Rare Disease Report

Patient Engagement at the FDA

OCTOBER 23, 2017
Scott Winiecki, MD

The role that patients and caregivers are playing in the development and approval of orphan drugs is dramatically shifting within the offices at the U.S. Food and Drug Administration (FDA).  

At NORD’s Rare Disease and Orphan Drug Breakthrough Summit held in Washington D.C., October 15-16, 2017, we talked with Scott Winiecki, MD, of the Center for Drug Evaluation and Research (CDER) at the FDA about the growing number of ways that patients and patient advocates can be involved in the drug development and review process.

Rare Disease Report: How is the FDA embracing the role that patients play in drug development?
Dr. Winiecki: in the past 4 years we've done 24 patient-focused drug development meetings listening to patients and systematically gathering their input on their experience and their condition, and how it affects their lives. We also have the patient representative program where people can go through training and serve on advisory committees and attend other meetings so that patients and caregivers and their advocates can give personal input into the actual decision-making process at the FDA. This allows the advisory committee to get the unique patient perspective. Finally, we have the patient engagement initiative which is a brand-new program where caregivers and patients can get together and give FDA input on matters related to patient engagement at the FDA.

RDR: How can patients learn more about getting involved?
Dr. Winiecki: Part of our mission is to facilitate engagement with patients, advocates, and caregivers. We have 2 upcoming meetings—1 is on effective strategies for engaging on rare disease drug development that's on October 30th at the FDA white oak campus.  The other 1 is the CDER roadmap or program for navigating engagement with CER that's held annually by professional affairs and stakeholder engagement. The next meeting is coming up in the spring of 2018.

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