New Drug Development for Rare Pediatric Disorders Sustaining High Growth in the Global Orphan Drugs Market, 2019–2025

New Drug Development for Rare Pediatric Disorders Sustaining High Growth in the Global Orphan Drugs Market, 2019–2025

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).

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.
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.


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?

  • 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
  • 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
      • Table Total Orphan Drugs Market: Market Engineering Measurements, Global, 2019
    • Research Process and Methodology
    • Forecast Assumptions
    • Total Revenue Forecast
      • Table Total Orphan Drugs Market: Revenue Forecast, Global, 2017–2025
    • Total Revenue Forecast by Product Group
      • Table Total Orphan Drugs Market: Revenue Forecast by Product Group, Global, 2017–2025
    • Percent Revenue Breakdown by Product Group
    • Total Revenue Forecast by Region
      • Table Total Orphan Drugs Market: Revenue Forecast by Region, Global, 2017–2025
    • 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
  • Competitive Landscape
    • Market Share by Companies
    • Competitive Landscape Discussion
    • Competitive Environment
  • Key Collaboration Agreements
    • M&A Assessment
    • Collaboration Assessment
    • Key Companies to Watch
  • 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
    • 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
      • Table Total Orphan Drugs Market: Drug Patent Expiration Landscape, Global, 2019–2026
    • 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
  • Appendix
    • Abbreviations

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