World Orphan Drug Congress USA 2021

World Orphan Drug Congress USA 2021 is organized by Terrapinn Pte Ltd and will be held from Aug 25 - 27, 2021 at Gaylord National Resort & Convention Center, National Harbor, Maryland, United States of America.

Who Attends:
• Pharma, biotechs
? VP Clinical Development
? Chief Medical Officer
? Chief Scientific Officer
? Regulatory Affairs
? President, Head of Rare Diseases

• Pharma, biotechs
? Chief Patient Officer
? VP Patient Advocacy
? Medical Affairs
? Patient Engagement

• Patient Advocacy Groups

• Pharma, biotechs
? VP Gene Therapy
? Chief Medical Officer
? Chief Manufacturing Officer
? Chief Scientific Officer
? VP Manufacturing &

• Technical Operations

• Pharma, biotechs
? Chief Executive Officer
? VP, Head of Market Access
? VP, Head of Pricing and Reimbursement
? VP of Health Economics/HEOR
? VP of HTA
? Head of Value Demonstration
? VP, Evidence and Data
? Payers (public and private)
? Government & Regulatory agencies

• Pharma, biotechs
? Chief Data Officer
? Chief Technology Officer
? Chief Operating Officer
? VP, Analytics
? Head of Innovation
? Head of Digital

• Hospitals and healthcare centers
• Payers

• Pharma, biotechs
? CEO
? Presidents
? Chief Business Officer
? VP Business Development
? VP External Innovation
? VP R&D
• Venture Capital Firms
• Grant Agencies (Public and Private)
• Licensing pharma

Description:
The 11th annual World Orphan Drug Congress USA is back — and in-person — with our most comprehensive program and inclusive gathering of
rare disease stakeholders yet! 

Becoming the largest rare disease and orphan drug conference globally has allowed us to bring together leading industry, government and regulatory authorities, patient advocacy groups, payers, investors and solution providers.

The World Orphan Drug Congress USA allows attendees to meet, brainstorm and dialogue on ways to advance orphan drug development and improve access to life-saving therapies.

Our conference has become the industry defining meeting on Advanced Therapies for rare diseases, featuring discussions on clinical development,
manufacturing and commercialization of cell and gene therapies. It also is the annual gathering for artificial intelligence, digital health solutions, and drug development platforms transforming the rare disease industry

Topic Areas:
• Patient centric clinical development
• Clinical trial design
• Real World Evidence in clinical development
• Expanded Access Programs / Managed Access Programs
• Patient Data
• Patient Registries & Patient Reported Outcomes (PROs)
• Evolving role of patient groups in R&D
• Genetic testing & diagnosis
• Advocacy & Policy
• Setting up patient groups
• Industry collaboration
• Gene therapy development
• Cell therapy development
• Translational science and path to clinical trials
• Regulation
• Advanced therapies pricing & reimbursement
• Real World Evidence
• Health Technology Assessment
• Value Based Assessment
• Next generation pricing and innovative pricing models
• AI in drug discovery
• Patient identification
• Data analytics
• Data collection through wearables and applications
• Advanced therapies investment
• Risk assessment
• Partnering and co-development opportunities
• Funding
• Accelerating orphan drug development
• Successful case studies