SADS Externally-led Patient Focused Drug Development

June 20, 2023

“Patient-focused drug development (PFDD) is a systematic approach to help ensure that patients’ experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into drug development and evaluation. … As experts in what it is like to live with their condition, patients are uniquely positioned to inform the understanding of the therapeutic context for drug development and evaluation.”

Your voice matters

Thank you to all who attended our EL-PFDD on June 20! You can register here to watch the recording of the EL-PFDD. Comment submission is still open until July 21, and we still want to hear from YOU! All the comments we receive will be summarized in the final report for the FDA. Register below to watch; the comment form is right below the recording.

Below, hear the top 6 things you need to know about our  EL-PFDD for ARVC – and why it’s so important for our ARVC community to join the meeting.

  • ARVC families need better therapies, and this meeting is a huge step in informing health care providers, scientists, and those developing therapies about what ARVC families really need.
  • This meeting is groundbreaking because it’s the first of its kind for any of our SADS conditions.
  • Every person with ARVC who joins makes a difference. This is not just another webinar for patients to watch. Every ARVC patient and caregiver voice matters and adds value to this meeting so we need as many as possible to participate!
  • It helps the FDA make decisions about new therapies. The patient perspective is critical in helping FDA make decisions about new treatments.
  • Doctors and researchers will learn from the EL-PFDD too. The EL-PFDD meeting will be followed up by a scientific meeting a month later to allow doctors and scientists the opportunity to discuss what they learned from the patients and how to apply it.

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

Externally-Led Patient-Focused Drug Development (EL-PFDD) meetings give the Food and Drug Administration (FDA) and other key stakeholders — including researchers, biopharma companies, healthcare providers, product developers and federal regulatory 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, patients’ experiences with currently available treatments, and patient’s priorities for therapeutic outcomes. These inputs may also inform FDA’s decisions and oversight both during drug development and their reviews of marketing applications for new therapies.

Learn more from the FDA.

What happens during the meeting?

The meeting and participants, will be virtual. However, the hosts and co-moderators will be looking for the ARVC community to participate in polls, by sending in comments, or calling in live! There will be a brief 10-15 minute overview of ARVC by a clinical expert, followed by two sessions with patient panelists, callers, reading of submitted comments, and live anonymous polling.

Please plan on viewing and participating in the entire meeting, but if you can only attend part of the meeting, that would also be greatly appreciated. The meeting recording will immediately replace the live feed after the meeting ends.

What happens after the meeting?

The comments and information shared before, during, and after the meeting will be consolidated into a Voice of the Patient report. This report will be publicly available and will be provided to the FDA and to researchers developing new therapies for ARVC.

During the EL-PFDD, you’ll hear from patients and caregivers whose lives have been affected by ARVC tell their stories. Here, you can meet the panelists who will be presenting their stories during these sessions.

ARVC: Stories from the EL-PFDD

FAQ – Frequently Asked Questions

  • Is this meeting free? Absolutely – we want all ARVC community members to participate
  • Can families outside of the USA participate? Yes – the meeting is open to all ARVC families
  • What about families with deceased loved ones? We want and need to hear and learn from you as well.
  • Do I need a computer to attend? No, you can participate from your mobile device or tablet – if you can read this web page (and you are right now), you will see the meeting here.
  • What will be the primary meeting language? It will be English.
  • Will the meeting be recorded? Yes – it will be available immediately after the meeting ends on this page (hosted by YouTube so everyone can easily access it).

Do you have other questions? Please contact us

Our Supporters

Our supporters are helping us to invite families from around the globe to participate


Our Sponsors

These generous supporters have made this meeting possible…On behalf of the SADS Foundation, thank you!