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  1. Division of Patient-Centered Development

Patient Preference Information (PPI) in Medical Device Decision Making

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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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 among 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
  • Sponsors and 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

Risk And benefit wooden blocks on seesaw

PPI can be especially helpful to CDRH to assist in the identification of patient preference-sensitive areas for medical device review, including when:

  • FDA staff are looking to better understand the full impact of the disease or condition and treatment options on patients and/or caregivers.
  • Patients may value the benefits and risks of a technology or treatment differently from health care professionals and/or caregivers.
  • Population-level differences in patient perspectives are not well understood because of differences in:
    • Demographic characteristics
    • Stages of a disease
    • Disease phenotype
  • There is significant public health impact (such as high prevalence rates of the disease or few treatment options available such as in rare diseases).

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 FDA-decision making, including Premarket Approval Applications (PMAs), Humanitarian Device Exemption (HDE) Applications, De Novo classification requests, and premarket notifications (510(k)s).

For example:

  • Treatment for adult patients with refractory or recurrent ascites due to liver cirrhosis  is typically treated with paracentesis.  A medical device company developed an implantable pump for liver ascites to remove excess fluid through the bladder. The company conducted a PPI study, which confirmed that patients with recurrent or refractory ascites are interested in alternative treatment options. Patients indicated that they were willing to tolerate risks beyond what has been observed in the clinical experience to reduce the need for therapeutic paracentesis.
  • 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 the FDA’s final guidance: Incorporating Voluntary Patient Preference Information over the Total Product Life Cycle

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

Completed and Ongoing Regulatory Science 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:

Sample Blogs and Articles

Previous FDA Actions: Workshop Transcripts and Recordings

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:

Contact Information

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

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