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The Food and Drug Administration’s Office of Pediatric Therapeutics is announcing a 1-day workshop, “Advancing the Development of Pediatric Therapeutics (ADEPT 6): Pediatric Clinical Trial Endpoints for Rare Diseases With a Focus on Pediatric Patient Perspectives.”  The purpose of this workshop is to discuss pediatric patient-specific engagement in the development of clinical trial endpoints for rare diseases.  

In this workshop, FDA will obtain the pediatric patient perspective on their disease/condition and what is most important to consider when designing rare disease trials.  There will also be discussion regarding patients’ thoughts on clinical endpoints that are currently being used in clinical trials, potential areas of innovation, and how to create processes that might include pediatric patients and their caregivers as collaborators in endpoint development in early stages of medical product development (e.g., protocol design).  

The morning session will focus on identifying endpoints that capture important aspects of how pediatric patients feel and function.  The afternoon session will focus on steps for development of clinical outcome assessment (COA) tools for use in pediatric patient populations and the potential role of child and youth friendly technology in endpoint assessments.

Location:

The public workshop will be held at the FDA White Oak Campus, 10903 New Hampshire Ave., Building 31 Conference Center, the Great Room (1503-A), Silver Spring, MD 20993-0002.  Entrance for the public workshop participants (non-FDA employees) is through Bldg. 1 where routine security check procedures will be performed.  For parking and security information, please refer to Public Meetings at the FDA White Oak Campus.

Registration:

Persons interested in attending this workshop must register online using this registration link. Please provide complete contact information for each attendee, including name, title, affiliation, email, and telephone.  Registration for onsite participation or via webcast is free and based on space availability, with priority given to early registrants.  Early registration is recommended because seating is limited; therefore, we suggest you register before close of business on November 5, 2019.

Workshop Materials:

• Federal Register Notice (October 10, 2019)
• Final Agenda
• Speakers' Bios
• Patient Advocates' Bios
• Presentations 

Webcast:

• Webcast Part 1
• Webcast Part 2
• Webcast Part 3
• Webcast Part 4

More Information:

Contacts:

Terrie L. Crescenzi, RPH
(301) 796–8646
email: [email protected]

Betsy Sanford
(301) 796-8659
email: [email protected]

FAX: 301-847-8640.

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