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

AI-powered Early Disease Diagnosis and Drug Repurposing will Create New Growth Opportunities

RELEASE DATE
12-Aug-2020
REGION
North America
Research Code: MF5C-01-00-00-00
SKU: HC03311-GL-MT_24530
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Description

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.

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?

Author: Khushbu Jain

RESEARCH: INFOGRAPHIC

This infographic presents a brief overview of the research, and highlights the key topics discussed in it.
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Table of Contents

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

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

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

Market Share by Companies

Competitive Landscape Discussion

Competitive Environment

M&A Assessment

M&A Assessment (continued)

Collaboration Assessment

Key Companies to Watch

Key Companies to Watch (continued)

Pipeline Analysis

Pipeline Summary by Therapy Area

Pipeline Summary by Technology Type

High-potential Pipeline Products

Macro to Micro Visioning

Reimbursement—Challenges and Opportunity

Competitive Strategies

Competitive Strategies—First-to-market vs Late Entrant

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

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

Abbreviations

List of Exhibits

List of Exhibits (continued)

List of Exhibits (continued)

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.--BEGIN PROMO--

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?

Author: Khushbu Jain

More Information
No Index No
Podcast No
Author Khushbu Jain
Industries Healthcare
WIP Number MF5C-01-00-00-00
Is Prebook No
GPS Codes 9600-B1,9571-B1,9568-B1,9611-B1