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FDA Discusses Effective Patient Engagement at #BIOSummit19

Theresa Brady
Theresa Brady
November 1, 2019

For the FDA, engagement with patients, caregivers and patient advocacy organizations is a vital part of the drug development and review process. However, how that engagement happens can determine how successful it is.

This was the key question addressed by the “Practices and Principles: Navigating the FDA” panel on day one of BIO’s Patient and Health Advocacy Summit.

BIO Patient & Health Advocacy Summit 2019

 “It’s really been heartening to see the ways that not just the FDA but industry is incorporating patients and advocates into the drug development and review process much earlier on,” said Jeff Allen, president and CEO of the Friends for Cancer Research. “Those doors are opening, now it’s just about empowering people to step through those doors.”

Captain Robyn Bent is the director of the Patient Focus Drug Development Program at the FDA’s Center for Drug Evaluation and Research (CDER). She discussed the FDA’s ongoing effort to develop a series of four “guidances” that will provide a framework for the process of collecting and submitting patient input.

The overall goal of this process is to ensure that the focus is on what’s important to the patient.

“The chief complaint of the patient wasn’t something that we were necessarily measuring as the end point of our trials,” Bent said. “We need to be moving toward that goal of ensuring that what we measure is something that matters to the patient.”

Guidance 1 – which discusses sampling methods to be used when planning to collect patient input – has been released. Guidance 2 – which outlines methods to identify what is important to patients – is in draft form. Guidance 3 – which will address the process of refining the list of important impacts and concepts to develop potential study instruments – just completed the public workshop phase. And the process for Guidance 4 – which will address the methodologies, standards, and technologies to be used for the collection, capture, storage, and analysis of clinical outcome assessment data – is scheduled to begin with a public workshop on Dec. 6.

Bent also emphasized that public input is key to developing guidelines that work.

Speaking about the guidance document development process, Bent indicated that “We go through every single one of your comments and discuss every comment we receive,” she said. “In the end we all have the same goal, which is to hear the voice of the patient.”

Samir Shaikh, who is deputy director of Patient Affairs Staff in the FDA’s Office of the Commissioner, told the audience about rare disease listening sessions that the FDA is holding in partnership with the National Organization for Rare Disorders, to help the FDA better understand patients’ experience with a rare disease or condition.

“The goal is to inform the work the FDA is doing on clinical trial design,” Shaikh said. “One of the unique aspects of the listening sessions is the format. It’s a rapid resource for FDA staff to meet with the community.”

The FDA is currently scheduling rare disease listening sessions for 2020. Those who are interested in participating should fill out the webform at And while these sessions aren’t public, the FDA does post summaries of each one on its website.

Once patient input is gathered, the question becomes how to best share it with those who need it. Annie Kennedy with the Muscular Dystrophy Association, emphasized that it can be a full-time job to ensure that the right data is getting to the right people at the right time.

“We’d rather spend time generating data and answering questions than telling people where the data is,” Kennedy noted.

Allen pointed out that collaboration and sharing best practices is key.

“We’re all learning how to engage people better together,” he said. “If everyone does it separately it’s going to take four times as long, if things are too siloed each organization is going to do their own thing. A rising tide lifts all boats.”

All panelists agreed that working together is the most important element to ensure that patient input is both shared – and used — successfully.

“We need more real-time input to get a more holistic picture of what patients are experiencing so products can be developed in a robust way to ensure their quality,” said Allen. “There are a lot of opportunities, a lot of strategies to unify efforts. We just need to work through the kinks and ensure we’re fully capitalizing on these opportunities.”