Global Orphan Drugs Market Report: 2016 Edition
A rare disease is the one that occurs uncommonly or rarely in the general population. However, there exists no clear definition for categorizing “rare diseases” and is usually defined on the basis of the prevalence, and some other factors including the severity of disease and availability of treatment options. Rare diseases are often serious, chronic and progressive. Orphan drugs are medicinal products intended for the diagnosis, prevention or treatment of rare diseases. These drugs are referred to be “orphan” as under normal conditions because these drugs are not cost effective to be developed by the pharmaceuticals industry, intended for a small number of patients suffering from rare conditions.
Increasing sales of prescription drugs, substantial benefits for new entrants in rare disease drug market, increased spending on medicines, rising healthcare expenditure and improving economic conditions of nations are some of the significant factors driving growth of the Orphan drugs market. However, the growth of the market is hindered by certain challenges including tougher regulatory approvals, limitation on charging higher prices and no approved drugs for several rare diseases including Polychethemia Vera (PV).
The global orphan drugs market is expected to see numerous developments including approval of several Ultra-Rare drugs, increasing scope of Gene therapy, development of drugs for rare blood disease, higher success prospects for Hematology compared to Solid Tumors, attractive pricing option for orphan drug competitors and striking opportunities in developing orphan drugs.
The report, “Global Orphan Drugs Market” analyzes the current prevailing condition of the market along with its future scope of development. The global market along with specific market of the U.S., Canada, Europe and Japan, is being discussed in the report. The major trends, growth drivers as well as issues being faced by the industry are being presented in this report. The major players in the industry are being profiled, along with their key financials and strategies for growth.
Increasing sales of prescription drugs, substantial benefits for new entrants in rare disease drug market, increased spending on medicines, rising healthcare expenditure and improving economic conditions of nations are some of the significant factors driving growth of the Orphan drugs market. However, the growth of the market is hindered by certain challenges including tougher regulatory approvals, limitation on charging higher prices and no approved drugs for several rare diseases including Polychethemia Vera (PV).
The global orphan drugs market is expected to see numerous developments including approval of several Ultra-Rare drugs, increasing scope of Gene therapy, development of drugs for rare blood disease, higher success prospects for Hematology compared to Solid Tumors, attractive pricing option for orphan drug competitors and striking opportunities in developing orphan drugs.
The report, “Global Orphan Drugs Market” analyzes the current prevailing condition of the market along with its future scope of development. The global market along with specific market of the U.S., Canada, Europe and Japan, is being discussed in the report. The major trends, growth drivers as well as issues being faced by the industry are being presented in this report. The major players in the industry are being profiled, along with their key financials and strategies for growth.
1. ORPHAN DISEASES: INTRODUCTION
1.1 Causes
1.2 Prevention
1.3 Diagnosis
1.4 Treatment Options
1.5 Orphan Drugs
2. MARKET ANALYSIS
2.1 Global Orphan Drugs Market
Market Value
Growth Rate
Market Segmentation
2.1.1 Non-Hodgkin’s Lymphoma (NHL) Market
3. REGIONAL ANALYSIS
3.1 The U.S. Orphan Drugs Market
Applications & Designations
Spending Growth Forecast
3.1.1 The Ultra-Rare Drugs Market
3.2 Canada
3.3 Europe
3.4 Japan
4. MARKET DYNAMICS
4.1 Key Trends and Developments
4.1.1 Striking Opportunities in Developing Orphan Drugs
4.1.2 Attractive Pricing Option for Orphan Drug Companies
4.1.3 Higher Success Prospect for Hematology vs. Solid Tumors
4.1.4 Development of Drugs for Rare Blood Diseases
4.1.5 Increasing Scope of Gene Therapy
4.1.6 Approval of Ultra-Orphan Drugs
4.2 Growth Drivers
4.2.1 Increasing Sales of Prescription Drugs
4.2.2 Substantial Benefits for New Entrants in Rare Diseases Market
4.2.3 Increased Spending on Medicines
4.2.4 Rising Healthcare Expenditure
4.2.5 Rising Global GDP
4.3 Challenges
4.3.1 Limitation on Charging High Price for Orphan Drugs
4.3.2 No Approved Drug for Polycythemia Vera First-line Treatment
4.3.3 Tough Regulatory Approval for Orphan Drugs
5. COMPETITIVE LANDSCAPE
5.1 Global Market
Market Share
Product Comparison
5.2 The U.S. Market
Market Share
Product Comparison
6. COMPANY PROFILES
6.1 Roche Holdings
6.1.1 Business Overview
6.1.2 Financial Overview
6.1.3 Business Strategies
6.2 Novartis
6.2.1 Business Overview
6.2.2 Financial Overview
6.2.3 Business Strategies
6.3 Celgene Corporation
6.3.1 Business Overview
6.3.2 Financial Overview
6.3.3 Business Strategies
6.4 Novo Nordisk
6.4.1 Business Overview
6.4.2 Financial Overview
6.4.3 Business Strategies
6.5 Eli Lilly and Company
6.5.1 Business Overview
6.5.2 Financial Overview
6.5.3 Business Strategies
1.1 Causes
1.2 Prevention
1.3 Diagnosis
1.4 Treatment Options
1.5 Orphan Drugs
2. MARKET ANALYSIS
2.1 Global Orphan Drugs Market
Market Value
Growth Rate
Market Segmentation
2.1.1 Non-Hodgkin’s Lymphoma (NHL) Market
3. REGIONAL ANALYSIS
3.1 The U.S. Orphan Drugs Market
Applications & Designations
Spending Growth Forecast
3.1.1 The Ultra-Rare Drugs Market
3.2 Canada
3.3 Europe
3.4 Japan
4. MARKET DYNAMICS
4.1 Key Trends and Developments
4.1.1 Striking Opportunities in Developing Orphan Drugs
4.1.2 Attractive Pricing Option for Orphan Drug Companies
4.1.3 Higher Success Prospect for Hematology vs. Solid Tumors
4.1.4 Development of Drugs for Rare Blood Diseases
4.1.5 Increasing Scope of Gene Therapy
4.1.6 Approval of Ultra-Orphan Drugs
4.2 Growth Drivers
4.2.1 Increasing Sales of Prescription Drugs
4.2.2 Substantial Benefits for New Entrants in Rare Diseases Market
4.2.3 Increased Spending on Medicines
4.2.4 Rising Healthcare Expenditure
4.2.5 Rising Global GDP
4.3 Challenges
4.3.1 Limitation on Charging High Price for Orphan Drugs
4.3.2 No Approved Drug for Polycythemia Vera First-line Treatment
4.3.3 Tough Regulatory Approval for Orphan Drugs
5. COMPETITIVE LANDSCAPE
5.1 Global Market
Market Share
Product Comparison
5.2 The U.S. Market
Market Share
Product Comparison
6. COMPANY PROFILES
6.1 Roche Holdings
6.1.1 Business Overview
6.1.2 Financial Overview
6.1.3 Business Strategies
6.2 Novartis
6.2.1 Business Overview
6.2.2 Financial Overview
6.2.3 Business Strategies
6.3 Celgene Corporation
6.3.1 Business Overview
6.3.2 Financial Overview
6.3.3 Business Strategies
6.4 Novo Nordisk
6.4.1 Business Overview
6.4.2 Financial Overview
6.4.3 Business Strategies
6.5 Eli Lilly and Company
6.5.1 Business Overview
6.5.2 Financial Overview
6.5.3 Business Strategies
LIST OF CHARTS
Global Orphan Drugs Market (2010-2015E)
Orphan Drugs as Percent of Prescription Drugs (2010-2015E)
Global Orphan Drugs Market by Type (2014)
Average Cost per Patient for Orphan & Non-orphan Drugs (2010-2014)
Median Cost per Patient for Orphan & Non-orphan Drugs (2010-2014)
Non-Hodgkin’s Lymphoma Stage at Diagnosis (2014)
Non-Hodgkin’s Lymphoma (NHL) by Sub-Types (2014)
Incidence of Non-Hodgkin’s Lymphoma in Major Countries (2010-2014)
Orphan Drug (OD) Applications & Designations per Year (2009-2014)
The U.S. Orphan Drugs Spending (2009-2014E)
The U.S. Orphan Drugs Spending Forecast (2015-2018)
Prevalence of LEMS in the U.S. (2015-2020E)
Firdapse Drug’s Sales and Penetration Rate (2015-2020E)
The U.S. Epilepsy, Tourette and West Syndrome Prevalence (2015-2020E)
The U.S. CPP-115 Revenue (2018-2022)
Orphan Drugs Spending in Canada (2009-2014E)
Canada’s Orphan Drugs Spending Forecast (2015-2018)
Orphan Drug Designations per Year in EU (2009-2014)
Orphan Designations in EU by Indication (2014)
Orphan Disease Prevalence in EU by Indication (2014)
Orphan Designations per Year in Japan (2009-2014)
Median Patient Volume in Phase 3 Trial
Median Phase 3 Trial Costs
Success Probability for Hematology vs. Solid Tumors
Global Sales of Prescription Drugs (2010-2015E)
Annual Prices of Key Orphan Drugs in the U.S
Global Spending on Medicines (2010-2020E)
Global Healthcare Expenditure Per Capita (2009-2014E)
Global Gross Domestic Product Growth (2009-2014)
Ranking of Factors for Inclusion/Restriction in Plans by the U.S. Payers
Global Orphan Drugs Market by Company (2014)
Global Orphan Drugs Market by Company (2020E)
The U.S. Orphan Drugs Market by Company (2014)
Roche’s Revenue by Business Segments (2014)
Roche’s Revenue and Net Income (2010-2014)
Novartis Revenue by Business Segments (2014)
Novartis Revenues (2010-2014)
Celgene Corporation Revenue by Segments (2013/2014)
Celgene Corporation Revenue and Net Income (2010-2014)
Research and Development Expense by Category (2012-2014)
Novo Nordisk Revenue Share by Business Segments (2014)
Novo Nordisk’s Sales and Net Income (2010-2014)
Eli Lilly’s Revenue by Segments (2014)
Eli Lilly’s Sales and Net Income (2010-2014)
Global Orphan Drugs Market (2010-2015E)
Orphan Drugs as Percent of Prescription Drugs (2010-2015E)
Global Orphan Drugs Market by Type (2014)
Average Cost per Patient for Orphan & Non-orphan Drugs (2010-2014)
Median Cost per Patient for Orphan & Non-orphan Drugs (2010-2014)
Non-Hodgkin’s Lymphoma Stage at Diagnosis (2014)
Non-Hodgkin’s Lymphoma (NHL) by Sub-Types (2014)
Incidence of Non-Hodgkin’s Lymphoma in Major Countries (2010-2014)
Orphan Drug (OD) Applications & Designations per Year (2009-2014)
The U.S. Orphan Drugs Spending (2009-2014E)
The U.S. Orphan Drugs Spending Forecast (2015-2018)
Prevalence of LEMS in the U.S. (2015-2020E)
Firdapse Drug’s Sales and Penetration Rate (2015-2020E)
The U.S. Epilepsy, Tourette and West Syndrome Prevalence (2015-2020E)
The U.S. CPP-115 Revenue (2018-2022)
Orphan Drugs Spending in Canada (2009-2014E)
Canada’s Orphan Drugs Spending Forecast (2015-2018)
Orphan Drug Designations per Year in EU (2009-2014)
Orphan Designations in EU by Indication (2014)
Orphan Disease Prevalence in EU by Indication (2014)
Orphan Designations per Year in Japan (2009-2014)
Median Patient Volume in Phase 3 Trial
Median Phase 3 Trial Costs
Success Probability for Hematology vs. Solid Tumors
Global Sales of Prescription Drugs (2010-2015E)
Annual Prices of Key Orphan Drugs in the U.S
Global Spending on Medicines (2010-2020E)
Global Healthcare Expenditure Per Capita (2009-2014E)
Global Gross Domestic Product Growth (2009-2014)
Ranking of Factors for Inclusion/Restriction in Plans by the U.S. Payers
Global Orphan Drugs Market by Company (2014)
Global Orphan Drugs Market by Company (2020E)
The U.S. Orphan Drugs Market by Company (2014)
Roche’s Revenue by Business Segments (2014)
Roche’s Revenue and Net Income (2010-2014)
Novartis Revenue by Business Segments (2014)
Novartis Revenues (2010-2014)
Celgene Corporation Revenue by Segments (2013/2014)
Celgene Corporation Revenue and Net Income (2010-2014)
Research and Development Expense by Category (2012-2014)
Novo Nordisk Revenue Share by Business Segments (2014)
Novo Nordisk’s Sales and Net Income (2010-2014)
Eli Lilly’s Revenue by Segments (2014)
Eli Lilly’s Sales and Net Income (2010-2014)
LIST OF TABLES
Orphan Drugs as Proportion of New Molecular Entities Approved in the U.S. (2010-2014)
Orphan Drugs Approved in Japan (2014)
Prices and Prevalence of Orphan Drugs in the U.S
Current Ongoing Trials for Rare Blood Diseases
Recently Approved Ultra-Orphan Drugs
Globally Operating Top Orphan Drugs Competitor’s Overview
The U.S. Orphan Drugs Competitor’s Overview (2014)
Orphan Drugs as Proportion of New Molecular Entities Approved in the U.S. (2010-2014)
Orphan Drugs Approved in Japan (2014)
Prices and Prevalence of Orphan Drugs in the U.S
Current Ongoing Trials for Rare Blood Diseases
Recently Approved Ultra-Orphan Drugs
Globally Operating Top Orphan Drugs Competitor’s Overview
The U.S. Orphan Drugs Competitor’s Overview (2014)
