Final Guidance Shows How Patient Preferences Enter FDA Device Decisions

The Food and Drug Administration issued final guidance on March 30 titled "Incorporating Voluntary Patient Preference Information over the Total Product Life Cycle." The guidance replaces FDA's 2016 guidance on patient preference information, which covered its use in premarket approval applications, humanitarian device exemption applications and De Novo requests.

The agency's Federal Register notice says patient preference information can now be used in FDA decision making across the product life cycle, including reviews of investigational device exemption applications, requests for Breakthrough Device designation, PMAs, humanitarian device exemptions, De Novo requests, 510(k) premarket notifications, and administrative, enforcement or other actions. RAPS reported that the final version changes little from a September 2024 draft and fulfills a commitment FDA made in its current device user fee agreement, MDUFA V.

What counts as patient preference information

FDA's patient preference page describes it as information capturing "the value patients place on features of medical devices," and says well-designed studies show which attributes matter to patients, how much, and what tradeoffs they are willing to make. It is different from patient-reported outcomes, which are measured in clinical trials and record how patients feel and function.

The guidance says the information may be most useful when patient decisions are "preference sensitive": when several options exist and none is clearly superior for all patients, when the evidence favoring one option is considerably uncertain for some populations, or when patients' views on the most important benefits and acceptable risks vary widely or differ from those of health care professionals.

It lists ways companies can use such studies, from identifying unmet needs and choosing trial endpoints to setting effectiveness targets and understanding how preferences differ among patient subgroups. For quantifying tradeoffs, it names methods including discrete choice experiments, best-worst scaling, the threshold technique, swing weighting and multi-dimensional thresholding.

Voluntary, but reviewable

FDA does not require the information. "Although PPI is not required for FDA's consideration as part of our decision-making, as discussed further below, it can be valuable for FDA to consider patients' viewpoints when the information meets applicable legal requirements," the guidance says. For it to count, a study should be designed to produce valid scientific evidence, the standard FDA uses to decide whether there is reasonable assurance a device is safe and effective.

RAPS reported that FDA added text on how preference studies should be conducted, including that "the validity and reliability of study results depend in large part on compliance of research staff and study participants with the study protocol." The final version also adds recommendations on when sponsors might benefit from meeting with FDA about their study plans.

When FDA approves a PMA, approves a humanitarian device exemption or grants a De Novo request, it typically publishes a decision summary, and the guidance says preference studies FDA considered are generally included in it. FDA's page says such studies have informed past decisions; one example it gives is a home hemodialysis system whose labeled indications were expanded after a study showed some patients were willing to accept the risks of dialyzing at home alone.