February 3rd, 2022

Externally-Led Patient-Focused Drug Development Meeting on Dravet Syndrome

On February 3, 2022, DSF held a virtual Externally-Led Patient-Focused Drug Development meeting on Dravet syndrome. This meeting was a long-awaited opportunity for the Dravet syndrome patient community to educate representatives of the Food and Drug Administration (FDA) and pharmaceutical companies, as well as academic researchers and clinicians, about the challenges that individuals living with Dravet syndrome face daily. Our objective was to give patient families a platform to share what it means to have a Dravet syndrome diagnosis, which symptoms should be prioritized, and how this disease impacts the quality of life for the patient and family so that the FDA and pharmaceutical companies can understand the patient experience.

We would like to offer our thanks to the patient panelists, phone callers, and individuals who submitted email comments. You bravely shared your experiences with symptoms in your daily lives. With your input, we were able to document the significant physical and emotional impact for people living with Dravet syndrome and hopes for future treatments. We heard directly from caregivers of patients living with Dravet about their challenges. This information was included in the Voice of the Patient report. It will help key stakeholders understand what people living with Dravet syndrome want from future treatments and clinical trials. It will ensure that the concerns of patients are taken into account.

We appreciate the efforts of the Dravet syndrome community in making this a successful meeting!

What is an Externally-Led Patient-Focused Drug Development Meeting?

An Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting gives the 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 review of a marketing application. EL-PFDD meetings strengthen understanding of disease and treatment burden. They also raise awareness and channel engagement within the patient community. This engagement can help lead to approval of new treatments in the future.


Externally-Led Patient-Focused Drug Development Meetings – U.S. Food & Drug Administration
The Voice of the Patient: A Series of Reports from FDA’s Patient-Focused Drug Development Initiative – U.S. Food & Drug Administration
FDA Credits Recent Drug Approval to Patient Community Engagement; We Applaud the Agency for the Recognition of Its Legacy of PFDD – FDA Law Blog

This is a recording of the meeting that was streamed live on February 3rd, 2022. 

Voice of the Patient Report & Infographic

This report summarizes information from the Externally Led Patient-Focused Drug Development (EL-PFDD) meeting. It is a comprehensive document created from the feedback gathered at the meeting. From this meeting, DSF developed the Voice of the Patient report, which documents the severe disease burden and unmet medical need in patients’ own voices. It includes written statements and transcripts from the meeting which will be summarized in the report and shared in full in the appendices. Once complete, the report was submitted to the FDA’s Division of Neurology Products, Office of New Drugs, for inclusion in the framework used to evaluate future Dravet syndrome therapies, with the intent to help provide context of the disease burden. This report will serve a critical function in communicating to both FDA review staff and industry the improvements that patients want and hope to see in their daily lives, and will speak loudly and clearly to the immense unmet medical need of our community. Future applications for therapy approvals will use the report as a reference when evaluating the effectiveness of the treatment. The infographic offers top level highlights from the report.

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