Patients provide valuable input to the FDA in a variety of forms. Patient input includes a wide range of information and perspectives including anecdotal comments in correspondence to the FDA or testimony at Advisory Committee meetings, patient opinions expressed publicly including through social media, patient responses to qualitative surveys, and quantitative measurements of patient-reported outcomes.
Patient perspectives refer to a type of patient input, and includes information relating to patients' experiences with a disease or condition and its management. This may be useful for better understanding the disease or condition and its impact on patients, identifying outcomes most important to patients, and understanding benefit-risk tradeoffs for treatment. This web page focuses on patient preference information as one specific type of patient perspective.
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The Center for Devices and Radiological Health (CDRH) recognizes that scientists, clinicians, device developers, and regulators play critical roles in evaluating and communicating the benefits and risks of medical devices. However, only patients live with their medical conditions and make daily choices regarding their health care. Their voice and perspective are critical to understanding the impact of medical devices.
Patient preference information (PPI) is one approach that incorporates the patient perspective in CDRH's regulatory decision-making. CDRH defines patient preference information as qualitative or quantitative assessments of the relative desirability or acceptability to patients of specified alternatives or choices among outcomes or other attributes that differ among alternative health interventions. Patient preference information captures the value that patients place on aspects of the medical device in a way that accounts for differing patient perspectives on benefits and risks that come with using that device or treating the condition. Well-designed and conducted patient preference information studies inform medical product developers, regulators, and clinicians about what attributes are important to patients, how important they are, and what tradeoffs patients are willing to make between attributes. Patient preference information has also been referred to as health preference assessment, stated-preference health survey, health preference research, and broadly described as patient-centered research in other sources of scientific literature.
Many groups are conducting patient preference information studies, including:
- Patient groups
- Members of the medical device industry
- Healthcare providers
- Public-private partnerships
As the medical device community conducts more patient preference studies, CDRH gains a better understanding of the values patients have and the tradeoffs they are willing to make to have access to medical devices.
Patient preference information 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. This information can help CDRH by:
- Identifying the most important benefits and risks of a technology from a patient's perspective
- Assessing the importance of clinical study outcomes to patients
- Determining a meaningful change in study outcomes
- Clarifying how patients think about the tradeoffs of these benefits and risks for a given technology
- Showing how patient preferences vary across a population, regarding benefits and risks of various treatment options.
Patient preference information has been used in multiple regulatory submissions and has informed CDRH's regulatory decision-making. Seventeen patient preference studies sponsored by industry have been completed or are in the pipeline.
- CDRH conducted a patient preference information study that was used to inform deliberations surrounding the approval of a medical product – the first approved device for treating obesity. Deliberations considered information from an FDA-sponsored survey on patient preferences related to obesity devices.
- Home hemodialysis has rare but serious adverse events that require having a caregiver present. The sponsor conducted a patient preference information study that showed some patients were willing to accept the risk in exchange of doing dialysis at home alone. This study informed a label expansion for the home hemodialysis device.
- Collecting qualitative feedback from patients proved helpful in enhancing the safety of a continuous glucose monitor and insulin pump for children. CDRH discussed concerns with patients and parents about the safety of using an insulin pump in the young pediatric population. Based on this feedback, CDRH worked with the company to develop additional risk mitigations that included a lockout feature preventing unintended insulin boluses.
For more information, see How Patient Preferences Contribute to Regulatory Decisions for Medical Devices.
CDRH is committed to integrating the patient voice into our decision-making. We acknowledge that patient preference information is an active and evolving research area. We continue to partner with patient groups, public-private partnerships, professional societies, and other stakeholders to advance the field.
- Medical Device Innovation Consortium (MDIC) – Science of Patient Input
- MDIC – Patient Centered Benefit-Risk Framework
Published Studies and Ongoing Projects
FDA scientists have collaborated with a variety of stakeholders to conduct patient preference studies to inform clinical trial design and medical device regulatory decision-making. These patient preference studies have included:
- Medical devices for obesity
- Medical devices for Parkinson's disease
- Medical devices for amputation
- Minimally invasive glaucoma surgical devices
Blogs and Articles
- FDA Brings Patients into the Process (09/20/2013)
- FDA's Patient Preference Initiative: The Need for Evolving Tools and Policies (09/16/2016)
- Why Partnerships are Key to the Science of Patient Input (10/02/2016)
- How Patient Preferences Contribute to Regulatory Decisions for Medical Devices (09/25/2017)
- Abstract: A Framework for Incorporating Patient Preferences Regarding Benefits and Risks into Regulatory Assessment of Medical Technologies
- 2013 Workshop – The Patient Preference Initiative: Incorporating Patient Preference Information into the Medical Device Regulatory Processes
- 2017 Workshop – Advancing Use of Patient Preference Information as Scientific Evidence in Medical Product Evaluation
Sponsors or other parties interested in initiating or conducting a patient preference study should email CDRH-PPI@fda.hhs.gov and may consider requesting a q-submission meeting (PDF – 215 KB) to discuss a potential or current patient preference study. We encourage medical device manufacturers and other stakeholders to consult with CDRH early when considering patient preference studies.
In considering patient preference information, the FDA remains committed to assuring that devices are safe and effective. The FDA encourages medical device manufacturers to consider the impact of patient preference information across the entire life cycle of a device—from the development and evaluation to the surveillance of the device once it is sold in the United States. The inclusion of patient preference information does not change any review standards for medical devices.
For more information, refer to these guidance documents:
- 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 (PDF - 766 KB)
- Factors to Consider When Making Benefit-Risk Determinations in Medical Device Premarket Approval and De Novo Classifications (PDF – 1.1 MB)
- Factors to Consider When Making Benefit-Risk Determinations for Medical Device Investigational Device Exemptions (PDF - 749 KB)
- Factors to Consider Regarding Benefit-Risk in Medical Device Product Availability, Compliance, and Enforcement Decisions (PDF - 870 KB)
If you have questions about patient preference information, email CDRH-PPI@fda.hhhs.gov.