The U.S. Food and Drug Administration (FDA or the Agency) values the experience and perspectives of patients. The Agency understands that patients and care-partners who live with a disease or condition on a daily basis and utilize devices in their care may have developed their own insights into and perspectives on the benefits and risks of devices reviewed under the premarket approval, humanitarian device exemption (HDE), or de novo classification pathway. FDA believes that patients can and should bring their own experiences to bear in helping the Agency evaluate the benefit-risk profile of certain devices. This kind of input can be important to consider during FDA’s decision making for these devices.
For this reason, FDA’s guidance document “Factors to Consider When Making Benefit-Risk Contains Nonbinding Recommendations Determinations in Medical Device Premarket Approval and De Novo Classifications” (hereafter referred to as the Benefit-Risk Guidance) explains that reviewers may consider certain data measuring patient perspectives during the premarket review process for premarket approval applications (PMAs) and de novo classification requests, when such information is available. That guidance specifies that patient tolerance for risk and perspective on benefit, in addition to several other factors, may be considered in FDA’s assessment of the benefit-risk profile of certain devices when the information qualifies as valid scientific evidence.
This guidance document takes the next step and provides guidance on patient preference information (PPI) that may be used by FDA staff in decision making related to PMAs, HDE applications, and de novo requests. The objectives of this guidance are:
1) to encourage submission of PPI, if available, by sponsors or other stakeholders to FDA and to aid in FDA decision making;
2) to outline recommended qualities of patient preference studies, which may result in valid scientific evidence;
3) to provide recommendations for collecting and submitting PPI to FDA; and
4) to discuss FDA’s inclusion of PPI in its decision summaries and provide recommendations for the inclusion of such information in device labeling.
This guidance also includes several hypothetical examples that illustrate how PPI may inform FDA’s decision making.
The policy described in this guidance document is consistent with FDA’s Benefit-Risk Guidance. In particular, the Worksheet for Benefit-Risk Determinations from the BenefitRisk Guidance has been updated to reflect the Agency’s current thinking on patient preference information.