Posted 03 November 2014 | By Alexander Gaffney, RAC,

When Congress passed the Food and Drug Administration Safety and Innovation Act (FDASIA) into law in 2012, its patient-centered provisions were among the biggest changes set to impact the culture of the US Food and Drug Administration (FDA). Now, two years later and with major planning already underway regarding the future of FDA's patient-centered activities, agency regulators are reaching out to the public to ask what more they can do to improve their efforts.

Background

FDA's Patient-Focused Drug Development (PFDD) program was born out of a concern—harbored by some patient groups and legislators—that FDA was too stringent at regulating products intended for diseases that lacked adequate treatment options.

Under FDASIA, FDA was tasked with setting up more than a dozen meetings with specific patient groups with the intent of hearing how the patients lived with their respective diseases, what they wanted to see in an ideal treatment, the benefits they desired and the risks they felt were meaningful.

To date, FDA has announced 11 meetings with patients under the program, and is scheduled to hold at least six more by the end of 2015, when the first class of announced disease groups is set to expire.

In preparation for that expiration, FDA recently announced a preliminary list of 16 additional diseases on which it hopes to hold meetings as part of its patient-focused drug development program. The meetings would be held in fiscal years 2016 and 2017, FDA said.

FDA: Give Us Your Feedback

But outside of asking the public what diseases it should focus on, FDA has asked for little feedback on what patients actually think of the process, and whether regulators can do more to improve.

That's no longer the case. On 3 November 2014, FDA released a new Federal Register posting indicating that it will establish a federal docket to allow members of the public—and in particular patients and patient groups—to weigh in on "FDA activities performed under the FDASIA Patient Participation in Medical Product Discussions."

"This notice announces FDA’s intent to gather input from stakeholders on strategies to obtain the views of patients during the medical product development process and ways to consider patients’ perspectives during regulatory discussions," FDA wrote.

General Patient Input

As FDA explains in the Register notice, it's not only interested in hearing feedback about its PFDD program.

Under Section 1137 of FDASIA, FDA was also ordered to "develop and implement strategies to solicit the views of patients during the medical product development process and consider the perspectives of patients during regulatory discussions."

While similar to FDA's PFDD, this approach differs in that it is supposed to be applied to individual products under review at FDA, as opposed to PFDD's more broad but systematic approach which focuses on all products intended for a particular disease.

For example, FDASIA ordered FDA to allow patients to serve as special government employees in "appropriate agency meetings with medical product sponsors and investigators" (such as FDA's various Advisory Committee meetings) and also to allow non-conflicted patients to serve as advisors to FDA. This, it was hoped, would allow FDA to consider the views of neutral patients while it evaluated the risks and benefits of a particular product.

FDA said it wants to receive feedback from patients about this program as well.

Don't Forget About Devices

Also mentioned by FDA in the Federal Register notice is its little-noticed Patient Preference Initiative for medical devices. Announced in February 2013, the initiative is intended to eventually bring the same patient-focused approach to device regulation that is now taking hold at FDA's drug regulator, the Center for Drug Evaluation and Research (CDER).

"Once the Patient Preference Initiative helps to define or refine the methods to measure patient preferences, FDA intends to incorporate patient views into the total product life cycle of medical devices," the agency explained in its most recent Register announcement.

However, the initiative is substantially less developed than its pharmaceutical counterparts discussed above, and therefore less well known to patients.

Call for Comments

Regardless of the status of each program, patients will have 30 days in which to give FDA their feedback regarding the state of patient involvement in the product development and evaluation process. Patients can either respond to FDA's notice on FederalRegister.gov, or find FDA's soon-to-be-posted docket, FDA-2014-N-1698, on Regulations.gov.

Federal Register announcement