Skip to content

Session 4: Regulatory Landscape and Future Directions for Neonatal and Rare Disease Drug Development



Panel Discussion: Regulatory Perspective: How can Expedited Programs, Incentive Pathways, and other Regulatory Initiatives be Leveraged for Neonatal and Rare Disease Drug Development?

Moderator:
Cynthia Rothblum-Oviatt, PhD
Rare Diseases Team (RDT), Division of Rare Diseases and Medical Genetics (DRDMG), CDER, FDA

Panelists:

Amy C. Rick, JD
Rare Disease Innovation Hub, FDA

Erika N. Torjusen, MD, MHS
Office of Orphan Products Development (OOPD), OCMO/OC/FDA

Janet Maynard, MD, MHS
Office of Rare Diseases, Pediatrics, Urology and Reproductive Medicine (ORPURM), CDER, FDA

Najat Bouchkouj, MD
Office of Therapeutic Products (OTP), Center for Biologics Evaluation and Research (CBER), FDA

Ralph Bax, MD, MA
Paediatric Medicines Office, Scientific Evidence Generation Department, Human Division, EMA

Panel Discussion: Stakeholder perspective: Lessons Learned, Gaps and Future Directions

Moderator:
Gerri Baer, MD
Division of Hepatology and Nutrition (DHN), CDER, FDA

Panelists:

Yuliya Yasinskaya, MD
DRDMG, CDER, FDA

Annapurna Poduri, MD, MPH
Harvard Medical School

Susan McCune, MD
Scendea

Antonello Pileggi, MD, PhD, MSCTI
NICHD, NIH

Ronald J. Bartek
Friedreich’s Ataxia Research Alliance (FARA)

Betsy Pilon
Hope for HIE

Leave a Comment

Your email address will not be published. Required fields are marked *