FDA finalizes guidance on patient preference information for devices

The US Food and Drug Administration (FDA) has finalized guidance on incorporating patient preference information (PPI) for assessing the benefit-risk profile of certain medical devices throughout the total product life cycle.

This PPI can be used to help FDA review investigational device exemption (IDE) applications, premarket approval (PMA) applications, humanitarian device exemption (HDE) applications, de novo classification requests, premarket notifications (510(k)s), requests for a breakthrough device designation, or for decisions involving administrative or enforcement actions.

PPI can also be utilized in the postmarket area to update patient labeling, ensuring that the device’s risks and benefits are clearly communicated to patients and healthcare professionals.

The agency defines PPI 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.”

The guidance replaces the agency’s 2016 final guidance and finalizes a draft version issued in September 2024, with only minor changes from the 2024 draft document. (RELATED: FDA updates patient preference guidance to span the product life cycle, Regulatory Focus 5 September 2024)

The final guidance fulfills a commitment in the Medical Device User Fee Amendments (MDUFA V) to update the 2016 PPI guidance to broaden its application across the lifecycle of the device.

FDA states in the guidance that the submission of PPI to the agency is voluntary and may not be relevant for all types of medical devices. However, it can be beneficial to collect this information for certain devices, especially those where the choices made by patients are influenced by personal preferences. These include devices that directly interact with patients, devices that provide health or aesthetic benefits, devices that impact health-related quality of life, certain life-saving devices that are also high-risk, and devices to address unmet medical needs or to treat rare diseases or conditions.

The guidance discusses how patient input can impact decision making, why PPI should be included in decision making, how PI differs from patient reported outcomes (PROs); the best ways to collect patient preferences; effective communications of benefit, risk and uncertainty; seeking feedback of study plans; maintaining the integrity of PPI; including PPI in device summaries; and offers some hypothetical examples to demonstrate how FDA might consider PPI when making benefit-risk assessments.

The final guidance includes a few minor changes from the draft version. In response to comments received, the FDA has revised the guidance to clarify terminology. It also adds recommendations for when sponsors might benefit from meeting with the FDA and suggests situations where involving patients in the design and implementation of a study could be advantageous.

Another change from the draft version is the inclusion of a new factor to consider for when the inclusion of PPI would be useful. FDA now says that PPI may be useful to include for devices for which the clinical experiences relating to key endpoints are subjective.

New text has also been added on study conduct in Section V for patient preference studies to determine whether there is “reasonable assurance” that the device is safe and effective.

The guidance now states that “the validity and reliability of study results depend in large part on compliance of research staff and study participants with the study protocol. If not self-administered, a PPI study should be administered by trained research staff. If the PPI study is self-administered, the participants should go through a tutorial and a quiz before answering questions, to help to ensure adequate comprehension and compliance with the study protocol.”

Final guidance