Clinical Development & Regulatory

The pivotal role patient advocacy organizations play in driving rare disease therapeutic development

• Understand how patient advocacy organizations drive therapeutic development in the rare disease space
• How to effectively engage and partner with patient advocacy organizations to develop meaningful treatments
• Gain insight into the “how, when and where” patient advocacy can significantly impact the drug development process

Neena Nizar, Assoc. Director, Patient Advocacy Strategy, ICON Centre for Rare Diseases; President, The Jansens Foundation

Jana Benesh, Director of Therapeutic Expertise, ICON Centre for Rare Diseases

Is there a need for convergence in the use of scientific evidence by decision makers in healthcare?

Moderator:
Jean-Pascal Fournier-Roussy, Head, Global Public Affairs, Rare Diseases & Rare Blood Disorders, Sanofi

Annie Kennedy, Chief of Policy, Advocacy, & Patient Engagement, EveryLife Foundation for Rare Diseases

Patient-first innovation: FDA shifting rare diseases endpoints

Jamie Sullivan, Vice President of Public Policy, EveryLife Foundation for Rare Diseases

Julie Breneiser, Executive Director, Gorlin Syndrome Alliance

Rare but not forgotten: Challenges and opportunities for rare disease drugs

Samantha Zappia, MS, RAC, Principal Consultant, SK Regulatory Solutions

Mike Fusakio, Lead Consultant, Regulatory Affairs, Halloran Consulting Group

Navigating difficult pathways in rare drug development: How, when and why sponsors should engage with FDA ombuds

Moderator:
Jennifer McNary, Founder, One Rare

Sherry Lard, PhD, Associate Director for Product Management (ADPM), U.S. Food and Drug Administration

Putting science first in rare disease studies

Anthy Tsatoumas, Manager, Project Management, Veristat

Robin Bliss, VP Strategic Consultant, Veristat

Engaging minority communities: Collaborative practices and long-term success

Moderator:
Neena Nizar, Assoc. Director, Patient Advocacy Strategy, ICON Centre for Rare Diseases; President, The Jansens Foundation

Deborah Requesens, Director, JumpStart, Orphan Disease Center, University of Pennsylvania, Perelman School of Medicine

Victoria Arteaga, Co-founder, SHER Hispanic Society for Rare Diseases

Mary McGowan, Chief Executive Officer, Foundation for Sarcoidosis Research

RARE Revolution: Authentic storytelling in a challenging regulatory landscape

Nicola Miller, Editor in Chief, RARE Revolution Magazine

Geoffrey Case, Digital Editor, RARE Revolution Magazine