FDA Clarifies How Patient Preference is Used In Device Approvals

In a continued effort to give patients more of a voice in medical device development, the U.S. Food and Drug Administration (FDA) has issued a new draft guidance that outlines ways patient preference information (PPI) might be collected and used in device approvals and labeling.

“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insights and perspectives on the benefits and risks of devices under PMA, HDE, or de novo review,” the FDA said in its draft guidance.

Patient involvement in medical device development has been an agency and industry priority for the past few years. In 2012, the FDA published a document about benefit-risk assessments that encouraged patient-centered metrics in determining positive outcomes and tolerance of possible side effects.

In 2013, the Center for Devices and Radiological Health (CDRH) launched a program called the Patient Preference Initiative, which established a patient engagement panel as part of its Medical Device Advisory Committee (MDAC) and reiterated the importance of patient preferences in device safety and efficacy evaluations.

This newest draft guidance is described by the FDA as the “next step” in an ongoing process initiated by the agency to help the industry bring patients into the discussion without compromising their privacy or safety. With this guidance, the FDA also hopes to iron out some of the details of a patient-centered regulatory process.

In the newly proposed system, device manufacturers would collect its own PPI and submit it along with their application if they deemed it appropriate, and the FDA noted that the additional data might be particularly useful for devices with novel technology or those indicated for an unmet need.

CDRH describes and gave examples of two possible methods that the industry might use to collect the information: “stated preference, in which preferences are elicited by offering choices to participants” and “revealed-preference methods, in which patient preferences are obtained from the actual clinical choices made by patients.” Patient preferences studies, they said, should be representative of all the patients who would use the device and be large enough for researchers to form generalized opinions.

The guidance also explained the importance of studying differing opinions in patient subgroups. An acceptable risk for one patient subset might not be acceptable for another, and the FDA describes how those differences of opinion might be documented in a device’s labeling.

The agency was careful to point out that the guidance does not represent a change in how a submission will be evaluated, but rather an explanation of how patient preferences are handled by regulators in the decision making process. Furthermore, while patient preferences might allow a risky device to be approved, that approval may be conditional on the prompt initiation of further clinical studies post market.

Coinciding with the new draft guidance, the Medical Device Innovation Consortium (MDIC) recently released a report — written in collaboration with the FDA — about a possible framework for PPI inclusion in the entire life-cycle of a medical device, as well as a “catalogue of methodologies” that could be used to analyze data collected from patients.

“The FDA is committed to integrating the patient voice into its regulatory decision-making and is pleased with the significant progress that MDIC has made in laying the groundwork for this work,” Robert M. Califf, M.D., FDA deputy commissioner for medical products and tobacco, said in an MDIC press release.

The agency will accept comments on the draft guidance until August 2015.