Keynote Sessions

Expediting drug development for rare disease

Moderator:
Pamela Gavin, Executive Vice President, National Organization for Rare Disorders (NORD)

Jason Tardio, Chief Operating Officer, Ovid Therapeutics

Peter Marks, Director, Center For Biologics Evaluation And Research (CBER), U.S. Food and Drug Administration

Charting the path forward: Regulatory innovations and orphan drug designations in rare disease development

Robert M. Califf, Commissioner, U.S. Food and Drug Administration

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

Challenge and opportunity with the current regulatory and legislative landscape in the US

Moderator:
Rachel Smith, Global Head, Rare Disease, Parexel

Kinnari Patel, President & COO, Rocket Pharma

Peter Marks, Director, Center For Biologics Evaluation And Research (CBER), U.S. Food and Drug Administration

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

Mark Trusheim, Strategic Director NEWDIGS, Center for Biomedical System Design, Tufts Medical Center

Unlocking patient-centric insights and feedback to elevate engagement

Moderator:
Anthony Scatamacchia, Senior Vice President, Innovation, Patient Services, Eversana

Patricia Weltin, Chief Executive Officer, Beyond the Diagnosis

MARIA KIRSCH, President, Patient Services, EVERSANA

Joshua Resnikoff, Chief Executive Officer, TMA precision health

From patient finding to payer pull-through: leveraging advanced analytics to fuel the rare disease revolution

Moderator:
Brandi Greenberg, VP Marketing-Analytics & Insights, Real Chemistry

Ivan Kouchlev, Managing Principal, Real-World Data Solutions, Real Chemistry

Lindsay Murphy, Senior Group Director, Integrated Intelligence, Real Chemistry

Christine Juday, Head, Market Access, Real Chemistry

Fireside chat: Access for rare disease patients in Europe and beyond

Yann Le Cam, Founder and past-CEO, EURORDIS

Denise Scots Knight, Chief Executive Officer, Mereo Biopharma

Can you overcome complexity and deliver value for rare disease patients in the EU health ecosystem?

Sponsored by Sciensus

Moderator:
Andrew Cummins, VP Access and Reimbursement, Sciensus

Laura Smith van Carroll, Head of Insight & Advocacy, Metabolic Support UK

Grant Castor, SVP Commercial Strategy & Operations, Sentynl Therapeutics

Leslie Gordon, Medical Director, Progeria Research Foundation

Unlocking access: Transformative policies and strategies for orphan drug accessibility

Moderator:
Jayson Slotnik, Partner, Health Policy Strategies, LLC

Christoph Glaetzer, Chief Global Value and Access Officer, Johnson & Johnson

Ron Bartek, President, Co-Founder, Friedreich's Ataxia Research Alliance

Heidi Ross, Vice President of Policy and Regulatory Affairs, National Organization for Rare Disorders

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

Breaking borders: Advancing cross-state access to care for rare diseases

Moderator:
Bert Bruce, US Rare Disease Lead, Pfizer, Pfizer

Matt Salo, Former Executive Director, National Association of Medicaid Directors (NAMD), Founder and Chief Executive Officer, Salo Health Strategies

Colleen Dansereau, Senior Director - Gene Therapy Program and Office of Emerging Medical Discoveries, Boston Children's Hospital

Heidi Ross, Vice President of Policy and Regulatory Affairs, National Organization for Rare Disorders

AI and digital health for rare diseases: Making data inclusive

Moderator:
Harsha Rajasimha, Founder and Chairman, IndoUSrare

Joshua Resnikoff, Chief Executive Officer, TMA precision health

Spencer Huggett, Founder, Synapze