The U.S. Food and Drug Administration (FDA or the Agency) values the experience and perspectives of patients with devices. The Agency understands that patients and caregivers who live with a disease or condition on a daily basis and utilize devices in their care may have developed their own insights and perspectives on the benefits and risks of devices under PMA, HDE, or de novo review. 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 regulatory decision-making for certain devices.
For this reason, FDA’s guidance document “Factors to Consider When Making Benefit-Risk 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 meets FDA’s standards for valid scientific evidence.
This draft guidance document takes the next step and provides guidance on patient preference information that may be used by FDA staff in decision-making relating to PMAs, Humanitarian Device Exemption (HDE) applications, and de novo requests. The objectives of this draft guidance are: 1) to encourage voluntary submission of patient preference information by sponsors or other stakeholders, in certain circumstances; 2) to outline recommended qualities of patient preference studies, which may result in valid scientific evidence; 3) to provide recommendations for collecting patient preference information to FDA; and 4) to provide recommendations for including patient preference information in labeling for patients and health care professionals. This draft guidance also includes several hypothetical examples that illustrate how patient preference information may inform FDA’s regulatory decision-making.