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Tuesday, June 17
 

10:30am EDT

#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

1:45pm EDT

#244: How Medical Writing and Regulatory Strategy Can Support Accelerating the Development of Rare Disease Therapies: Insights from the FDA’s START Program Pilot
Tuesday June 17, 2025 1:45pm - 2:45pm EDT
151A
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-25-582-L04-P; CME 1.00; RN 1.00

This session will explore the FDA START program for accelerating rare disease therapies. Three speakers from companies involved in the START pilot and an FDA representative will provide insights.

Learning Objectives

Explain the objectives of the FDA START Program and its role in supporting rare disease development; Discuss how the cadence of medical writing and regulatory affairs shifts for those participating in the START program.

Chair

Robin Whitsell

Speaker

Panelist
Wei Liang, PhD

Panelist
Quyen Tran, PharmD

Panelist
Paulette Robinson, PhD, RAC

Panelist
Madiha Khalid, PhD

Panelist
Jhon Cores, PhD


Speakers
avatar for Jhon Cores

Jhon Cores

Associate Director, Medical Writing, Moderna, Inc, United States
Jhon is an Associate Director of Medical Writing at Moderna Inc., where he serves as the Medical Writing lead for various rare disease an oncology programs. He has 10 years of experience in the field working in different sectors, including university startups and contract research... Read More →
avatar for Madiha Khalid

Madiha Khalid

Senior Director, Medical Writing, Larimar Therapeutics, United States
Madiha is a pharmaceutical industry professional with a background in fundamental bench research. Her experience as a regulatory medical writer working with small, mid-size, and large pharmaceutical companies over the past 10 years has given her an in-depth understanding of all phases... Read More →
avatar for Wei Liang

Wei Liang

Pharmacologist, OTAT, CBER, FDA, United States
Dr. Liang is a Pharmacology/Toxicology Reviewer in the Office of Tissues and Advanced Therapies (OTAT) in the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA). She joined OTAT in 2006. Her primary focus is the review of preclinical testing... Read More →
PR

Paulette Robinson

Senior Director, Regulatory Affairs, Neurogene Inc., United States
Paulette Robinson, PhD, RAC is the Senior Director, Regulatory Affairs at Neurogene Inc. Her regulatory affairs experience has focused on gene therapies in rare central nervous system and ophthalmic indications. She continues to lead the global regulatory strategy for the NGN-401... Read More →
QT

Quyen Tran

Science Policy Analyst, OND, CDER, FDA, United States
Dr. Quyen Tran is a Science Policy Analyst on CDER’s Rare Diseases Team. Dr. Tran joined the FDA in 2017 and has worked in the Office of New Drugs and Office of Medical Policy, CDER. She has notable experience in rare disease drug development, policy development and implementation... Read More →
avatar for Robin Whitsell

Robin Whitsell

President, Whitsell Innovations, Inc., United States
Founder and president of Whitsell Innovations, Inc., a medical writing firm headquartered in Chapel Hill, NC, Ms. Whitsell has over 25 years of experience, specializing in global submission strategy, regulatory medical writing, emerging technologies, and data visualization. Prior... Read More →
Tuesday June 17, 2025 1:45pm - 2:45pm EDT
151A Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  04: MedAffairs-SciComm, Session
 
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143ABC
144ABC
145AB
146ABC
146BC
147AB
150AB
151A
151B
152AB
201
202A
204ABC
206
207A
207B
209ABC
Ballroom
Ballroom Foyer 3rd Floor
EH
Exhibit Hall / Zone A
Exhibit Hall / Zone B
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Exhibit Hall Lunch
Innovation Theater 1
Innovation Theater 2
Innovation Theater 3
L Street Bridge
Nationals Park
Planet Word
Room 145-147 Lobby
Salon B
Salon C
TBD
Virtual
West Salon
  • 00: Plenary
  • 01: ClinSafety-PV
  • 02: ClinTrials-Ops
  • 03: Data-Tech
  • 04: MedAffairs-SciComm
  • 05: Patient-Impact-Product-Dev
  • 06: PersonalizedMed-ComboProd-Diagnostics
  • 07: ProjectManagement-StrategicPlanning
  • 08: RD-Quality-Compliance
  • 09: Regulatory
  • 10: RegCMC-Product Quality
  • 11: Statistics-Data Science
  • 12: ProfDevelopment
  • 13: Spotlight
  • 14: DIAmond
  • 15: Content-Hubs
  • 16: Community-Rounds
  • 17: Posters
  • 18: InnovationTheaters
  • 19: Networking Opportunities
  • 20: Short-Courses
  • Level
  • Advanced
  • Basic
  • Intermediate
  • Keyword
  • Advanced Analytics
  • Artificial Intelligence (AI)
  • Bioethics
  • Biomarkers
  • Cell and Gene Therapy
  • Clinical Trial Design
  • Diagnostics
  • Digital Technology
  • Global Perspectives
  • Non-CE
  • Patient Experience
  • Payers
  • Pediatrics
  • Rare Disease
  • Real-World Data (RWD) / Real-World Evidence (RWE)
  • Regulator Perspectives
  • Regulatory Collaboration
  • Regulatory Convergence and Harmonization
  • Regulatory Reliance
  • Student Programming