Roundtables

Roundtable 02: Patient Recruitment for Neurodegenerative and Neuromuscular Clinical Trials

• How to reach more patients: clinical trial agnostic and clinical trial specific approaches
• How Patient Navigators play a crucial role in the patients’ CT journey
• Understand how to involve all stakeholders through the use of platform

Jytte van Huijstee, Director Clinical Trial Operations, myTomorrows

Terri Ellsworth, Patient Advocate Partnerships, myTomorrows

Dennis Akkaya, Chief Commercial Officer, myTomorrows

Madeleine Carrier, Patient Navigator, myTomorrows

Roundtable 04: Running EAP’s in sanctioned countries

Stuart Bell, Senior Vice President, Inceptua Early Access, Inceptua Group

Paul Stanton, Senior Director, Global Strategy, Early Access, Inceptua Group

Mark Bainbridge, Senior Director, Business Development, Early Access, Inceptua Group

Roundtable 05: Forecasting & business development: Sizing the market opportunity

Joe Musumeci, President, BluePrint Orphan

Chelsea Catsburg, Engagement Manager, BluePrint Orphan

Roundtable 06: Meeting the challenges of fast-track approval for orphan drugs

Jon Fryzek, Practice Director, EpidStrategies, A Division of ToxStrategies

Amy Kimzey, Toxicologist, EpidStrategies, A Division of ToxStrategies

Lauren Bylsma, Senior Epidemiologist, EpidStrategies, A Division of ToxStrategies

Naomi Sacks, Principal Scientist, HEORStrategies, A Division of ToxStrategies LLC

Gary M Hutchinson, SVP, Cold Chain Engineering, Modality Solutions

Roundtable 10: Grassroots to Science-Ready: Patient-Created Research

Moderated by Cushing's Support & Research Foundation (CSFR)

Leslie Edwin, President, Cushing's Support & Research Foundation

Gretchen Jordan, Associate Director, Cushing's Support & Research Foundation

Roundtable 03: RDCA-DAP task forces: How to facilitate drug development in rare disease?

Alexandre Bétourné, Executive Director, Critical Path Institute

Kasey Woleben, Founder, Cure Mito Foundation/Rare Village

Heidi Grabenstatter, Science Director, Critical Path Institute

Sophia Zilber, Board member, patient registry director, Cure Mito Foundation

Roundtable 04: Evolving the value proposition toward equity for adults living with rare conditions

Jamie Ring, Founder, Jamie Ring Advocacy Consulting

Alexis Elder, Board Member, Cure VCP Disease

Gemma Mayman, Specialty Care Patient Solutions and Engagement Lead, Pfizer

Lindsey Wahlstrom-Edwards, Partnerships Lead, Sano Genetics

Durhane Wong-Rieger, President, Chief Executive Officer & Chair, Canadian Organisation for Rare Disorder & Rare Diseases International

Lauren Holder, Producer/Host, Help 4 HD Live, Help 4 HD

Cynthia Cassandro, Vice President, Patient Advoacy, Vigil Neuroscience

Ronenn Roubenoff, Consultant, Ronenn Roubenoff

Roundtable 05: Breaking down barriers for individualized medicines for rare disease

Irina Antonijevic, Chief Medical Officer, EveryONE Medicines

Deb Gouveia, SVP, Development Operations, EveryONE Medicines

Roundtable 09: Radical Inclusion

Richie Kahn, Co-Founder & Principal, Canary Advisors

Donna Mackey, Vice President, Clinical Operations, Akouos, Inc.

Why Investing in Rare? Innovative Solutions for Health Systems Financing

Rare diseases pose unique challenges to healthcare systems worldwide due to their low prevalence and complex treatment requirements. Traditional financing mechanisms often fail to adequately address the needs of patients with rare diseases, leading to disparities in access to treatment and care. However, innovative solutions are emerging to bridge this gap and ensure equitable access to healthcare for individuals affected by rare diseases. This session will explore the importance of investing in rare diseases and showcase innovative financing strategies that can support health systems in providing comprehensive care to patients with rare diseases.

Moderator:
Veronica Lopez Gousset, MPH, Founder and President, VLG Global

Anne-Sophie Chalandon, Head of Global Public Affairs, Rare Diseases and CGT Policies, Sanofi

Roberto Iunes, Senior Health Economist, World Bank

Alexandra Heumber Perry, Chief Executive Officer, Rare Diseases International

James Chambers, Associate Professor, Tufts Medical Center