Collaborative Workshop hosted by the UMD Center of Excellence in Regulatory Science and Innovation (M-CERSI) and the U.S. Food and Drug Administration (FDA)

Audio Files

Friday, November 16: 8:00 a.m. - 5:00 p.m.

FDA's White Oak Campus
The target audience included clinicians, investigators, patients and parents of patients, device manufacturers, pharmaceutical companies, contract research organizations and regulators. Audio files and the agenda are available online.

Goals and Objectives:

  • The aim of the workshop was to discuss current barriers to expeditious pediatric IBD drug development and steps to overcome them.
  • Specific topics included a review of the legislation relevant to pediatric trials, extrapolation, trial design considerations, dose selection and the level of evidence required to establish safety and effectiveness in pediatric patients with IBD.

Session 1: Introduction

8:00-8:10 am
Opening Remarks
Jessica Lee, M.D.
8:10-8:30 am
Mona Khurana, M.D.
8:30-8:45 am
Eric Benchimol, M.D.
8:45 – 9:00 am
Epidemiology of Pediatric IBD (2)
(Data from ImproveCareNow Registry)
Richard Colletti, M.D. and Michael Kappelman, M.D.

Session 2: Dose Selection 

9:00 – 9:20  am
Dilara Jappar, Ph.D.
9:20-9:35 am
Regulatory Perspective on Dose Selection 
Anand Balakrishnan, Ph.D.
9:35-9:50 am
Case Study: Industry Perspective
Arnab Mukherjee, Ph.D. (Pfizer) 
9:50-10:10 am
Marla Dubinsky, M.D. 
10:10-10:25 am Break 
10:25-11:45 am
Panel Discussion and Q&A
Moderators:  Shirley Seo, Ph.D. and Lily Mulugeta, Pharm.D.
11:45-12:30 pm Lunch 

Session 3: Trial Design Considerations 

12:30-12:45 pm
Regulatory Perspective on IBD Trial Design
Tara Altepeter, M.D. 
12:45-1:05 pm
Margaret Gamalo-Siebers, Ph.D.
1:05-1:20 pm
Use of Exposure-Response Information to Establish Efficacy in Pediatric Patients
Marc Gastonguay, Ph.D.
1:20-1:35 pm
Case Study: Industry Perspective
Nael Mostafa, Ph.D. (Abbvie)  
1:35-2:25 pm 
Panel Discussion and Q&A
Moderators: Juli Tomaino, M.D. and Laurie Conklin, M.D.
2:25-2:35 pm

Session 4: Trial Conduct

2:35-2:50 pm
Bruce Sands, M.D. 
2:50-3:05 pm
Anne Robinson, Pharm.D. (Abbvie)
3:05-3:20 pm
Case Studies From Other Disease States 
Yodit Belew, M.D. 
3:20-3:35 pm
Caren Heller, M.D. 
3:35-4:40 pm
Panel Discussion and Q&A
Moderators: Ed deZoeten, M.D. and Jessica Lee, M.D.
4:40-5:00 pm
Jeffrey Hyams, M.D. 

For questions:

Please contact Dr. Jacquline Yancy.