The agency issued draft guidance on submitting patient preference information as part of PMA and HDE applications and de novo requests, as well as on incorporating the data in labeling, saying patient perspectives can be important to take into consideration when making benefit-risk determinations.
In Patient Preference Information – Submission, Review in PMAs, HDE Applications, and De Novo Requests, and Inclusion in Device Labeling, the FDA says it wants patients who use devices for medical treatment to share their experiences, saying the input would help the agency assess the benefit-risk profile of certain devices under PMA, HDE or de novo review.
The FDA defines patient preference information as assessments — qualitative or quantitative — of the “relative desirability or acceptability of attributes” that vary among “alternative diagnostic or therapeutic strategies.” Device attributes include effectiveness, duration of effect and use, and other characteristics that may impact benefit-risk considerations.
While the submission of patient preference information is voluntary, the FDA says there are circumstances under which it may be useful, particularly when usage decisions are “preference-sensitive.” This may be the case when there are multiple treatment options, but no option that is “clearly superior for all preferences.” Devices with certain attributes may yield such circumstances, including ones with a direct patient interface, ones with novel technology and ones intended to directly affect the quality of life, for example.
The draft document goes a step further than the regulator’s 2012 benefit-risk guidance, which says patients’ tolerance for risk and perspective on benefits may be considered during device reviews — by providing guidance on patient preference data that may be considered. In issuing the guidance, the FDA is looking to:
- Encourage the voluntary submission of this information.
- Define qualities of patient preference studies.
- Provide recommendations for the collection of patient preference information.
- Provide recommendations for the inclusion of collected data in labeling for patient and health care professionals.
The FDA says patient preference information provides valid scientific evidence about patients’ risk tolerance, also noting that patients’ perspectives on benefits may inform its evaluation of a device’s benefit-risk profile. In addition to explaining how the regulator might consider patient preference data throughout such evaluations, the guidance goes over how patient preference information can be used throughout the total product life cycle. The FDA lists examples, including using the data to inform device design and features during the discovery and ideation phase, and to inform redesign or device improvement as post-market patient-centered data accumulates.
The guidance document also explains study qualities the FDA will consider when deciding whether patient preference data constitutes valid scientific evidence, listing 11 recommended qualities, including representativeness of the sample and generalizability of results, patient-centeredness, minimal cognitive bias and study conduct.
Also covered in the guidance document were “additional considerations,” which covered the maintenance of the integrity of patient preference data and conditions of approval, as well as submission of the information.
The agency also made recommendations for incorporating the patient preference information into device labeling, noting that for devices for which the regulator considered this type of information, labeling should describe study data.