Executive Summary

New office will develop agency-wide patient engagement policy and serve as entry point for patient groups new to FDA, but will not interfere with existing relationships between advocates and the drugs and biologics centers, FDA Commissioner Gottlieb says.

The US FDA’s proposed Office of Patient Affairs will develop and implement patient engagement best practices across the agency’s medical product centers but will not interfere with existing relationships that advocacy groups have with the drugs and biologics centers and individual review divisions, FDA Commissioner Scott Gottlieb said Sept. 13.

Speaking at the Blueprint for Breakthroughs meeting sponsored by Friends of Cancer Research (FOCR) and Alexandria Real Estate Equities, Gottlieb said the new office will “play more of a coordinating function to try to set agency-wide policy with respect to how we think about engaging patients in the regulatory process.”

However, “this isn’t a desire to have sort of a one-stop where everyone has to come through a certain front door, or to displace the relationships that currently exist with the centers directly with different patient groups and how the centers are operating,” he said.

Gottlieb also shared more details about the agency’s work on 10 new disease-specific guidance documents. (See sidebar for related story.)

‘Small’ Office That Will Develop Policy

The commissioner’s comments about the patient affairs office suggest the proposal is indeed coming to fruition, but perhaps will be smaller in scope than originally envisioned when FDA first floated the idea.

In March, FDA invited public comments on the establishment of an Office of Patient Affairs to provide a “more transparent, accessible and robust experience for patient communities,” and to handle patient engagement activities across FDA’s medical product centers and other offices.

The office was envisioned as a “single, central entry point” for patients that would help move their inquiries to the proper place, and which also would host and maintain data management systems that incorporate and formalize knowledge shared with FDA by patient stakeholders. (Also see “US FDA Patient Affairs Office Could Accelerate Involvement With ‘Central Entry Point'” – Pink Sheet, 14 Mar, 2017.)

Although the idea drew generally positive reactions from the patient community, it raised concerns that engagement efforts would be siloed within the new office and existing relationships between patient groups and the medical product centers could be interrupted. (Also see “Patient Advocates Continue To Push US FDA For Central Office” – Pink Sheet, 5 Sep, 2017.)

Responding to a question from the audience, Gottlieb’s remarks seemed aimed at addressing those types of concerns.

Emphasizing that the new entity will be a “small office,” Gottlieb said it is intended to provide “cross-center coordination around the policies that we adopt with respect to how patients are being incorporated into the process.”

He noted, for example, that the Center for Devices and Radiological Health “is doing a lot of novel things with how they’re engaging patients in their processes. If they have a best practice that may be relevant to other centers, we want to have the opportunity to have a forum where we could look at that as a matter of policy and see how we might adopt it on an agency-wide basis.”

“If patients are being engaged in a certain way in the end of Phase II studies that we think could be paralleled in other regulatory context, there again that’s something that we might want to look at and see how we develop broader policy around that,” Gottlieb said. “That’s going to be the goal of this group.”

“A lot of groups now currently have relationships … particularly that relationship with the program review level. Those are very important, and when those exist I want to see those maintained.” – FDA’s Gottlieb

While the office will serve as an entry point for patient groups that are new to interacting with the agency, it will not hinder groups’ established relationships with FDA centers.

“For groups and patients that don’t know how to access the agency, and there are some, then this would also provide a clear avenue for those,” Gottlieb said.

“A lot of groups now currently have relationships. I want them to maintain those relationships, particularly that relationship with the program review level. Those are very important, and when those exist I want to see those maintained,” he said. “For new groups and groups that might not already have those kinds of relationships, this could also be … a front door for them, but not a front door in that everyone gets diverted.”

https://pink.pharmamedtechbi.com/PS121537/US-FDAs-Centralized-Patient-A…