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

RELEASE DATE
01-Mar-2024
REGION
North America
Deliverable Type
Market Research
Research Code: PFA6-01-00-00-00
SKU: HC_2024_609
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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

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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

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).--BEGIN PROMO--

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

More Information
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