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Global Orphan Drug Growth Opportunities
Global Orphan Drug Growth Opportunities
Reduced Exclusivity and Increased Competition will Drive Demand for More Efficient R&D and Digital Solutions
01-Mar-2024
North America
Market Research
Description
In this study, Frost & Sullivan’s Transformational Health team provides critical insights into the global orphan drug (OD) industry and highlights growth opportunities, revenue, regulatory changes, and technology trends influencing growth. Approximately 5% of rare diseases (RDs) have received US FDA approval for a drug, while up to 15% of RDs have at least 1 drug that exhibits potential in terms of disease treatment or prevention. The growing number of unaddressed RD needs is a major catalyst for R&D. There is a need for novel medicine to treat RDs that currently have limited therapeutic choices. Recent advancements in precision medicine and informatics, such as big data analytics, multi-omics, nanomedicine, gene-editing techniques, and next-generation diagnostics, have created opportunities to develop specific and individualized therapies for RDs. The convergence of cancer and RDs is becoming evident. Precision oncology and tailored medicine for rare tumors are emerging as prominent themes in the discipline, facilitating the OD industry’s expansion.
Various techniques are used to develop ODs, mainly due to the rise of R&D in personalized medicine, including nucleic acid drugs, gene therapies, cell therapies, and engineered proteins. Small and medium-sized enterprises (SMEs) develop more than half of ODD medication because they benefit from lower prices for scientific assistance, pre- and post-authorization processes, and marketing authorization applications. The early investments obtained from venture capitalists buoy the momentum.
RD treatment access has improved with OD policies, benefiting local and global populations. These actions showcase countries' commitment to public health and research, boosting probable partnerships and investments. Healthcare systems face increasing requirements to contain present and future healthcare spending. Payers are diligently scrutinizing medicine costs and patient access levels to guarantee optimal equilibrium. Healthcare plan sponsors are considering their next move as governments contemplate Orphan Drug Act changes and the impact of the Inflation Reduction Act (IRA).
Key Issues Addressed:
How much revenue will the global OD industry generate in 2028?
Which segments will influence revenue growth rate?
What challenges do patients face in their RD journeys?
Which business models, technologies, and trends must stakeholders and participants watch during the forecast period?
What are the major OD growth opportunities?
Author: Surbhi Gupta
RESEARCH: INFOGRAPHIC
This infographic presents a brief overview of the research, and highlights the key topics discussed in it.Click image to view it in full size
Table of Contents
Why is it Increasingly Difficult to Grow?
The Strategic Imperative 8™
The Impact of the Top 3 Strategic Imperatives on the Orphan Drug Industry
Growth Opportunities Fuel the Growth Pipeline Engine™
Scope of Analysis
Segmentation
Rare Disease Patient Journey Challenges
Critical Future Steps in the Development of Novel Drugs for Rare Diseases
Growth Opportunities
Vendor Landscape
Growth Drivers
Growth Restraints
Pipeline Analysis and Summary by Therapeutic Segment
Pipeline Summary by Technology Type
Incentives to Develop Treatments for Rare Diseases—A Regional Overview
Regional Synopsis
Regional Synopsis (continued)
Regional Attractiveness
Recent Regulatory Policies and Initiatives by Region
Impact of the IRA on the US Orphan Drugs Industry
Special US FDA Designations for Drug Development—Orphan, Fast Track, Accelerated Approval, Priority Review, and Breakthrough Therapy
OD Business Models
OD Business Models (continued)
M&A Assessment
M&A Assessment (continued)
Venture Financing Assessment
Market Trends Advancing Sustainability in the Pharma Value Chain
Digital Novel Solution Applications
Industry Use Case—UCB’s Drug for Myasthenia Gravis
Research Process and Methodology
Forecast Considerations
Forecast Considerations (continued)
Growth Metrics
Revenue Forecast
Revenue Forecast Analysis
Revenue Forecast by Product Type
Forecast Analysis—Small Molecules
Forecast Analysis—Biologics
Key Assets to Watch During the Forecast Period
Percent Revenue Breakdown by Technology Type
Percent Revenue Forecast by Therapy Type
Key Oncology Rare Indications
Approved Oncology Drugs’ Characteristics—Orphans versus Non-orphans
Approved Oncology Drugs’ Characteristics—Orphans versus Non-orphans (continued)
Key Non-oncology Indications—Immunology/Musculoskeletal System
Key Non-oncology Indications—CNS
Key Non-oncology Indications—Blood Disorders and Cardio-metabolic Diseases
Pricing Trends
Competitive Environment
Revenue Share
Revenue Share Analysis
Growth Opportunity 1: Nucleic Acid-based Therapeutics
Growth Opportunity 1: Nucleic Acid-based Therapeutics (continued)
Growth Opportunity 2: Leveraging Bioinformatics for Drug Repurposing
Growth Opportunity 2: Leveraging Bioinformatics for Drug Repurposing (continued)
Growth Opportunity 3: Strategic Partnerships in the Middle East to Improve Diagnosis and Treatment
Growth Opportunity 3: Strategic Partnerships in the Middle East to Improve Diagnosis and Treatment (continued)
Growth Opportunity 4: Utilizing AI to Improve Diagnosis and Treatment
Growth Opportunity 4: Utilizing AI to Improve Diagnosis and Treatment (continued)
Your Next Steps
Why Frost, Why Now?
List of Exhibits
Legal Disclaimer
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Key Issues Addressed:
How much revenue will the global OD industry generate in 2028?
Which segments will influence revenue growth rate?
What challenges do patients face in their RD journeys?
Which business models, technologies, and trends must stakeholders and participants watch during the forecast period?
What are the major OD growth opportunities?
Author: Surbhi Gupta
| Deliverable Type | Market Research |
|---|---|
| Author | Surbhi Gupta |
| Industries | Healthcare |
| No Index | No |
| Is Prebook | No |
| Keyword 1 | Rare Disease Market |
| Keyword 2 | Precision Medicine Market Growth |
| Keyword 3 | Orphan Drugs Market |
| Podcast | No |
| Predecessor | MF5C-01-00-00-00 |
| WIP Number | PFA6-01-00-00-00 |
