Information in Medical Device Evaluation
• List of Patient Preference-Sensitive Priority Areas

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About Patient Preference Information

Patient-preference information (PPI) captures the value patients place on features of medical devices. PPI includes different patient perspectives on the benefits and risks of using medical devices and treating medical conditions. PPI is different from patient-reported outcomes which are part of a clinical trial and measure how patients feel and function.

Well designed and conducted PPI studies elicit which attributes are important to patients, how important they are, and what tradeoffs patients are willing to make amongst them. PPI is also referred to as health-preference assessment, stated-preference health survey, health-preference research, and patient-centered research in the scientific literature.

Many groups conduct patient preference studies, such as:

• Patient groups
• Members of the medical device industry
• Researchers
• Professional societies
• Health care providers
• Public and private partners

As the medical device community conducts more PPI studies, CDRH gains a better understanding of what patients value and the tradeoffs they are willing to make.

Patient Preference Information to Help CDRH Understand Medical Device Benefits and Risks

PPI can inform the design of a medical device, impact how a clinical study is designed, and be used to understand the impact of the clinical study results on patients.

PPI helps to:

• Find unmet patient needs early in medical device development.
• Identify the most important benefits and risks of a technology from a patient's perspective.
• Assess patients' views on different clinical study outcomes.
• Determine a meaningful change in clinical study outcomes.
• Clarify what patients think about the tradeoffs of the benefits and risks for a treatment or diagnostic.
• Clarify how patient preferences on benefits and risks vary among diverse groups and their willingness to accept uncertainty about the benefits and risks.

PPI has been used in multiple submissions for Premarket Approval Applications, Humanitarian Device Exemption Applications, and De Novo Requests which has informed CDRH's regulatory decision making.
For example:

• An FDA-sponsored PPI survey related to obesity devices informed an approval for a medical device to treat obesity.
• Ear infections are common in children and are often treated with antibiotics. Children who experience multiple ear infections may need ear tubes to prevent ear infections. These tubes are often placed in the operating room. A medical device company developed a product that allows doctors to place ear tubes in pediatric patients in their office. The company used a PPI study to help inform the design of the clinical trial for the product.
• Home hemodialysis has rare but serious adverse events so a caregiver or trained partner must be present. However, a medical device company conducted a PPI study that showed some patients were willing to accept risks so they could do dialysis at home alone. This PPI study informed CDRH's decision to expand the labeled indications for the home hemodialysis device.
• Collecting information from patients helped enhance glucose monitor and insulin pump safety for children. CDRH discussed concerns with patients and parents about the safety of using an insulin pump in pediatric patients. Based on this feedback, CDRH worked with a medical device company to develop additional risk mitigations to include a lockout feature and prevent unintended insulin doses.

For more information, see How Patient Preferences Contribute to Regulatory Decisions for Medical Devices.

Patient Preference Information Resources

CDRH is committed to including patients' voices into our decision making. We acknowledge PPI is an active and evolving research area. We continue to partner with patient groups, members of the medical device industry, researchers, professional societies, health care providers, and public and private partners to advance PPI studies.

Collaborations

• Medical Device Innovation Consortium (MDIC) - Science of Patient Input
• Setting Scoliosis Straight Foundation (SSSF)
• Centers of Excellence in Regulatory Science and Innovation
• MDEpiNet
• ISPOR

Published Studies and Ongoing Projects

FDA scientists frequently collaborate with a variety of stakeholders to conduct PPI studies to inform clinical trial design and medical device regulatory decision making. Examples include medical devices for:

• Obesity
• Parkinson's Disease
• Amputation
• Glaucoma
• Uterine Fibroids
• Prostate Cancer
• Chronic Pain
• Adolescent Scoliosis
• Heart Failure
• Kidney Disease

Sample Blogs and Articles

• Patient-Centered Clinical Trial Design for Heart Failure Devices via Bayesian Decision Analysis - PubMed (nih.gov)
• Leveraging Patient Preference Information in Medical Device Clinical Trial Design - PubMed (nih.gov)
• Patient Preferences Regarding Surgical Treatment Methods for Symptomatic Uterine Fibroids (springer.com)
• Development of a Patient Preference Survey for Wearable Kidney Replacement Therapy Devices (07/28/2022)
• Quantifying Benefit-Risk Preferences for Heart Failure Devices: A Stated-Preference Study (12/23/2021)
• Incorporating Patient Preferences via Bayesian Decision Analysis (03/03/2021)
• Integrating Patient Perspectives into Medical Device Regulatory Decision Making to Advance Innovation in Kidney Disease (03/03/2021)
• Legitimization and Incorporation of Patient Preferences: The Arrow that Hit the Achilles Heel of Status Quo Kidney Care (03/03/2021)
• Using Patient Preference Information to Inform Regulatory Decision Making: An Opportunity to Spur Patient-Centered Innovation in Kidney Replacement Therapy Devices (03/03/2021)
• Overview of Various Components of the Science of Patient Input (03/03/2021)
• Integrating the Voice of the Patient Into the Medical Device Regulatory Process Using Patient Preference Information (02/29/2020)
• Prioritizing outcome preferences in patients with ocular hypertension and open-angle glaucoma using best-worst scaling (09/03/2019)
• Why Partnerships are Key to the Science of Patient Input (10/02/2016)
• FDA's Patient Preference Initiative: The Need for Evolving Tools and Policies (09/16/2016)
• How Patient Preferences Contribute to Regulatory Decisions for Medical Devices External Link Disclaimer(09/25/2017)
• Abstract: A Framework for Incorporating Patient Preferences Regarding Benefits and Risks into Regulatory Assessment of Medical Technologies (10/19/2016)
• FDA Brings Patients into the Process (09/20/2013)

FDA Workshop Transcripts and Recordings

• 2020 ISPOR-FDA Summit - Using Patient-Preference Information in Medical Device Regulatory Decisions: Benefit-Risk and Beyond
• 2017 Workshop - Advancing Use of Patient Preference Information as Scientific Evidence in Medical Product Evaluation
• 2013 Workshop - The Patient Preference Initiative: Incorporating Patient Preference Information into the Medical Device Regulatory Processes

Conducting a Patient Preference Information Study

Groups interested in starting or conducting a PPI study should email CDRH-PPI@fda.hhs.gov. You may request a pre-submission meeting to discuss a potential or current PPI study. We encourage medical device manufacturers and other stakeholders to consult with CDRH early when considering PPI studies.

The FDA remains committed to assuring that medical devices are safe and effective. The FDA encourages medical device manufacturers to consider the impact of PPI across the entire life cycle of a medical device. Including PPI does not change any review standards for medical devices.

For more information, refer to these guidance documents:

• Consideration of Uncertainty in Making Benefit-Risk Determinations in Medical Device Premarket Approvals, De Novo Classifications, and Humanitarian Device Exemptions
• Benefit-Risk Factors to Consider When Determining Substantial Equivalence in Premarket Notifications (510(k)) with Different Technological Characteristics
• 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
• Factors to Consider When Making Benefit-Risk Determinations in Medical Device Premarket Approval and De Novo Classifications
• Factors to Consider When Making Benefit-Risk Determinations for Medical Device Investigational Device Exemptions
• Factors to Consider Regarding Benefit-Risk in Medical Device Product Availability, Compliance, and Enforcement Decisions

Contact Information

If you have questions about PPI, email CDRH-PPI@fda.hhs.gov.