DSF is very excited to announce that we will be holding an Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting on Thursday, February 3, 2022.

An EL-PFDD meeting is critical to ensure the FDA has an informed understanding of the patient experience for those living with Dravet syndrome. As our community knows, the patient voice is critical throughout the clinical trial process, including when the FDA reviews data and makes regulatory decisions for new drug approvals. An EL-PFDD meeting is an opportunity for the FDA and other key stakeholders – including pharmaceutical companies, health care providers and federal partners – to hear directly from patient families, caregivers, and patient advocates about the symptoms that matter most to them and the impact a particular disease has on patients. With the lessons and takeaways from the patient community in hand, the FDA will be better equipped to review and approve therapies to meet the needs of a patient community.

The purpose of this meeting is to share our experiences and expectations of what symptoms impact our lives the most as well as what impactful treatments would look like. During the course of this meeting, there will be panelist presentations as well as discussions with people from our patient and caregiver community who will share their insights and experiences in living with Dravet syndrome, including the many health comorbidities and daily challenges that patients face. Audience participation is encouraged to allow us to obtain additional perspectives and the meeting will have live polling to capture patient viewpoints on the disease experience as well as the potential benefits, risks, and preferences for treatment.

It is very important that as many of our community members join for this virtual meeting to make the meeting as representative of our community as possible. We need the global perspective of our patient community, so we encourage you to participate whether you live inside or outside of the United States. We want the FDA to understand that Dravet syndrome has no ethnic or geographical boundaries. Clinicians and researchers are also encouraged to attend – we need your support and partnership. In addition, the meeting is also open to pharmaceutical companies interested in or already working on treatments for Dravet syndrome. The role of these companies during this meeting will be to listen and learn – this meeting is purely about the patient and family voice. However, we do want to give companies a chance to hear our voices and concerns first-hand, so that they have the information that they need to help them develop therapies that address the most burdensome symptoms of Dravet syndrome.

Following the meeting, a Voice of the Patient report will be published that summarizes the meeting and articulates the takeaways from the meeting along with the polling data. It will inform and influence both drug development and regulatory efforts so that the significant unmet therapeutic needs of people living with Dravet syndrome are better understood. This is our chance to directly educate and inform the FDA. The FDA has cited EL-PFDD meetings and their accompanying Voice of the Patient report as a key source of information they considered when reviewing a new therapy for approval.

Great attendance from our community will show the FDA how important progress and new treatments are to us. Even if you don’t have prior experience in advocacy, don’t feel comfortable speaking out, or feel you don’t have anything new to say, we encourage you to join us in whatever way you are most comfortable. There are several ways you may take an active role in the meeting:

  • Watch for registration for the meeting to open up later this year, and be sure to sign up.
  • Encourage other members of the Dravet syndrome community to register to attend via the live webcast.
  • Actively participate in the live polling during the meeting.
  • Share your perspectives and experiences via the chat box or by calling in during the meeting.