FDA REMS Reforms Lacking Patient Engagement So Far

As new repository website launches, agency acknowledges that it must do more to create patient-focused risk management.

FDA's next steps to better integrate Risk Evaluation and Mitigation Strategies (REMS) into the healthcare system could include getting more routinized feedback from patients.

Theresa Toigo, CDER's associate director for drug safety operations, acknowledged that "I don’t have a good answer" to the question of how the agency would engage with patients to improve the REMS experience, which has proven burdensome to all pharmaceutical stakeholders – sponsors, prescribers, dispensers and consumers – even as the regulatory tool has allowed the approval of products that would otherwise be deemed too unsafe.

The question was asked during a June 15 session at DIA's annual meeting in Washington, D.C. by Sally Okun, vice president of advocacy, policy and patient safety at the patent research network PatientsLikeMe. Okun struck a friendly tone, and the agency and the group are on good terms; they announced a research collaboration the same day as the meeting.

But that friendliness underscores the emerging challenge for FDA: Having met with some success, patient groups are now going to be pressing the agency on all fronts to boost their participation in regulatory and review processes, and FDA isn't going to have a tidy set of trade associations it can interact with to make sure it's gotten sufficient feedback.

"Pharmacy – they’ve just come to us and shared," Toigo said about the agency heard of REMS concerns. "We haven’t had as much of that from the patients, so we are relying on when they reach out or schedule time, but we are certainly open to suggestions to hear about how we could better to get broader patient feedback."

FDA officials "sometimes hear from very vocal [patients] and it’s that one patient’s perspective, so I think our challenge is making sure that we hear from diverse perspectives, and we’ve been able to get that at least in health care system pharmacy, and on the retail side with big pharmacy and not as big," Toigo noted.

The agency's main focus of late with patients has been the disease forums mandated by the user fee program, and that patient-focused drug development effort seems poised to expand when the legislation is renewed in 2017 (Also see "FDA Oncology Office Hopes Better Advice Will Lead To More Patient-Reported Outcomes Data" - Pink Sheet, 13 Apr, 2015.).

REMS, though, has so far remained relatively under the radar in terms of targets for reform, even as everyone acknowledges the system could benefit from some streamlining. To that end, FDA is exploring how to implement the ideas raised in a report it released last year (Also see "REMS Enhancements Could Include Provider Education Credits" - Pink Sheet, 23 Sep, 2014.).

The agency just reached one of the four goal posts that were set in the report: There's now a clearinghouse website, REMS@FDA, which includes all the materials for all the approved REMS.

Despite that, Paul Seligman, former director of CDER's Office for Drug Safety and now Amgen Inc.'s executive director for global regulatory and R&D policy, told the DIA meeting that "I think one of the biggest gaps currently in REMS is the area of patient engagement."

FDA "has shown a willingness to reach out patients as they shown in terms of the risks and benefits of drugs, areas of design and endpoints and whole variety of issues," and REMS is an area "that open's for a greater area of engagement and maybe some pilot studies" of how best to communicate with patents, he said.