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 Agenda

Wednesday, 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



8:00 - 8:05 a.m.

Opening Remarks

Nikolay Nikolov, M.D.

8:05 - 8:20 a.m.

Regulatory Landscape

Carolyn Yancey, M.D.

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.

8:20 - 8:30 a.m

Parent/Patient Perspective

Vincent Delgaizo

Session 1

Disease Similarity in pJIA and RA

Session 1 Video


8:40 - 9:10 a.m.

Polyarticular JIA: nomenclature, presentation, and relationship to RA

Peter Nigrovic, M.D.

9:10 - 9:25 a.m.

Treatment paradigm and landscape of products used in pJIA

Dan Lovell, M.D.

9:25 - 9:55 a.m.

Panelists: Carolyn Yancey, Peter Nigrovic, Ricardo Suehiro, Skip Nelson, Dan Lovell, Guy S. Eakin

Moderators: Rachel Glaser, M.D., and Robert Colbert, M.D.

9:55 - 10:10 a.m.



Session 2

Dose Selection and Response Similarity in pJIA and RA

 Session 2 Video


10:10 - 10:30 a.m.

Landscape of pJIA drug development: Regulatory perspective

Jianmeng Chen, Ph.D.

10:30 - 10:45 a.m.

Exposure and response comparisons: RA vs PJIA

Renu Singh, Ph.D.

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.

11:05 - 11:25 a.m.

Use of exposure-response information in pediatric drug development

Marc Gastonguay, Ph.D.

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

12:15 - 1:00 p.m.


Session 3

Confirmation of Efficacy and Assessment of Safety

Session 3 Video


1:00 - 1:20 p.m.

"Learn & confirm" in JIA: Modeling and Simulations to Support Development and Extrapolation

Nael Mostafa, Ph.D.

1:20 - 1:50 p.m.

  1. Bayesian borrowing of data (adult/historical), Active control trials
  2. Enrolling pediatric patients into adult trials

Rebecca Rothwell, Ph.D.

Lisa Imundo, M.D.

1:50 - 2:05 p.m.

PJIA safety assessment considerations

Laura Schanberg, M.D.

2:05 - 2:25 p.m.

Challenges with conducting pediatric trials in pJIA

Industry Perspective: Bolanle Akinlade, M.D.

Academia Perspective: Hermine Brunner, M.D.

2:25 - 3:15 p.m.

Panel Discussion and Q/A
Panelists: Nikolay Nikolov, Rachel Glaser, Lisa Rider, Lisa Imundo, Vincent Delgaizo, Laura Schanberg, Hermine Brunner, Ricardo Suehiro, Nael Mostafa

Moderators: Rebecca Rothwell, Ph.D., and Dan Lovell, M.D.

3:15 - 3:30 p.m.



Session 4

Moving Forward

Session 4 Video


3:30 - 3:40 p.m.

Exposure-matching vs. confirmation of efficacy: Pros, cons, and remaining uncertainties

Nikolay Nikolov, M.D.

3:40 - 4:35 p.m.

Panel Discussion and Q/A
Panelists: Nikolay Nikolov, Dawn Territo, Laura Schanberg, Mara Becker, Hermine Bruner, Issam Zineh, Nael Mostafa, Vikram Sinha

Moderators: Lynne Yao, M.D., and Lisa Rider, M.D.

4:35 - 4:55 p.m.

Summary Remarks

Session Chairs

4:55 - 5:00 p.m.

Closing Remarks

Nikolay Nikolov, M.D.