SADS Externally-led Patient Focused Drug Development
Thank you to all who participated, viewed, commented and supported in Tuesday’s June 11 2024 EL-PFDD for LQTS and CPVT. We’re thankful for your willingness to share details of your experience living with LQTS and CPVT. This meeting was impactful because of your vulnerability and commitment to making a change.
From the bottom of our hearts, please accept our gratitude for you and the entire SADS community. You truly showed the FDA and researchers the importance of new and improved treatments for these conditions.
The subsequent “Voice of the Patient: CPVT” and “Voice of the Patient: LQTS” report is a compilation of the testimonies and survey responses of individuals affected by these conditions from all around the world and is the first-ever report of its kind for inherited heart conditions.
EL-PFDD for LQTS and CPVT Video Recording
Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT) Voice of the Patient Report
Long QT Syndrome (LQTS) Voice of the Patient Report
Externally-led Patient-Focused Drug Development meetings give FDA and other key stakeholders, including medical product developers, health care providers, 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 our review of a marketing application.
Click here to learn more about Externally-led Patient-Focused Drug Development meetings.
Our Sponsors
These generous sponsors have made this meeting possible…On behalf of the SADS Foundation, thank you!
FAQ – Frequently Asked Questions
- Is this meeting free? Absolutely – we want all community members to participate
- Can families outside of the USA participate? Yes – the meeting is open to all LQTS and CPVT 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