FDA Oncology Center of Excellence Public Workshop: Product Development in Hemophilia December 6, 2018

Workshop | In Person

• Date: December 6, 2018
• Location: White Oak Campus: The Great Room; Conference Center; 10903 New Hampshire Ave; Building 31, Room 1503; Silver Spring, MD 20993; United States

• Organized By: Oncology Center of Excellence

Twitter: #OCEhemophilia18

Date: December 6, 2018

Time: 8:30 am to 4:30 pm

Location: FDA White Oak Campus
Building 31, Room 1503 – Great Room
10903 New Hampshire Avenue
Silver Spring, MD 20993
FDA Campus Information, (e.g., local airports, directions, and local hotels)

Background:

The public workshop "Product Development in Hemophilia" was organized by the FDA’s Oncology Center of Excellence in collaboration with the Center for Biologics Evaluation and Research (CBER) and the Center for Drug Evaluation and Research (CDER). The workshop discussed issues related to development and regulation of novel hemophilia products. This public workshop was intended to provide a platform for engaging in a discussion with experts in hemophilia treatment, patients, and caregivers regarding further development of patient-experience and patient-reported outcomes for use in clinical trials, reliable and interpretable measurements of factor VIII/IX activity levels for gene therapy products, to discuss the need for long-term safety assessments in gene therapy clinical trials, and discern when to enroll pediatric patients in gene therapy trials.

Webcast Replay:

FDA archived workshop webcast is available at links below:

• Opening & Session 1
• Sessions 2 & 3
• Session 4
• Session 5 & Closing

Workshop Transcripts:

• 12-06-18 FDA Public Workshop (Condensed Transcript) (PDF - 4.77MB)
• 12-06-18 FDA Public Workshop (Full Transcript) (PDF - 1.28MB)

Workshop Information:

• Agenda (PDF - 130KB)
• List of Speakers and Panelists (PDF - 201KB)

Contact:

Joan Ferlo Todd, RN
Regulatory Health Project Manager
Office of Hematology and Oncology Products
Center for Drug Evaluation and Research, (CDER)
U.S. Food and Drug Administration
White Oak 22|Rm. 2139| 10903 New Hampshire Ave, Silver Spring, MD 20993
Joan.Todd@fda.hhs.gov

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Event Materials

Title	File Type/Size
OCE public workshop product development in hemophilia transcript condensed	pdf (4.77 MB)
OCE public workshop on product development in hemophilia full transcript	pdf (1.28 MB)
OCE public workshop on product development in hemophilia agenda	pdf (129.46 KB)
OCE public workshop on product development in hemophilia speakers and panelists	pdf (200.99 KB)

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