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#226: US and EU Orphan Drug Regulations: Shaping the Ecosystem for Rare Diseases
Tuesday June 17, 2025 10:30am - 11:30am EDT
146BC
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-25-573-L04-P; CME 1.00; RN 1.00

The US Orphan Drug Act and the European regulatory framework have accelerated orphan medicine development, boosting investments in rare disease treatments. This session will gather key stakeholders to discuss successes a and challenges

Learning Objectives

Discuss the impact of regulatory policies on the rare disease ecosystem; Discuss the challenges and opportunities associated with orphan drug regulations; Examine the role of regulatory frameworks in fostering innovation and access to therapies for rare diseases; Explain the importance of collaboration between regulatory agencies, industry stakeholders, and patient advocacy groups.

Chair

Imran Shah, PhD

Speaker

US and EU Orphan Drug Regulations: Shaping the Ecosystem for Rare Diseases
Anabela Marcal, PharmD

Panelist
Amy Comstock Rick, JD

Panelist
Carolyn Shore, PhD

Panelist
Victoria Gemme, MBA, MS


Speakers
avatar for Victoria Gemme

Victoria Gemme

Director, Policy and Regulatory Affairs, National Organization for Rare Disorders, United States
Victoria Gemme is a research associate at the Duke-Margolis Center for Health Policy, where she works on a range of policy topics related to medical product development and regulation. Prior to Duke-Margolis, Victoria worked at the Cystic Fibrosis Foundation where she oversaw a diverse... Read More →
avatar for Imran Shah

Imran Shah

SVP & Head, Global Regulatory, R&D Quality and Safety (RQS), EMD Serono, United States
avatar for Carolyn Shore

Carolyn Shore

Director, National Academies, United States
Carolyn Shore serves as global health lead and director of the Forum on Drug Discovery, Development, and Translation at the National Academies of Sciences, Engineering, and Medicine. Before joining the National Academies, Carolyn was an officer on Pew’s antibiotic resistance project... Read More →
SM

Segundo Mariz

European Medicines Agency, Netherlands
AC

Amy Comstock Rick

Director of Strategic Coalitions, Rare Disease Innovation Hub, FDA, United States
https://www.fda.gov/industry/fda-rare-disease-innovation-hub/amy-comstock-rick-jd
Tuesday June 17, 2025 10:30am - 11:30am EDT
146BC Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  09: Regulatory, Session

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