Join the RAPS Philadelphia Chapter for an engaging in-person discussion examining the rapidly evolving regulatory landscape for pediatric rare disease drug development. Bringing together experts from industry, academia, contract research organizations (CROs), public-private partnerships, and patient advocacy, this session will explore how recent regulatory and policy developments are influencing the way therapies for children with rare diseases are developed and evaluated.
The program will begin with a focused overview of key developments from the past two years and their implications for pediatric rare disease development. The presentation will highlight evolving approaches to evidence generation, innovative clinical trial designs, natural history and external control data, biomarkers and drug development tools, model-informed approaches, patient-focused drug development, and regulatory pathways for advanced and individualized therapies.
Building on this foundation, a moderated panel will move the conversation from policy to practice. Panelists will share perspectives on how evolving regulatory approaches are being translated into development strategies, including early engagement with health authorities, pediatric study planning, endpoint selection, evidence generation in small patient populations, global development considerations, and collaboration with patients and other stakeholders.
Throughout the discussion, the panel will consider a central question: How do we build regulatory strategies that are rigorous enough to support confident decisions, yet fit for the realities of pediatric rare disease development?
Panelists will discuss where recent regulatory developments are creating new opportunities, where uncertainty and implementation challenges remain, and what regulatory professionals can do differently today to position pediatric rare disease programs for success.
The session will conclude with an interactive audience Q&A and a forward-looking discussion on what comes next for pediatric rare disease regulation. Whether you are a regulatory professional, researcher, clinician, student, patient advocate, or work in drug development, this event offers an opportunity to understand what has changed, consider what it means for development strategy, and gain practical insights into advancing therapies for children with rare diseases.
Agenda
1. Networking (Food & Soft Drinks provided): 5:30-6:15
2. Panel: 6:15-7:30
3. Networking: 7:30-8
Location
Ensemble at the Navy Yard
150 Rouse Blvd
Philadelphia, PA 19112
Street parking available outside of venue. Generally free but always check signs. Septa Bus Route 45 also services the Navy Yard.
Cancellations and Refunds
RAPS reserves the right to cancel this program at its sole discretion. RAPS will not be responsible for travel or other costs incurred due to cancellation. All cancellation requests must be submitted in writing to [email protected]. Cancellations will receive a full refund minus a 20% administrative fee. RAPS is unable to accept cancellations by phone.
Substitutions
Paid registration substitutions may be accepted with written approval from RAPS for requests received before the start of the event. To transfer a registration, email [email protected] with the event title, name of the original registrant and the contact information for the new attendee.
Proof of Attendance
A certificate of attendance can be downloaded from the RAPS Learning Portal following the event.
Questions
Contact the RAPS Support Center:
Call +1 301 770 2920, ext. 200 (8:30 am–5:30 pm EST, Monday–Friday) or email [email protected].
Speakers
Eric Marsh, MD, PhD
Dunni Odumosu MS
Lee Severson PharmD, MBA
Alicia Baker McDowell DRSc, MS, EMBA
Patti Lehigh PhD, RAC
Moderator
Anna-Marika Bauer MRA, RAC-Drugs
