Wendy Erler | Senior Vice President, Patient Affairs
Sarepta Therapeutics

Day 1 - World Orphan Drug Congress USA 2026 @ 09:20

Building a Patient-Cantered Policy Framework for Rare Disease Drug Development

his keynote will examine how policymakers, regulators, patients, and industry can co-create strategies that embed patient insights into every stage of the drug development and access process—driving a more equitable, transparent, and sustainable ecosystem for rare disease therapies. 

Session Objectives: 

• Define what a patient-centered policy framework entails in the context of rare disease drug development. 

• Explore mechanisms for embedding patient perspectives into every stage of the regulatory and policy decision-making process. 

• Examine existing policy gaps that limit timely diagnosis, research funding, and equitable access to orphan drugs. 

• Discuss cross-sector collaboration opportunities between patient organizations, regulators, industry, and payers to shape sustainable policy reform. 

• Chart actionable next steps for integrating patient input into evolving rare disease frameworks at national and global levels. 

Wendy Erler, Senior Vice President, Patient Affairs, Sarepta Therapeutics

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

×

Annie Kennedy / Building a Patient-Cantered Policy Framework for Rare Disease Drug Development

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

A veteran leader in the rare disease patient focused drug development movement, Annie joined the EveryLife Foundation in 2018, where she’s led numerous, community-driven evidence development efforts including the National Economic Burden of Rare Disease study, The Guide to Patient Involvement in...

CloseMore

Amy Comstock Rick, Director of Strategic Coalitions, Rare Disease Innovation Hub, FDA

Day 1 - World Orphan Drug Congress USA 2026 @ 15:30

Patient- Focused Drug Development Across the Pipeline

Moderator: Wendy Erler, Senior Vice President, Patient Affairs, Sarepta Therapeutics

Camille Bedrosian, Chief Medical Officer, Amylyx Pharmaceuticals

×

Camille Bedrosian / Patient- Focused Drug Development Across the Pipeline

Camille Bedrosian, Chief Medical Officer,
Amylyx Pharmaceuticals

Dr. Bedrosian joined Amylyx as Chief Medical Officer (CMO) in 2023, bringing to the company nearly 30 years of experience in building successful clinical development and translation research programs in the pharmaceutical industry. Dr. Bedrosian served most of her nearly six years at Ultragenyx as...

CloseMore

Kelly Esperias, Chief Strategy and Operating Officer, NORD

×

Kelly Esperias / Patient- Focused Drug Development Across the Pipeline

Kelly Esperias, Chief Strategy and Operating Officer,
NORD

Kelly Esperias, EdD, MBA, CFRE, serves as Chief Strategy & Operations Officer at the National Organization for Rare Disorders (NORD®), the leading and longest-standing patient advocacy group for the 30 million Americans living with a rare disease.  An accomplished and dynamic executive who has...

CloseMore

Robert Ryan, CEO, Paradigm Therapeutics

×

Robert Ryan / Patient- Focused Drug Development Across the Pipeline

Robert Ryan, CEO,
Paradigm Therapeutics

Dr. Robert Ryan is a visionary leader in the field of rare diseases and orphan drugs,  and currently serves as the CEO of Paradigm Therapeutics, where he spearheads the development of an innovative therapy for Epidermolysis Bullosa (EB).  With a career spanning over 35 years in the pharmaceutical...

CloseMore