Collaborative workshop hosted by the University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI) and the Food and Drug Administration (FDA)
Download the AgendaWednesday, October 2, 2019:
FDA's White Oak Campus
10903 New Hampshire Ave., Bldg. 31
Silver Spring, MD 20993-0002
Goals and Objectives:
- This workshop addressed current barriers to expeditious pJIA drug development and steps to overcome them.
- Specific topics included extrapolation, trial design considerations, dose selection, modeling and simulation, and level of evidence required to establish safety and effectiveness in pediatric patients with pJIA.
For speakers who have given permission, the workshop slides and video links are posted below on this webpage (see hyperlinks).
Wednesday, October 2nd |
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8:00 - 8:05 a.m. |
Opening Remarks |
Nikolay Nikolov, M.D. |
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8:05 - 8:20 a.m. |
Carolyn Yancey, M.D. |
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8:20 - 8:30 a.m. |
Scientific Necessity in the Context of Pediatric Extrapolation of Efficacy from Adult Data |
Skip Nelson, M.D., Ph.D. |
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8:20 - 8:30 a.m |
Parent/Patient Perspective |
Vincent Delgaizo |
Session 1
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Disease Similarity in pJIA and RA
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Session 1 Video
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8:40 - 9:10 a.m. |
Polyarticular JIA: nomenclature, presentation, and relationship to RA |
Peter Nigrovic, M.D. |
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9:10 - 9:25 a.m. |
Dan Lovell, M.D. |
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9:25 - 9:55 a.m. |
Panelists: Carolyn Yancey, Peter Nigrovic, Ricardo Suehiro, Skip Nelson, Dan Lovell, Guy S. Eakin
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Moderators: Rachel Glaser, M.D., and Robert Colbert, M.D. |
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9:55 - 10:10 a.m. |
Break |
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Session 2
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Dose Selection and Response Similarity in pJIA and RA
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Session 2 Video
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10:10 - 10:30 a.m. |
Jianmeng Chen, Ph.D. |
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10:30 - 10:45 a.m. |
Exposure and response comparisons: RA vs PJIA |
Renu Singh, Ph.D. |
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10:45 - 11:05 a.m. |
Two Well-Controlled Phase 3 Trials in Patients With Juvenile Idiopathic Arthritis: Challenges and Lessons Learned |
Jocelyn H. Leu, Pharm.D., Ph.D. |
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11:05 - 11:25 a.m. |
Use of exposure-response information in pediatric drug development |
Marc Gastonguay, Ph.D. |
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11:25 - 12:15 p.m. |
Panel Discussion and Q/A Panelists: Nikolay Nikolov, Hermine Brunner, Mara Becker, Suzette Peng, Vikram Sinha, Chandrahas Sahajwalla, Yaning Wang, Rebecca Rothwell |
Moderators: Lily Mulugeta and Marc Gastonguay |
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12:15 - 1:00 p.m. |
Lunch |
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Session 3
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Confirmation of Efficacy and Assessment of Safety
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Session 3 Video
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1:00 - 1:20 p.m. |
"Learn & confirm" in JIA: Modeling and Simulations to Support Development and Extrapolation |
Nael Mostafa, Ph.D. |
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1:20 - 1:50 p.m. |
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Rebecca Rothwell, Ph.D. Lisa Imundo, M.D. |
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1:50 - 2:05 p.m. |
Laura Schanberg, M.D. |
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2:05 - 2:25 p.m. |
Industry Perspective: Bolanle Akinlade, M.D. Academia Perspective: Hermine Brunner, M.D. |
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2:25 - 3:15 p.m. |
Panel Discussion and Q/A |
Moderators: Rebecca Rothwell, Ph.D., and Dan Lovell, M.D. |
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3:15 - 3:30 p.m. |
Break |
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Session 4
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Moving Forward
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Session 4 Video
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3:30 - 3:40 p.m. |
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Nikolay Nikolov, M.D. |
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3:40 - 4:35 p.m. |
Panel Discussion and Q/A |
Moderators: Lynne Yao, M.D., and Lisa Rider, M.D. |
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4:35 - 4:55 p.m. |
Session Chairs |
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4:55 - 5:00 p.m. |
Closing Remarks |
Nikolay Nikolov, M.D. |
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