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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

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