Global Orphan Drugs Market, 2019-2020 & Forecast to 2025 - CDx enabled Personalized Medicine, Hybrid Clinical Trials,Drug Repurposing (DPRx), Rare Pediatric Disorders, Outsourcing Gene Therapy

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Feb 18, 2021, 08:00 ET

DUBLIN, Feb. 18, 2021 /PRNewswire/ -- The "New Drug Development for Rare Pediatric Disorders Sustaining High Growth in the Global Orphan Drugs Market, 2019-2025" report has been added to ResearchAndMarkets.com's offering.

This research service provides an overview of the orphan drugs market dynamics, estimating the market size broken down by different segments. It also dives deeper into the different technological, geographical, and therapeutic opportunities with an objective assessment of the disclosed pipelines. Notably, the study has an expanded coverage of not only rare diseases, but also hard-to-cure and "commercially non-viable" diseases.

Rare diseases have been an increasing area of focus as three waves have converged in recent years: genomic research and innovations becoming mainstream; the forthcoming regulatory policies and financial incentives put in place by the federal agencies; and the increasingly patient-centric and coordinated efforts of patient advocacy groups, caregivers, and centers of excellence (COEs).

The orphan drugs market has been characterized by severe unmet needs with therapies available for less than 8% of the identified diseases, creating an abundant opportunity for established and new companies alike to not only enter the market but also establish absolute dominance. However, this will require companies to navigate through R&D, clinical trial, small-volume manufacturing, and reimbursement/pricing challenges as there is a measurable departure from traditional pharma rules.

This study captures the competitive landscape and the different strategies employed by companies to stay ahead of the curve. It also identifies game-changing companies leading innovation from the front. Acknowledging that the market continues to be dominated by larger pharma companies, the study identifies opportunities for mid and small segment companies to tap this market. The study thus elaborates on opportunities in drug repurposing, personalized medicine, hybrid clinical trials, and untapped therapeutic areas.

Furthermore, this research service lays down strategic imperatives for prioritizing markets for new product launch, working with the right communities which act as conduits to the right patient populations, and the right set of technologies to be leveraged to accelerate innovation. In conjunction with the core manufacturers, the research service also provides in-depth analysis of other ecosystem stakeholders such as logistic service providers, contract service providers, diagnostic companies, investors, and advocacy groups.

Information is also given on some of the leading M&A activities impacting the market as well as unconventional collaboration agreements laying the foundation for propelling innovations toward licensure and delivery. Furthermore, present and future market trends like increasing price transparency and drug affordability, which would shape the market, are discussed.

Key Issues Addressed

What is the total market size and what is the projected growth for the global orphan drugs market? Which are the largest growth segments to be pursued?
How are the various regions around the globe positioned to monetize the market opportunities? What are the key regional barriers to entry?
What are the new risk mitigation strategies and alternative big bets to circumvent R&D challenges? Which are the lucrative partnership models for the long term?
What are the market-defining macro trends mandating the need to deploy new digital solutions suite and which cross sections of the value chain will benefit the most?
How are product offerings in this market likely to emerge? What is the rate of innovation adoption and how is it solving the unmet needs of customers?
Which are the key game-changing companies in the market and how are they riding the growth curve?

Key Topics Covered:

1. Executive Dashboard

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

2. 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

3. 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
Percent Revenue Breakdown by Oncology
Percent Revenue Breakdown for Other Segments
Total Opportunity with Orphan Drugs

4. Competitive Landscape

Market Share by Companies
Competitive Landscape Discussion
Competitive Environment

5. Key Collaboration Agreements

M&A Assessment
Collaboration Assessment
Key Companies to Watch

6. Pipeline Analysis

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

7. Visioning Scenario

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

8. Vision and Strategy - Growth Pipeline

Levers for Growth

9. 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

10. 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

11. Appendix

Companies Mentioned

Amicus Therapeutics
Bayer
Takeda
Glybera
Strimvelis

For more information about this report visit https://www.researchandmarkets.com/r/qul21z

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