- FDA held its first Patient Engagement Advisory Committee (PEAC) meeting on Wednesday, October 11, aiming to engage patients and caregivers in discussions regarding FDA’s regulatory activities.
“The FDA’s work requires us to establish objective, consistent criteria on which we base our decisions,” FDA Commissioner Scott Gottlieb, MD, wrote in a statement prior to the event. “But ultimately, the criteria we use to judge benefit and risk turn on the parameters that matter to patients. Involving the end-user – the patient – in identifying health priorities and outcomes desired from health interventions is critically important.”
The PEAC was funded by FDA’s Center for Devices and Radiological Health (CDRH) and aligns with the agency’s goals for patient-centered drug, treatment, and medical device development, Gottlieb said.
The patient engagement working group turns to patient and caregiver stakeholders to assess the utility of tools through the entire development, clinical trial, and marketing process.
“This includes new guidance on patient preference information for premarket approvals (PMAs), Humanitarian Device Exemption (HDE) applications, de novo requests, and for inclusion in device labeling,” Gottlieb explained. “This guidance describes how patient tolerance for risk and perspective on benefit can be considered as part of the FDA’s assessment of the benefit-risk of certain devices.”
Although FDA has not yet released remarks following the meeting, Gottlieb did share some of the inaugural meeting’s goals.
Foremost, the PEAC sought to assess patient perspectives on clinical trial design, recruitment, and results dissemination. The group was set to discuss elements such as outcomes data and patient-generated health data, two significant topics surrounding clinical trial development, Gottlieb said.
Additionally, the Committee discussed the use of patient preference data, which will be extremely helpful during the device development period.
“When assessing whether valid scientific evidence shows that a device’s probable benefit outweighs its likely risks, the FDA can also consider rigorous, systematically gathered patient preference information as a part of the totality of the evidence from clinical and nonclinical testing,” Gottlieb asserted.
“One of the mandates of this committee will be to help us develop the parameters for assessing these questions,” he continued. “When does this preference information meet the right standard, and address the right criteria, to be relevant to inform our decisions?”
Gottlieb also announced the formation of a new patient affairs team at FDA. The team will coordinate agency-wide and multi-center initiatives to support patient centricity.
That team is meant to supplement pre-existing efforts for improving patient engagement at FDA, Gottlieb said.
“This is not about creating a single front door, or a new door, for patient organizations to interface with the FDA,” he pointed out. “Groups that already have relationships directly to our medical product centers will maintain those engagements.”
FDA is focused on prioritizing the patient during medical device development, and is working on building its arsenal of patient engagements to achieve that goal.
“Our aim is to facilitate the development and use of patient-focused methods in more parts of our regulatory activities as well as develop and elevate common standards for how to integrate the patient voice, as a matter of science, into product development,” Gottlieb noted.
FDA first proposed the idea of a patient affairs office back in March, 2017. The agency welcomed a public comment period at the time and outlined the office’s plans to consult with patients and patient engagement advocacy groups regarding FDA activities.
“FDA has long recognized the importance of engaging with patients, caregivers, and their advocates in the medical product development process,” the FDA said in a public docket published in the Federal Register.
“Under this proposal to enhance mechanisms for patient engagement at FDA, a new ‘Office of Patient Affairs’ would be directly accountable to the medical product Centers through clear governance structures,” the agency added.
Specifically, the office of patient affairs would be in charge of the following:
- Offering a single, central entry point to the Agency for the patient community,
- Providing triage and navigation services for inbound inquiries from patient stakeholders,
- Hosting and maintaining robust data management systems that would incorporate and formalize knowledge shared with FDA by patient stakeholders and FDA's relationships with patient communities, and
- Developing a scalable and forward-looking platform for communicating with patient stakeholders, particularly online channels
Ultimately, the patient affairs team will be a part of FDA’s overarching strategy for aligning itself with industry-wide calls for patient-centricity, Gottlieb concluded in his statement about this week’s meeting.
“This new patient affairs team is one reflection of our commitment to the goal of elevating the patient role in our work,” he said. “The advisory committee that we’re inaugurating today is another measure of that promise.”