On September 4, the FDA released a draft guidance that significantly revises its previous guidance on the use of voluntary patient preference information (PPI) in medical devices, shifting the focus from the premarket stage to the entire product lifecycle.
The changes in the new draft guidance are based on the agency’s growing experience with industry-sponsored PPI studies, improved understanding of PPI trial design, and other factors. The FDA stated that the release of this draft guidance proposes revisions to the 2016 guidance to reflect the broader scope of the FDA’s current benefit-risk paradigm, which may, where appropriate, include PPI, and to provide additional considerations and practical advice based on more experience assessing patient preferences for devices.
Once finalized, the guidance will replace the 2016 guidance that the FDA has used since then for including PPI in premarket approval (PMA) applications, humanitarian device exemption applications, or reapplications.
The FDA noted that the draft guidance will fulfill certain provisions of the Medical Device User Fee Amendments (MDUFA V) agreement negotiated with the medical technology industry. Under MDUFA V, the agency agreed to expand its capacity and increase resources to respond to premarket submissions that include PPI data, and to update its 2016 guidance with practical insights and address common questions about PPI. While the 2016 guidance had a section addressing when and how PPI would be considered during premarket review, the new guidance details the circumstances in which the agency will use such information to support its review decisions.
Examples of Voluntary Patient Preference Information
The draft guidance provides several examples, including a PPI study sponsored by the Center for Devices and Radiological Health (CDRH) to evaluate the extent of risk patients are willing to accept to offset the potential benefits of obesity weight-loss devices. The study used a sufficiently large sample size with demographics representative of the U.S. population and offers other details for sponsors to consider when designing PPI studies.
The guidance also includes examples of using PPI studies to determine whether to expand device indications, update labeling, and establish performance thresholds.
Application and Consideration of Patient Preference Information
In the draft guidance, the FDA added more details on when and how PPI is considered in premarket submissions, reiterating that it will consider PPI at all stages of a device’s total product lifecycle (TPLC). For Investigational Device Exemptions (IDEs), it will consider not only the patient risk-benefit profile but also the social benefits that the knowledge gained from the study may provide.
“In clinical studies, patient preferences may vary, including the outcomes most important to specific patients, the level of risk they are willing to accept in exchange for a certain level of benefit, their preferred method of treatment/diagnosis (devices are often one of several options considered in clinical care pathways, which may include drugs or surgery), and the importance they place on the potential social benefits of the study itself and advancing future treatment options for patients,” the FDA stated.
The FDA noted that even if a 510(k) device does not meet its typical risk-benefit requirements, it may still review PPI study data to make a regulatory decision regarding the device.
“In some cases, although a device does not meet FDA requirements, some patients and caregivers may still wish to use the device,” the agency said. “In making decisions involving administrative, enforcement, and other actions, the FDA intends to consider patient impact, whether patients and caregivers are fully informed about the related benefits and risks, and patient preferences for non-compliant or non-conforming devices.”
The draft guidance also details important factors in designing PPI studies, obtaining FDA feedback on PPI study plans, and providing results, as well as different methods for collecting PPI data.
An appendix includes a new section on determining the appropriate research methods based on the device and its intended use. Stakeholders can comment on the draft guidance under docket number FDA-2015-D-1580 on www.regulations.gov by November 5. The agency will also hold a webinar to discuss the guidance on October 15.
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