Table of Content

Table of Contents

Executive Dashboard
Purpose of this Experiential Study
5-step Process to Transformational Growth
Key Questions this Study will Answer
Key Findings
Key Findings (continued)
Market Engineering Measurements
Scope and Segmentation
Big Market Themes
Strategic Imperatives for Growth

Market Overview and Dynamics
Definitions
Market Background
Market Drivers
Market Restraints
Orphan Drug Criteria
Designated and Approved Orphan Drugs
Approved Orphan New Molecular Entities (NMEs)
Legislative and Regulatory Policies
Financial and Non-Financial Incentives
Global Market Attractiveness Outlook
Resources to Leverage
Vendor Landscape

Forecast and Trends—Total Orphan Drugs Market
Market Engineering Measurements
Research Process and Methodology
Forecast Assumptions
Total Revenue Forecast
Total Revenue Forecast by Product Group
Percent Revenue Breakdown by Product Group
Total Revenue Forecast by Region
Percent Revenue Breakdown by Geography
Percent Revenue Forecast by Therapy Type
Magnified View of the Top 4 Non-Oncology Disease Areas
Magnified View of the Top 4 Non-Oncology Disease Areas (continued)
Percent Revenue Breakdown by Oncology
Percent Revenue Breakdown for Other Segments
Total Opportunity with Orphan Drugs

Competitive Landscape
Market Share by Companies
Competitive Landscape Discussion
Competitive Environment

Key Collaboration Agreements
M&A Assessment
M&A Assessment (continued)
Collaboration Assessment
Key Companies to Watch
Key Companies to Watch (continued)

Pipeline Analysis
Pipeline Analysis
Pipeline Summary by Therapy Area
Pipeline Summary by Technology Type
High-potential Pipeline Products

Visioning Scenario
Macro to Micro Visioning
Reimbursement—Challenges and Opportunity
Competitive Strategies
Competitive Strategies—First-to-market vs Late Entrant

Vision and Strategy—Growth Pipeline
Levers for Growth

Vision and Strategy—Growth Opportunities
Vision and Strategy—Growth Opportunities
Growth Opportunity 1—Outsourcing Gene Therapy Development and Manufacturing
Current Gene Therapy Landscape
Growth Opportunity 2—CDx enabled Personalized Medicine
Need for CDx
Current State of CDx in the Orphan Drugs Landscape
Growth Opportunity 3—Hybrid Clinical Trials
Definition and Applications of Hybrid Trials for Rare Diseases
Ecosystem Participants Facilitating Hybrid Clinical Trials for Rare Diseases
Growth Opportunity 4—Drug Repurposing (DPRx)
Current State of Repurposed Drugs in the Orphan Drugs Landscape
Drug Repurposing Strategies
Key Company to Watch—Healx
Growth Opportunity 5—Rare Pediatric Disorders
Business Opportunity by Therapy Areas
Key Assets at the Risk of Generic/Biosimilar Competition
Growth Opportunity Impact Analysis

The Last Word
Pitfalls to Avoid
Strategic Imperatives for Growth
Case Study—Takeda and Bayer
Case Study—Amicus Therapeutics
Case Study—Strimvelis
Case Study—Glybera
Top 3 Predictions for the Market
Legal Disclaimer

Appendix
Abbreviations
List of Exhibits
List of Exhibits (continued)
List of Exhibits (continued)