Drugs

Public Workshop on Assessment of Neurocognitive Outcomes in Inborn Errors of Metabolism and Advancing the Development of Pediatric Therapeutics: Assessment of Neurocognitive Outcomes

Meeting Information:

FDA’s Division of Gastroenterology and Inborn Errors Products and Division of Pediatric and Maternal Health in CDER, and the Office of Pediatric Therapeutics in the Office of the Commissioner are announcing a 2-day public workshop, “Assessment of Neurocognitive Outcomes in the Inborn Errors of Metabolism” and “Advancing the Development of Pediatric Therapeutics: Assessment of Pediatric Neurocognitive Outcomes.” Day 1 of the workshop will focus on approaches for assessing the efficacy of therapeutic products based on neurocognitive outcomes in patients diagnosed with inborn errors of metabolism disorders. The session will address the role of natural history studies and methodological approaches for selecting appropriate assessment scales and standardizing neurocognitive assessments. On Day 2 of the workshop, participants will discuss identification of signals in animal studies and clinical trials that warrant further clinical investigation and testing that may be predictive of neurocognitive outcome in children. Participants will also discuss strategies and methods to address the challenges of assessing long-term neurocognitive outcomes for products used to treat pediatric patients.

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The public workshop will be held on:
 
Date:     
April 16 and 17, 2015
 
Time:
 8:00 am to 5:00 pm
 
Location: 
The public workshop will be held at the FDA White Oak Campus, 10903 New Hampshire Ave., Bldg. 31, the Great Room (Rm. 1503A), 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 http://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.
 
Registration
Participation can be either in person attendance or by webcast. There is no fee to attend the public workshop, but attendees should register in advance. Space is limited, and registration for in person attendance will be on a first-come, first-served basis. Persons interested in attending this workshop must register by sending an email to Neurocognitive_workshop@fda.hhs.gov before March 31, 2015. Please include: 1) first and last name, 2) contact phone or email, 3) live attendance or via webcast 4) indicate if you plan to attend Day 1, Day 2 or both days. For those without Internet access, please contact Denise Pica-Branco at denise.picabranco@fda.hhs.gov to register. Onsite registration will not be available.
 
More Information
Contact for:
 
Day 1: CDR Matt Brancazio, (301) 796-5343, email: matthew.brancazio@fda.hhs.gov
 
Agenda Thursday, April 16, 2015 Assessment of Neurocognitive Outcomes in the Inborn Errors of Metabolism (PDF - 123KB)

Links for Webcast:Links for Webcast: April 16 (day 1) - https://collaboration.fda.gov/anoapril2015/

Slides (Zip file) (PDF - 15,541KB)

Transcript of the Meeting April 16, 2015 (PDF - 490KB)

April 16 Webcast Links
 


Day 2: Denise Pica-Branco, (301)796-1732; FAX (301)796-9858, e-mail denise.picabranco@fda.hhs.gov

Agenda Friday, April 17, 2015 Evaluation of Long-term Neurocognitive Development in Pediatrics (PDF - 54KB)

Links for Webcast:April 17 (day 2) - https://collaboration.fda.gov/apno2015/

Transcript of the Meeting April 17, 2015 (PDF - 518KB)

April 17

Agenda and Discussion Outline (PDF - 53KB)

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