SPONSOR THE WORLD ORPHAN DRUG CONGRESS USA 2024

WHO ATTENDS

Pharma, biotechs

• VP Clinical Development
• Chief Medical Officer
• Chief Scientific Officer
• Regulatory Affairs
• President, Head of Rare Diseases

TOPIC AREAS

- Patient centric clinical development

- Clinical trial design

- Real World Evidence in clinical development

- Expanded Access Programs / Managed Access Programs

WHO SHOULD SPONSOR

- Contract Research Organizations (CROs)

- Contract Development and Manufacturing Organizations (CDMOs)

- Clinical Trials Services - Home Clinical Trials

- Patient Recruitment & Identification

- Regulatory Consulting

WHO ATTENDS

Pharma, biotechs

• Chief Patient Officer
• VP Patient Advocacy
• Medical Affairs
• Patient Engagement

Patient Advocacy Groups

TOPICS AREAS

- 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

WHO SHOULD SPONSOR

- Pharma & biotechs

- Research Institutions

- Patient Registries

- Patient Engagement Platforms

- Genetic Testing providers

- Genomic Sequencing Platforms

- Diagnostics

WHO ATTENDS

Pharma, biotechs

• VP Gene Therapy
• Chief Medical Officer
• Chief Manufacturing Officer
• Chief Scientific Officer
• VP Manufacturing

Technical Operations

TOPICS AREAS

- Gene therapy development

- Cell therapy development

- Translational science and path to clinical trials

- Regulation

WHO SHOULD SPONSOR

- Advanced therapies pharma & biotechs

- Gene Editing biotechs

- Contract Research Organizations (CROs)

- Clinical trial design

WHO ATTENDS

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

TOPICS AREAS

- Advanced therapies pricing & reimbursement

- Real World Evidence

- Health Technology Assessment

- Value Based Assessment

- Next generation pricing and innovative pricing models

WHO SHOULD SPONSOR

- PRO consultancies and PRO technology providers

- Value communication organizations

- Pricing & Reimbursement consulting firms

- HEOR service providers

- Patient registries and database providers

WHO ATTENDS

Pharma, biotechs

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

Hospitals and healthcare centers

Payers

TOPICS AREAS

- AI in drug discovery

- Patient identification

- Data analytics

- Data collection through wearables and applications

WHO SHOULD SPONSOR

- Artificial Intelligence platforms

- Patient identification solutions

- Machine learning

- Data analytics

- Wearable technology

- Digital Health Apps

- Patient registries

WHO ATTENDS

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

TOPICS AREAS

- Advanced therapies investment

- Risk assessment

- Partnering and co-development opportunities

- Funding

- Accelerating orphan drug development

- Successful case studies

WHO SHOULD SPONSOR

- Advanced therapies investment

- Risk assessment

- Partnering and co-development opportunities

- Funders

WHO ATTENDS

Pharma, biotechs

• Chief Commercial Officer
• VP Commercial Operations
• VP of Marketing
• VP Medical Affairs

TOPIC AREAS

- Business models

- Forecasting and Epidemiology

- Global expansion and internationalization

- Business development

WHO SHOULD SPONSOR

- Specialty Pharmacy

- Logistics, Supply Chain, 3PL

- Strategic Consulting

- Real World Evidence

- Forecasting and Epidemiology

- Marketing, Communications Agency

WHO ATTENDS

Pharma, biotechs

• Presidents & Heads of Patient Diagnosis Programs
• R&D Strategic Alliance

Rare Disease advocacy organizations

• Newborn Screening

TOPIC AREAS

- Genetic counselling

- Industry partnerships

- Genomics

- Newborn Screening

- Access & Reimbursement of testing

WHO SHOULD SPONSOR

- Genetic testing companies

- Genetic counselling consulting

- Genomics companies

- Newborn screening providers

WHO ATTENDS

Pharma, biotechs

• Chief Operating Officer
• Chief Manufacturing & Technology Officer
• VP Technical Operations
• VP of CMC
• VP Manufacturing
• VP of Process Development
• VP of Quality Control

TOPIC AREAS

- Process development

- Outsourcing

- Viral vector development

- Manufacturing efficiency and capacity

- Analytical development

- Quality Control

WHO SHOULD SPONSOR

- Process development

- Outsourcing

- Viral vector development

- Manufacturing efficiency and capacity

- Analytical development

WHO ATTENDS

Pharma, biotechs

• VP Market Access
• Pricing and Reimbursement
• Chief Commercial Officer
• International Business

Regulators

Government

• Ministry of Health
• Policy makers

TOPIC AREAS

- Market entry strategies

- Stakeholder collaboration for market access

- Real World Evidence

- Improving access

- International regulation

WHO SHOULD SPONSOR

- Market entry strategies

- Stakeholder collaboration for market access

- Real World Evidence

- Improving access

- International regulation

WHO ATTENDS

Pharma, biotechs

• Heads of Discovery
• VP Clinical Development
• Chief Medical Officer
• Chief Scientific Officer
• Regulatory Affairs
• President, Head of Rare Diseases

TOPIC AREAS

- Discovery platforms

- Artificial Intelligence & Machine Learning applications

- Small molecule development

- Scientific innovations

- Collaborations

WHO SHOULD SPONSOR

- Drug discovery companies

- Artificial Intelligence and Machine Learning applications

- Predictive analytics platforms

- Contract Research Organizations (CROs)

WHO ATTENDS

Pharma, biotechs

• VP Cell Therapy
• Head of Oncology
• Head of Immunotherapy 
• Chief Medical Officer

TOPIC AREAS

- Diagnosis

- Biomarker R&D

- CAR-T development

- Advocacy and patient voice

- Clinical development and commercialization

WHO SHOULD SPONSOR

- Immunoncology pharma and biotechs

- Contract Manufacturing Organizations (CMOs)

- Contract Research Organizations (CROs)

- Genomic/DNA sequencing