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On March 11, 2022 the International Rett Syndrome Foundation and the Rett Syndrome Research Trust hosted an Externally-Led Patient-Focused Drug Development meeting to provide an opportunity for the Food and Drug Administration (FDA), biopharmaceutical companies and other key stakeholders to hear directly from families about the symptoms that matter most to them, the impact the disease has on our children’s daily lives, our experiences with currently available treatments and our hopes for the future.

The Rett community responded enthusiastically with articulate, poignant and thoughtful perspectives.  If you weren't able to particpate in the live meeting we invite you to watch the meeting.

Voice of the Patient Report

The meeting itself and the comments that were submitted post meeting have been summarized in the Voice of the Patient Report which can be accessed by our community, and by clinicians, scientists and drug development companies. The report will also be available on the FDA website.

Download the report.

What is an externally-led patient-focused drug development meeting?

Externally-led Patient Focused Drug (EL-PFDD) meetings give the Food and Drug Administration (FDA) and other key stakeholders — including medical product developers, health care providers, and federal partners — an important opportunity to hear directly from patients, their families, caregivers, and patient advocates about the symptoms that matter most to them, the impact the disease has on patients’ daily lives, and patients’ experiences with currently available treatments. This input can inform FDA’s decisions and oversight both during drug development and during their review of a marketing application for a new drug.

Learn more about externally-led Patient-Focused Drug Development meetings.

January 27th Webinar Discusses Importance of EL-PFDD