Global Orphan Drugs Market Trends and Drivers, Restraints, and Opportunities 2017-2023
  • ID: 1169
  • September 6, 2017
  • 125 pages
  • Infoholic Research

Orphan Drugs Market – Global Drivers, Restraints, Opportunities, Trends, and Forecasts: 2017–2023
Overview: Orphan diseases or rare diseases rarely occur among the people, which is nearly 7 out of 10,000 individuals. Around 5,000 to 8,000 rare diseases have been recognized and the origin of approximately 80% of these diseases is from the genes. Regardless of the alleged huge number of individuals being affected by rare life-threatening conditions, the disease burden estimates of the public health are unreliable. About 50% of the orphan genetic ailments are in kids’ with 30% of them cannot live for more than 5 years.
New-born screening, legislation, and construction of national strategies are the major activities being carried out in the orphan drugs market. Legislation has helped in driving the development of more orphan drugs. For instance, the US congress passed Orphan Drug Act in 1983, and it was mainly intended to encourage development of new drugs for rare diseases. Currently, numerous legislation and strategies are developed to facilitate research of orphan drugs globally in more than 35 countries.
Cancer, metabolic diseases, blood diseases, immunologic disease, and neurologic diseases are the types of diseases that are addressed with orphan drugs. Cystic fibrosis, glioma, pancreatic cancer, acute myeloid leukemia, multiple myeloma, renal cell carcinoma, ovarian cancer and Duchenne muscular dystrophy are some of the common indications among the various disease types.
Market Analysis: The “Global Orphan Drugs Market” is estimated to witness a CAGR of 11.9% during the forecast period 2017–2023. The market is analyzed based on three segments, namely types, applications, and regions.
Regional Analysis: The regions covered in the report are North America, Europe, Asia Pacific, and Rest of the World (RoW). North America is the leading region for the orphan drugs market growth followed by Europe. Asia Pacific and RoW are set to be the emerging regions. India, China, and Japan are set to be the most attractive destinations due to the large untapped market.
Types Analysis: The global orphan drugs market by products is segmented into biologics and non-biologics. Biologics orphan drugs is the largest segment in the market with a market share of more than 60% in 2016. It is expected to grow at a double-digit CAGR in the market. However, non-biologic orphan drugs are still expected to hold more than quarter of the market share by 2023. The market is also witnessing various mergers, acquisitions, and collaborations among the top players, which is defining the future of the global orphan drugs market.
Key Players: Novartis AG, F. Hoffmann-La Roche Ltd, Celgene, Bristol-Myers Squibb Company, Shire pharmaceuticals, Pfizer, Sonafi, and Bayer Healthcare are the key players in the market. These top ten players occupy nearly 50% of the orphan drugs market.
Competitive Analysis: Currently, large pharmaceutical companies are extremely active in the orphan drugs market. Merger and acquisition is the major trend in the market. For instance, around 40% of the biotechnology companies are acquired between the year 2008-2012, and they have an orphan drug in development. Novartis, GSK, Roche, and Pfizer are the largest orphan drug companies. Pfizer, Gilead, Roche, Shire, BMY, and Celgene are leading orphan drug acquirers. Around 50% of the top 20 orphan drugs were either acquired or in-licensed by large pharmaceutical companies.
Benefits: The report provides complete details about the usage and adoption rate of orphan drugs during the forecast period and among the regions. With that, key stakeholders can know about the major trends, drivers, investments, vertical player’s initiatives, government initiatives toward the orphan drugs adoption in the upcoming years along with the details of commercial drugs available in the market. Moreover, the report provides details about the major challenges that are going to impact the market growth. Additionally, the report gives complete details about the key business opportunities to key stakeholders to expand their business and capture the revenue in the specific verticals to analyze before investing or expanding the business in this market.
Key Stakeholders:

Table of Contents
1 Industry Outlook 10
1.1 Industry Overview 10
1.2 Industry Trends 10
2 Report Outline 12
2.1 Report Scope 12
2.2 Report Summary 12
2.3 Research Methodology 13
2.4 Report Assumptions 13
3 Market Snapshot 15
3.1 Market Definition – Infoholic Research 15
3.2 Segmented Addressable Market (SAM) 15
3.2.1 Advantages of Development of Orphan Drug 16
3.2.2 Trends of orphan drug market 16
3.3 Related Addressable Markets (RAM) 17
3.3.1 Oncology (Cancer) Drugs 17
3.3.2 Active pharmaceutical ingredients (APIs) 18
3.3.3 Over the counter drugs (OTC) 18
4 Market Outlook 20
4.1 Overview 20
4.2 Regulatory Framework for orphan drugs 20
4.3 Funding Scenario for orphan drugs 22
4.4 Market Segmentation 23
4.5 Porter 5(Five) Forces 24
4.6 PEST Analysis 25
5 Market Characteristics 26
5.1 Market Dynamics 26
5.1.1 Drivers 26
5.1.1.1 Conducive government legislation 26
5.1.1.2 Marketing exclusivity for orphan drugs 26
5.1.1.3 Growing unmet need for treatment of orphan diseases 27
5.1.2 Opportunities 27
5.1.2.1 Growing Novel indications for known orphan drugs 27
5.1.2.2 Untapped emerging market 27
5.1.3 Restraints 27
5.1.3.1 High cost of treatment 27
5.1.3.2 Lack of Adequate patients for clinical trials 28
5.1.4 DRO – Impact Analysis 28
5.1.5 Key Stakeholders 29
6 Drug Details: Market Size and Analysis 30
6.1 Overview 30
7 Types: Market Size and Analysis 46
7.1 Overview 46
7.1.1 Biologics 46
7.1.2 Non-Biologics 47
8 Application: Market Size and Analysis 50
8.1 Overview 50
8.1.1 Oncology 50
8.1.2 Metabolic Diseases 51
8.1.3 Hematology 52
8.1.4 Infectious Diseases 53
8.1.5 Neurology 53
8.1.6 Others 54
9 Pipeline Molecules: Market Size and Analysis 55
9.1 Overview 55
10 Regions: Market Size and Analysis 71
10.1 Overview 71
10.2 North America 72
10.2.1 Overview 72
10.2.2 US 72
10.2.3 Canada 73
10.3 Europe 74
10.3.1 Overview 74
10.3.1.1 UK 75
10.3.1.2 Germany 75
10.3.1.3 France 75
10.4 APAC 75
10.4.1 Overview 75
10.4.1.1 India 77
10.4.1.2 Japan 78
10.4.1.3 China 78
10.5 Rest of the World 79
10.5.1 Overview 79
10.5.1.1 Brazil 80
10.5.1.2 Mexico 80
10.5.1.3 Africa 80
10.5.1.4 Middle East 80
11 Competitive Landscape 81
12 Vendor Profiles 83
12.1 Novartis AG 83
12.1.1 Overview 83
12.1.2 Business Unit 86
12.1.3 Geographic Presence 87
12.1.4 Business Focus 88
12.1.5 SWOT Analysis 88
12.1.6 Business Strategy 89
12.2 F.Hoffmann-La Roche Ltd 90
12.2.1 Overview 90
12.2.2 Business Unit 94
12.2.3 Geographic Presence 95
12.2.4 Business Focus 95
12.2.5 SWOT Analysis 96
12.2.6 Business Strategy 96
12.3 Celgene Corporation 97
12.3.1 Overview 97
12.3.2 Geographic Presence 99
12.3.3 Business Focus 99
12.3.4 SWOT Analysis 99
12.3.5 Business Strategy 100
12.4 Bristol-Myers Squibb 101
12.4.1 Overview 101
12.4.2 Geographic Presence 105
12.4.3 Business Focus 105
12.4.4 SWOT Analysis 105
12.4.5 Business Strategy 106
12.5 Shire PLC 107
12.5.1 Overview 107
12.5.2 Business Unit 110
12.5.3 Geographic Presence 112
12.5.4 Business Focus 112
12.5.5 SWOT Analysis 113
12.5.6 Business Strategy 113
13 Companies to Watch For 114
13.1 Pfizer Inc., 114
13.1.1 Overview 114
13.1.2 Highlights 115
13.2 Sanofi 116
13.2.1 Overview 116
13.3 Bayer AG 117
13.3.1 Overview 117
13.4 Alexion Pharmaceutical Inc. 118
13.4.1 Overview 118
13.5 Biogen Pharmaceuticals. 119
13.5.1 Overview 119
13.6 Eli Lilly and Company. 121
13.6.1 Overview 121
13.7 Amgen Inc. 122
13.7.1 Overview 122
Annexure 125
Abbreviations 125

Tables
TABLE 1 REGULATORY FRAMEWORK FOR ORPHAN DRUGS 18
TABLE 2 FUNDED PROJECTS OF 2017 20
TABLE 3 LIST OF ORPHAN DRUGS APPROVED IN THE MARKET 26
TABLE 4 MARKET REVENUE OF TOP 15 DRUGS, 2016 AND 2023 ($BILLION) 44
TABLE 5 MOLECULES IN DEVELOPMENT (2015) 46
TABLE 6 AUTOIMMUNE DISEASES MOLECULES 50
TABLE 7 BLOOD DISORDER MOLECULES 52
TABLE 8 RARE CANCER MOLECULES 52
TABLE 9 BLOOD CANCER MOLECULES 55
TABLE 10 CARDIOVASCULAR DISORDERS MOLECULES 56
TABLE 11 DIGESTIVE DISORDERS MOLECULES 57
TABLE 12 EYE DISORDERS MOLECULES 57
TABLE 13 GENETIC DISORDERS MOLECULES 57
TABLE 14 GROWTH DISORDERS MOLECULES 60
TABLE 15 INFECTIOUS DISORDERS MOLECULES 60
TABLE 16 NEUROLOGIC DISORDERS MOLECULES 61
TABLE 17 RESPIRATORY DISORDERS MOLECULES 62
TABLE 18 TRANSPLANTATION MOLECULES 62
TABLE 19 OTHER DISORDERS MOLECULES IN THE PIPELINE 63
TABLE 20 ORPHAN DRUGS MARKET REVENUE BY REGIONS, 2016–2023 ($BILLION) 65
TABLE 21 TOP 5 ORPHAN DRUGS IN THE US MARKET 66
TABLE 22 REGULATORY AUTHORITIES IN ASIA PACIFIC 69
TABLE 23 NOVARTIS AG: OFFERINGS 76
TABLE 24 NOVARTIS AG: RECENT DEVELOPMENTS 76
TABLE 25 F. HOFFMANN-LA ROCHE LTD.: OFFERINGS 82
TABLE 26 F. HOFFMANN-LA ROCHE LTD.: RECENT DEVELOPMENTS 82
TABLE 27 CELGENE CORPORATION: OFFERINGS 88
TABLE 28 CELGENE CORPORATION: RECENT DEVELOPMENTS 88
TABLE 29 BRISTOL–MYERS SQUIBB: OFFERINGS 92
TABLE 30 BRISTOL–MYERS SQUIBB: RECENT DEVELOPMENTS 92
TABLE 31 SHIRE PLC: OFFERINGS 98
TABLE 32 SHIRE PLC: RECENT DEVELOPMENTS 98
TABLE 33 PFIZER: RECENT DEVELOPMENTS 104
TABLE 34 SANOFI: RECENT DEVELOPMENTS 106
TABLE 35 BAYER AG: RECENT DEVELOPMENTS 107
TABLE 36 ALEXION PHARMACEUTICAL, INC.: RECENT DEVELOPMENTS 108
TABLE 37 BIOGEN PHARMACEUTICALS: RECENT DEVELOPMENTS 109
TABLE 38 ELI LILLY AND COMPANY: RECENT DEVELOPMENTS 110
TABLE 39 AMGEN INC.: RECENT DEVELOPMENTS 111

Charts
CHART 1 PEST ANALYSIS OF ORPHAN DRUGS MARKET 11
CHART 2 RESEARCH METHODOLOGY OF GLOBAL ORPHAN DRUGS MARKET 13
CHART 3 GLOBAL ORPHAN DRUGS MARKET REVENUE, 2016–2023 ($BILLION) 16
CHART 4 SEGMENTATION OF GLOBAL ORPHAN DRUGS MARKET 21
CHART 5 PORTER 5 FORCES OF ORPHAN DRUGS MARKET 21
CHART 6 MARKET DYNAMICS – DRO ANALYSIS 22
CHART 7 DRO – IMPACT ANALYSIS OF GLOBAL ORPHAN DRUGS MARKET 25
CHART 8 KEY STAKEHOLDERS OF GLOBAL ORPHAN DRUGS MARKET 25
CHART 9 ORPHAN DRUGS MARKET REVENUE BY TYPES SEGMENTATION, 2016 VS. 2023 ($BILLION) 42
CHART 10 BIOLOGICS ORPHAN DRUGS MARKET REVENUE BY TYPES SEGMENTATION, 2016–2023 ($BILLION) 43
CHART 11 NON-BIOLOGICS ORPHAN DRUGS MARKET REVENUE BY TYPES SEGMENTATION, 2016–2023 ($BILLION) 44
CHART 12 ORPHAN DRUGS MARKET SHARE BY APPLICATION SEGMENTATION, 2016 (%) 45
CHART 13 HEMATOLOGY MARKET SHARE, 2016 47
CHART 14 ORPHAN DRUG THERAPEUTIC AREA IN PIPELINE, 2016 49
CHART 15 ORPHAN DRUGS IN PIPELINE, 2016 50
CHART 16 ORPHAN DRUGS MARKET SHARE BY REGIONAL SEGMENTATION, 2016 VS. 2023 (%) 64
CHART 17 ORPHAN DRUGS MARKET REVENUE IN NORTH AMERICA, 2016–2023 ($BILLION) 65
CHART 18 ORPHAN DRUGS MARKET REVENUE IN EUROPE, 2016–2023 ($BILLION) 68
CHART 19 ORPHAN DRUGS MARKET REVENUE IN APAC, 2016–2023 ($BILLION) 70
CHART 20 ORPHAN DRUGS MARKET REVENUE IN REST OF THE WORLD, 2016–2023 ($BILLION) 72
CHART 21 ORPHAN DRUGS MARKET SHARE, 2016 75
CHART 22 NOVARTIS AG: OVERVIEW SNAPSHOT 77
CHART 23 NOVARTIS AG: BUSINESS UNITS 78
CHART 24 NOVARTIS AG: GEOGRAPHIC REVENUE 79
CHART 25 NOVARTIS AG: SWOT ANALYSIS 80
CHART 26 F. HOFFMANN-LA ROCHE LTD.: OVERVIEW SNAPSHOT 84
CHART 27 F. HOFFMANN-LA ROCHE LTD.: BUSINESS UNITS 84
CHART 28 F. HOFFMANN-LA ROCHE LTD.: GEOGRAPHIC REVENUE 85
CHART 29 F. HOFFMANN-LA ROCHE LTD.: SWOT ANALYSIS 86
CHART 30 CELGENE CORPORATION: OVERVIEW SNAPSHOT 88
CHART 31 CELGENE CORPORATION: GEOGRAPHIC REVENUE 89
CHART 32 CELGENE CORPORATION: SWOT ANALYSIS 90
CHART 33 BRISTOL–MYERS SQUIBB: OVERVIEW SNAPSHOT 93
CHART 34 BRISTOL–MYERS SQUIBB: BUSINESS SEGMENTS 94
CHART 35 BRISTOL–MYERS SQUIBB: GEOGRAPHIC REVENUE 96
CHART 36 BRISTOL–MYERS SQUIBB: SWOT ANALYSIS 96
CHART 37 SHIRE PLC: OVERVIEW SNAPSHOT 100
CHART 38 SHIRE PLC: BUSINESS UNITS 101
CHART 39 SHIRE PLC: GEOGRAPHIC REVENUE 102
CHART 40 SHIRE PLC: SWOT ANALYSIS 102

None

Novartis AG, F. Hoffmann-La Roche Ltd, Celgene, Bristol-Myers Squibb Company, Shire pharmaceuticals, Pfizer, Sonafi, and Bayer Healthcare

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