Rett Perspectives to Be Shared in Online Meeting With FDA

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by Steve Bryson, PhD |

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Rett syndrome community members are invited to participate in a public meeting with the U.S. Food and Drug Administration (FDA) to share their perspectives about the condition and help influence the development of new therapies.

Hosted by the International Rett Syndrome Foundation and the Rett Syndrome Research Trust, the Rett Syndrome Externally Led Patient-Focused Drug Development Meeting (EL-PFDD) will take place online on March 11 from 10 a.m. to 3 p.m. EST.

Parents, caregivers, and family members of people with Rett in the U.S. — whose perspectives are crucial to understanding treatment benefits and risks, according to the FDA — are encouraged to attend, along with anyone interested in Rett syndrome, including biopharmaceutical company staff, Rett clinicians, scientists, and patient advocates.

To register, click here to receive an email with a link to the meeting.

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EL-PFDD meetings aim to give the FDA and other essential stakeholders — such as pharmaceutical companies, healthcare providers, and federal partners — an opportunity to hear from patients, caregivers, families, and patient advocates about the impact of the disease.  Participants are asked to share Rett’s impact on their daily lives, their experience with current treatments, and the symptoms patients experience.

The FDA calls the involvement of patients, caregivers, and families key to the meeting’s success, helping federal regulators decide about developing treatments and supporting its application review to approve new therapies.

The meeting will open with an overview of the disease and clinical trials by Eric Marsh, MD, PhD, a pediatric neurologist in the division of neurology at Children’s Hospital of Philadelphia, in Pennsylvania.

The morning session will feature panelist caregivers offering pre-recorded testimonials about Rett symptoms, and which ones have the most impact on patients. Panelists also will discuss how symptoms change as patients age, and other concerns.

Rett symptom management, treatment efficacy, and complications also will be discussed by panelists during the afternoon session, and participants will have the opportunity to describe their ideal Rett therapy, in the absence of a cure.

Both sessions will be accompanied by polling questions, which participants can use their devices to respond to, along with discussions.

Participants can submit comments and questions up to 30 days after the meeting.

A report of the meeting, titled “Voices of the Patient” and containing a summary of caregiver and audience testimonials, polling data, and written comments will be submitted to the FDA. The report also will be made available to the public.

A webinar about the meeting posted by the Rett Syndrome Research Trust can be found here.