Externally-Led PFDD Meeting

on Barth Syndrome

~

About

BSF is excited to have been selected by the U.S. Food and Drug Administration (FDA) to host an Externally-Led Patient Focused Drug Development Meeting on July 18, 2018 from 12:00pm - 5:00pm EDT.

The event will take place at:

Hilton Clearwater Beach Resort
400 Mandalay Ave.
Clearwater Beach, FL  33767

We welcome you whether you’re joining us in person or plan to participate through our live webcast.

Attire: Dress appropriately.
(Please do not plan on coming in directly from the beach or pool.)

For accommodations and directions to the venue, please visit Hilton Clearwater Beach Resort or call +1-727-461-3222.

On-site parking is available at a rate of $13 per day. Please inform the front desk that you are attending the PFDD meeting for the reduced rate.


Agenda

12:00pm – 12:30pm: Registration

12:30pm – 12:35pm: Welcome
Emily Milligan, MPH; Executive Director, BSF

12:35pm – 12:45pm: Opening Remarks
Celia M. Witten, PhD, MD; Deputy Director, FDA Center for Biologics Evaluation and Research (CBER)

12:45pm – 1:00pm: Clinical Overview of Barth Syndrome (BTHS)
Colin Steward, PhD, FRCP, FRCPCH; Professor of Pediatric Stem Cell Transplantation and Consultant in Bone Marrow Transplantation, Bristol Royal Hospital for Children; Medical Advisor to NHS Barth Syndrome Clinic

1:00pm – 1:15pm: Introduction to Barth Syndrome Foundation (BSF) and One Family’s Experience with BTHS
Kate McCurdy; Founding BSF Board Member and Ex- officio Member, BSF Scientific and Medical Advisory (SMAB)

1:15pm – 1:25pm: Introduction and Overview of Meeting
James Valentine, JD, MHS; Meeting Moderator

1:25pm – 1:35pm: Audience & Remote Demographic Polling

1:35pm – 2:50pm: Panel 1: Living with BTHS

  • Presentations by Five Affected Individuals and Caregivers (30 minutes)
  • Audience & remote polling (10 minutes)
  • Moderated audience discussion (35 minutes)

2:50pm – 3:05pm: Break

3:05pm – 3:15pm: FDA Comments
Scott K. Winiecki, MD; Director, Safe Use Initiative, FDA Center for Drug Evaluation and Research (CDER)

3:15pm – 3:25pm: Video of BTHS individual

3:25pm – 4:40pm: Panel 2: Current and Future Treatments

  • Presentations by Five Affected Individuals and Caregivers (30 minutes)
  • Audience & remote polling (10 minutes)
  • Moderated audience discussion (35 minutes)

4:40pm – 4:50pm: Closing Comments – What I Heard Today
Shelley Bowen; Director of Family Services & Awareness, BSF

4:50pm – 5:00pm: Closing Remarks
Elizabeth Hart, MD; Medical Officer, Division of Gastroenterology and Inborn Error Products, Office of New Drugs, CDER (10 minutes)

Download PFDD Program


Register

Register now!

Powered by Firespring