orphan drugs market

1

Orphan Drugs Market, by Type (Biological

Orphan Drugs, Non-Biological Orphan

Drugs), Therapeutic Area (Oncology,

Neurology Hematology, Infectious,

Disease, Metabolic Disease, and Others) -

Global Revenue, Trends, Growth, Share,

Size and Forecast to 2022

November 2016

2

About Scalar Market Research

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Table of Contents

1 Introduction .................................................................................................................................. 12

2 Market Overview .......................................................................................................................... 13

2.1 Definitions ............................................................................................................................. 13

2.2 Research Methodology ......................................................................................................... 14

2.2.1 Primary Data ................................................................................................................. 14

2.2.2 Secondary Data ............................................................................................................. 15

2.2.3 Machine Learning .......................................................................................................... 15

2.2.4 Research Scope ............................................................................................................. 16

2.2.5 Years Considered .......................................................................................................... 17

2.2.6 Currency & Pricing......................................................................................................... 17

2.3 Market Segmentation ........................................................................................................... 18

2.4 Key Objectives & Benefits of the Report............................................................................... 19

2.4.1 Objectives of the Report ............................................................................................... 19

2.4.2 Key Benefits of the Report ............................................................................................ 19

2.5 Assumptions .......................................................................................................................... 20

3 Executive Summary ....................................................................................................................... 21

4 Market Dynamics .......................................................................................................................... 23

4.1 Drivers ................................................................................................................................... 23

4.2 Restraints .............................................................................................................................. 27

4.3 Opportunities ........................................................................................................................ 28

4.4 Premium Industry Trends ..................................................................................................... 29

5 Industry Analysis ........................................................................................................................... 30

5.1 Industry Overview ................................................................................................................. 30

5.2 Regulatory Analysis ............................................................................................................... 31

5.2.1 European Union ............................................................................................................ 32

5.2.2 U.S. FDA ......................................................................................................................... 34

5.2.3 Other Countries ............................................................................................................. 35

5.3 Time to Entry Analysis ........................................................................................................... 36

5.4 Pipeline Analysis .................................................................................................................... 37

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5.5 Pricing Analysis ...................................................................................................................... 40

6 Orphan Drugs Market, By Type ..................................................................................................... 42

6.1 Share Segmentation .............................................................................................................. 44

6.2 Type ....................................................................................................................................... 46

6.2.1 Biological Orphan Drugs ................................................................................................ 46

6.2.2 Non-Biological Orphan Drugs ........................................................................................ 48

7 Orphan Drugs Market, By Therapeutic Area ................................................................................. 50

7.1 Share Segmentation .............................................................................................................. 51

7.2 Therapeutic Area ................................................................................................................... 53

7.2.1 Oncology ....................................................................................................................... 53

7.2.2 Neurology ...................................................................................................................... 54

7.2.3 Hematology ................................................................................................................... 55

7.2.4 Infectious Disease ......................................................................................................... 56

7.2.5 Metabolic Disease ......................................................................................................... 57

7.2.6 Other Diseases .............................................................................................................. 58

8 Orphan Drugs Market, By Geography ........................................................................................... 59

8.1 North America ....................................................................................................................... 63

8.1.1 By Regions ..................................................................................................................... 64

8.1.1.1 U.S. ............................................................................................................................ 64

8.1.1.2 Canada ...................................................................................................................... 65

8.1.2 By Type .......................................................................................................................... 65

8.1.3 By Therapeutic Area ...................................................................................................... 66

8.2 Europe ................................................................................................................................... 68

8.2.1 By Regions ..................................................................................................................... 69

8.2.1.1 Germany .................................................................................................................... 70

8.2.1.2 U.K ............................................................................................................................. 70

8.2.1.3 France ........................................................................................................................ 70

8.2.1.4 Italy ............................................................................................................................ 70

8.2.1.5 Rest of Europe ........................................................................................................... 71

8.2.2 By Type .......................................................................................................................... 71


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8.3 Asia-Pacific ............................................................................................................................ 74

8.3.1 By Regions ..................................................................................................................... 75

8.3.1.1 China ......................................................................................................................... 76

8.3.1.2 India........................................................................................................................... 76

8.3.1.3 Australia .................................................................................................................... 76

8.3.1.4 Japan ......................................................................................................................... 76

8.3.1.5 Singapore .................................................................................................................. 76

8.3.1.6 Rest of Asia-Pacific .................................................................................................... 77

8.3.2 By Type .......................................................................................................................... 77


8.4 Rest of the World (ROW) ...................................................................................................... 79

8.4.1 By Regions ..................................................................................................................... 80

8.4.1.1 Latin America ............................................................................................................ 81

8.4.1.2 Middle East & Africa.................................................................................................. 81

8.4.2 By Type .......................................................................................................................... 81


9 Competitive Landscape ................................................................................................................. 84

9.1 Market Share Analysis ........................................................................................................... 84

9.2 Market Positioning ................................................................................................................ 86

10 Key Market Players ....................................................................................................................... 87

10.1 Amgen Inc. ............................................................................................................................ 87

10.1.1 Overview ....................................................................................................................... 87

10.1.2 Key Strategies ................................................................................................................ 87

10.1.3 Recent Developments ................................................................................................... 87

10.2 Alexion Pharmaceuticals ....................................................................................................... 88

10.2.1 Overview ....................................................................................................................... 88

10.2.2 Key Strategies ................................................................................................................ 88


10.3 Pfizer Inc. ............................................................................................................................... 89

10.3.1 Overview ....................................................................................................................... 89

10.3.2 Key Strategies ................................................................................................................ 89

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10.4 Celgene Corporation ............................................................................................................. 90

10.4.1 Overview ....................................................................................................................... 90

10.4.2 Key Strategies ................................................................................................................ 90


10.5 Novartis International AG ..................................................................................................... 91

10.5.1 Overview ....................................................................................................................... 91

10.5.2 Key Strategies ................................................................................................................ 91


10.6 Shire Plc ................................................................................................................................. 92

10.6.1 Overview ....................................................................................................................... 92

10.6.2 Key Strategies ................................................................................................................ 92


10.7 Hoffman-La Roche (Roche) ................................................................................................... 93

10.7.1 Overview ....................................................................................................................... 93

10.7.2 Key Strategies ................................................................................................................ 93


10.8 BioMarin Pharmaceuticals Inc. ............................................................................................. 94

10.8.1 Overview ....................................................................................................................... 94

10.8.2 Key Strategies ................................................................................................................ 94


11 Scalar 360 Degree Market Perspective ......................................................................................... 95

12 Appendix ....................................................................................................................................... 97

12.1 Related Reports ..................................................................................................................... 98

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List of Tables

Table 1. Assumptions ...................................................................................................................... 20

Table 2. Global Orphan Drugs Market Summary ............................................................................ 22

Table 3. Prevalence of Rare Diseases by Major Countries ............................................................. 24

Table 4. List of Some of the Known Rare Diseases ......................................................................... 25

Table 5. Market Exclusivity for Orphan Drugs by Major Regions ................................................... 26

Table 6. Average Number of Patients in Clinical Trials ................................................................... 29

Table 7. Market Attractiveness for Orphan Drugs Setup ............................................................... 30

Table 8. Regulatory Analysis Snapshot ........................................................................................... 31

Table 9. EMA Approved Orphan Drugs (2015 – 2016) ................................................................... 33

Table 10. Orphan Drug Regulations in Other Countries ................................................................... 35

Table 11. Time to Entry Analysis (Standard Drugs vs. Orphan Drugs) .............................................. 36

Table 12. Average Time for FDA Approval ........................................................................................ 36

Table 13. Orphan Drugs in Phase 3 Stage of the Development (2015) ............................................ 39

Table 14. Pricing with respect to prevalence of rare disease ........................................................... 40

Table 15. Average Price of Orphan Drugs by Type ........................................................................... 40

Table 16. Pricing of Top Orphan Drugs with Average Market Price (2015) ...................................... 41

Table 17. List of 10 Top Marketed Orphan Drugs (2015) ................................................................. 42

Table 18. Global Orphan Drugs Market Size (US$ billion), By Type (2014 – 2022) .......................... 44

Table 19. Global Biological Orphan Drugs Market Size (US$ billion), By Regions (2014 – 2022) ..... 46

Table 20. Global Non-Biological Orphan Drugs Market Size (US$ billion), By Regions (2014 – 2022) .

........................................................................................................................................... 48

Table 21. Global Orphan Drugs Market Size (US$ billion), By Therapeutic Area (2014 – 2022) ...... 51

Table 22. Global Orphan Drugs Market for Oncology; Market Size (US$ billion) (2014 – 2022) ..... 53

Table 23. Global Orphan Drugs Market for Neurology, Market Size (US$ billion) (2014 – 2022) .... 54

Table 24. Global Orphan Drugs Market for Hematology, Market Size (US$ billion) (2014 – 2022) . 55

Table 25. Global Orphan Drugs Market for Infectious Disease, Market Size (US$ billion) (2014 –

2022) ........................................................................................................................................... 56

Table 26. Global Orphan Drugs Market for Metabolic Disease, Market Size (US$ billion) (2014 –

2022) ........................................................................................................................................... 57

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Table 27. Global Orphan Drugs Market for Other Diseases, Market Size (US$ billion) (2014 – 2022)

........................................................................................................................................... 58

Table 28. Orphan Drugs Market Prospects, by Geography (2016 – 2022) ....................................... 60

Table 29. Global Orphan Drugs Market Size (US$ billion), By Geography (2014 – 2022) ................ 61

Table 30. Global Orphan Drugs Market, Market Size (%), By Major Regions (2014 – 2022) ........... 62

Table 31. Orphan Drugs Market in North America, Market Size (US$ billion) (2014 – 2022) .......... 64

Table 32. Orphan Drugs Market in U.S., Market Size (US$ billion), (2014 – 2022) .......................... 64

Table 33. Orphan Drugs Market in Canada, Market Size (US$ billion), (2014 – 2022) ..................... 65

Table 34. Orphan Drugs Market in North America, Market Size (US$ billion), By Type (2014 – 2022)

........................................................................................................................................... 65

Table 35. Orphan Drugs Market in North America, Market Size (US$ billion), By Therapeutic Area

(2014 – 2022) ........................................................................................................................................ 66

Table 36. Orphan Drugs Market in Europe, Market Size (US$ billion) ............................................. 69

Table 37. Orphan Drugs Market in Germany, Market Size (US$ billion), (2014 – 2022) .................. 70

Table 38. Orphan Drugs Market in U.K, Market Size (US$ billion), (2014 – 2022) ........................... 70

Table 39. Orphan Drugs Market in France, Market Size (US$ billion), (2014 – 2022) ...................... 70

Table 40. Orphan Drugs Market in Italy, Market Size (US$ billion), (2014 – 2022) .......................... 70

Table 41. Orphan Drugs Market in Rest of Europe, Market Size (US$ billion), (2014 – 2022) ......... 71

Table 42. Orphan Drugs Market in Europe, Market Size (US$ billion), By Type (2014 – 2022) ....... 71

Table 43. Orphan Drugs Market in Europe, Market Size (US$ billion), By Therapeutic Area (2014 –

2022) ........................................................................................................................................... 73

Table 44. Orphan Drugs Market in Asia-Pacific, Market Size (US$ billion) ....................................... 75

Table 45. Orphan Drugs Market in China, Market Size (US$ billion), (2014 – 2022) ........................ 76

Table 46. Orphan Drugs Market in India, Market Size (US$ billion), (2014 – 2022) ......................... 76

Table 47. Orphan Drugs Market in Australia, Market Size (US$ billion), (2014 – 2022) .................. 76

Table 48. Orphan Drugs Market in Japan, Market Size (US$ billion), (2014 – 2022) ....................... 76

Table 49. Orphan Drugs Market in South Korea, Market Size (US$ billion), (2014 – 2022) ............. 76

Table 50. Orphan Drugs Market in Rest of Asia-Pacific, Market Size (US$ billion), (2014 – 2022) .. 77

Table 51. Orphan Drugs Market in Asia-Pacific, Market Size (US$ billion), By Type (2014 – 2022) . 77

Table 52. Orphan Drugs Market in Asia-Pacific, Market Size (US$ billion), By Therapeutic Area

(2014 – 2022) ........................................................................................................................................ 78

Table 53. Orphan Drugs Market in Rest of the World (ROW), Market Size (US$ billion) ................. 80

Table 54. Orphan Drugs Market in Latin America, Market Size (US$ billion), (2014 – 2022)........... 81

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Table 55. Orphan Drugs Market in Middle East & Africa, Market Size (US$ billion), (2014 – 2022) 81

Table 56. Orphan Drugs Market in Rest of the World (ROW), Market Size (US$ billion), By Type

(2014 – 2022) ........................................................................................................................................ 81

Table 57. Orphan Drugs Market in Rest of the World (ROW), Market Size (US$ billion), By

Therapeutic Area (2014 – 2022) ........................................................................................................... 82

Table 58. Revenue from Orphan Drugs (US$ billion) By Major Players (2014 & 2015) .................... 85

Table 59. Orphan Drugs Market Snapshot ....................................................................................... 96

Table 60. Acronyms .......................................................................................................................... 97

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List of Figures

Research Methodology ..................................................................................................... 16

Orphan Drugs Timeline ..................................................................................................... 30

Orphan Drugs by Stage of Development (2016) ............................................................... 37

Orphan Drugs in Pipeline, by Therapeutic Area (2016) .................................................... 38

Global Orphan Drugs Market By Type: Market Size vs. Market Penetration (2015) ........ 43

Global Orphan Drugs Segment Share (%), By Type (2016 & 2022) ................................... 44

Global Orphan Drugs Market Size (US$ billion), By Type (2016 & 2022) .......................... 45

Global Biological Orphan Drugs Segment Share (%), By Regions (2016 & 2022) ............. 46

Global Biological Orphan Drugs Market Size (US$ billion), By Regions (2016 & 2022) .... 47

Global Non-Biological Orphan Drugs Segment Share (%), By Regions (2016 & 2022) . 48

Global Non-Biological Orphan Drugs Market Size (US$ billion), By Regions (2016 &

2022) ....................................................................................................................................... 49

Market Attractiveness for Orphan Drugs, By Therapeutic (2016) ................................ 50

Global Orphan Drugs Segment Share (%), By Therapeutic Area (2016 & 2022)........... 51

Global Orphan Drugs Market Size (US$ billion), By Therapeutic Area (2016 & 2022).. 52

Global Orphan Drugs Market for Oncology, Market Size (US$ billion) (2016 & 2022) . 53

Global Orphan Drugs Market for Neurology, Market Size (US$ billion) (2015 & 2022)54

Global Orphan Drugs Market for Hematology, Market Size (US$ billion) (2016 & 2022)

....................................................................................................................................... 55

Global Orphan Drugs Market for Infectious Disease, Market Size (US$ billion) (2016 &

2022) ....................................................................................................................................... 56

Global Orphan Drugs Market for Metabolic Disease, Market Size (US$ billion) (2016 &

2022) ....................................................................................................................................... 57

Global Orphan Drugs Market for Other Diseases; Market Size (US$ billion) (2016 &

2022) ....................................................................................................................................... 58

Global Orphan Drugs Market, Segment Share (%), By Geography (2016 & 2022) ....... 61

Orphan Drugs Market in North America, Snapshot ...................................................... 63

Orphan Drugs Market in North America, Market Size (US$ billion) (2014-2022) ........ 63

Orphan Drugs Market in North America, Segment Share (%) By Regions, (2016 &

2022) ....................................................................................................................................... 64

Orphan Drugs Market in North America, Market Size (US$ billion), By Type, 2016

&2022 ....................................................................................................................................... 66

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Orphan Drugs Market in North America, Market Size (US$ billion), By Therapeutic

Area (2016 & 2022) ............................................................................................................................... 67

Orphan Drugs Market in Europe, Snapshot .................................................................. 68

Orphan Drugs Market in Europe, Market Size (US$ billion) (2014-2022) ..................... 69

Orphan Drugs Market in Europe, Segment Share (%) By Regions, (2016 & 2022) ....... 69

Orphan Drugs Market in Europe, Market Size (US$ billion), By Type (2016 & 2022) ... 71

Orphan Drugs Market in Europe, Market Size (US$ billion), By Therapeutic Area (2016

& 2022) ....................................................................................................................................... 73

Orphan Drugs Market in Asia-Pacific, Snapshot ........................................................... 74

Orphan Drugs Market in Asia-Pacific, Market Size (US$ billion) (2014 – 2022) ........... 75

Orphan Drugs Market in Asia-Pacific, Segment Share (%) By Regions, (2016 & 2022) 75

Orphan Drugs Market in Asia-Pacific, Market Size (US$ billion), By Type (2016 & 2022)

....................................................................................................................................... 77

Orphan Drugs Market in Asia-Pacific, Market Size (US$ billion), By Therapeutic Area

(2016 & 2022) ....................................................................................................................................... 78

Orphan Drugs Market in Rest of the World, Snapshot ................................................. 79

Orphan Drugs Market in Rest of the World, Market Size (US$ billion) (2014 – 2022) . 80

Orphan Drugs Market in Rest of the World, Segment Share (%) By Regions, (2016 &

2022) ....................................................................................................................................... 80

Orphan Drugs Market in Rest of the World, Market Size (US$ billion), By Type (2016 &

2022) ....................................................................................................................................... 82

Orphan Drugs Market in Rest of the World, Market Size (US$ billion), By Therapeutic

Area (2016 & 2022) ............................................................................................................................... 83

Market Share (%) of Major Players (2015) .................................................................... 84

Market Positioning of Key Players ................................................................................ 86

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1 Introduction

The ‘Orphan Drugs Market Global Industry Analysis Report’ presents major

qualitative and quantitative trends in the global orphan drugs market. The report also

identifies important factors driving market growth and provides a profile of key industry

players.

The report provides market size, segmentation, and growth forecast for the global

orphan drugs market. This report segments the market on the basis of type and

therapeutic area. The product type segments included in this report are biological

orphan drugs, and non-biological orphan drugs. The therapeutic areas included in the

report are oncology, neurology, Hematology, infectious disease, metabolic disease

and other. The report includes detailed market size and forecast information for all

these market segments. Furthermore, this report also features the detailed geographic

analysis by major continents and countries. The first section on market overview

discusses the definitions, assumptions and research methodology considered while

preparing the report. The following section of market dynamics examines the impact

of market drivers, restraints, opportunities and trends for years 2016, 2019 and 2022

on global orphan drugs market. The discussion of industry analysis offers information

on regulatory analysis, pipeline analysis, time to entry analysis, and pricing analysis

of top orphan drugs. Section 9 provides with the competitive landscape which gives

the market share analysis and market positioning of key market players in the Orphan

Drugs market for the year 2015. This report also features profiles of top companies in

the global orphan drugs market.

In total, the information contained in this report is intended to communicate a fairly

complete understanding of global orphan drugs market through a series of easily

digestible nuggets. On behalf of Scalar Market Research team and Alcor group, we

hope that you explore and find value in this report. Suggestions for customization,

further information or other improvements are most welcome.

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2 Market Overview

2.1 Definitions

Global orphan drugs market is defined as the market size and market share estimation

of the various segments of the Orphan Drugs market, capturing ongoing drivers,

trends, challenges of the market, industry analysis involving the regulatory analysis

and studying about the major participants and players in Orphan Drugs market

environment.

The 'Global Orphan Drugs Market – Market Size and Forecast (2014 – 2022)'

report includes market size and forecast estimates for the period of 2014 to 2022. The

scope of this report is commercially available orphan drugs, regulatory analysis with

respect to geographies, pipeline analysis, and its classification in oncology, neurology,

Hematology, infectious diseases, metabolic diseases, and others. The report has 2014

as the historical year, 2015 as base year and 2016 to 2022 as forecast years.

This report defines the Orphan Drugs as –

Orphan Drugs are the pharmaceutical drugs which can be biologically derived or non-

biologically derived and are majorly used for the treatment of rare diseases. Hence,

these drugs are called as ‘orphan’ drugs. The orphan drug designation is different for

different countries. For Example; a pharmaceutical drug gets an orphan status if the

affected population with a rare disease is less than 200,000 in the U.S. and 5 out of

10,000 in Europe.

Orphan Drugs Market by Type as –

Orphan Drugs that are biological, non-biological and that are commercially available

in the market for treatment of rare and ultra-rare diseases.

Orphan Drugs Market by Therapeutic Area as –

Orphan Drugs in therapeutic areas such as oncology, neurology, hematology,

infectious disease, metabolic disease and other.

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2.2 Research Methodology

We utilize a mix of primary research, secondary research and machine learning

algorithms to generate comprehensive data about market sizes and revenue

forecasts. Our primary focus on continuous market tracking, rigorous fact-checking,

data-triangulation, and multiple layers of quality control ensures high-quality data that

can be leveraged for actionable research insights.

2.2.1 Primary Data

Structured interviews with industry specialists, VPs, CEOs, directors, technology

consultants were taken in order to collect the key primary data. The primary data is

focused on both the demand and supply sides of the market. Market engineering has

been carried out from the data and excessive analysis has been done on it. Primary

data is also extensively considered while validating the secondary data.

Perspective Primary Research

Supply Side

Manufacturers

Wholesalers

Distributors

Demand Side End-users

Consumers

Report Parameters Primary Sources

Key Industry Participants (KPIs)

Suppliers

Government Agencies

Regulatory Bodies

Research & Development Companies

Investment Research Companies

Business Consulting Service Providers

Academic Institutions/Universities

Venture Capitalists

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2.2.2 Secondary Data

The data from important secondary sources is utilized to obtain industry supply chains,

company profiles and other key areas such as market drivers, opportunities, trends

from the market perspective. In addition, secondary data is gathered using paid

databases such as Bloomberg, Hoovers, Factiva, and government publishing sources.

Report Parameters Perspective Secondary Research

Market Size, Revenue,

Industry Analysis,

Premium Industry

Trends, Market

Segmentation,

Geographic Analysis, Key

Market Players,

Competitive Landscape

Supply Side

Company Annual Reports

SEC Filings

Financial Journals

Trade Journals

Market Journals

Press Releases

Directories

White Papers

Company Websites

Magazines

Demand Side Case Studies

Customer References

2.2.3 Machine Learning

In addition to assigning weights to all the relevant market parameters, to quantify their

market impact for generating projected market growth rate, we use advanced machine

learning algorithms to collate essential market data. The machine learning method

utilizes algorithms such as regression, correlation and decision tree, among others, to

derive the desired output.

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Perspective Advantages of Machine Learning-based Forecasts

Using Machine Learning for

Data Forecast

Unlimited Data

Highly Detailed Forecasts

Profound Knowledge of Demand

Broad applicability

Customizable Model Identifies Drivers

Analysis - Product, Value Chain, Supply Chain,

Competitor

2.2.4 Research Scope

We have utilized the top-down, bottom-up approach to study the market size, industry

dynamics, key regional markets, and major market trends. The global market has been

estimated by integrating the regional markets, estimating the market data for the base

year 2015 and annual share and revenue forecasts to 2022 in US$.

Research Methodology

Global

Market Size

Market-specific Revenues of Key Players

Total Revenues of Key Players

Global Market Size

Segment-specific Market Size

Regional

Market

Size

Bottom-up

Top-down

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2.2.5 Years Considered

Market Specifics Report Scope

Historical Year 2014

Base Year 2015

Estimated Year 2016

Forecast Period 2016 – 2022

Revenue Currency US$

2.2.6 Currency & Pricing

The currency used in the report is the U.S. dollars, with market size indicated in US$

million and US$ billion.

Revenue is obtained from the annual company reports (For all the companies

reporting their revenue in US$)

Revenue is converted as per the annual currency exchange rate for that

particular year (For the companies reporting their revenue in currencies other

than US$)

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2.3 Market Segmentation

Source: Scalar Market Research Analysis

Orphan Drugs Market, By Type

Biological Non-Biological

Orphan Drugs Market, By Region

North America Europe Asia-Pacific Rest of World

Orphan Drugs Market, By Therapeutic Area

NeurologyOncology OthersHaematologyInfectious

DiseaseMetabolic

Disease

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2.4 Key Objectives & Benefits of the Report

2.4.1 Objectives of the Report

To offer in-depth information about the key market drivers and restraints

influencing the growth of this market during the forecast period

To analyze the major challenges and opportunities expected to play crucial role

in the revenue and market size growth of the key players in this market

To forecast the market size, in terms of the market size and revenue for all its

segments with respect to the major geographic regions including North

America, Europe, Asia-Pacific, Middle East & Africa, and Latin America

To comprehensively analyze the strategic developments such as mergers &

acquisition, new product launches, business expansions, and the new business

agreements in the Orphan Drugs market

To define the high-growth segments of the market and offer in-depth analysis

of the competitive landscape in the market for various key players

2.4.2 Key Benefits of the Report

In-depth analysis of the market segments that will showcase the greatest

growth potential

Deeper understanding of the behavior of the key players and innovators

influencing this market

Granular understanding of the historical market trend and the resultant market

forecast

Information about the technological advancements influencing the growth of the

global market

Extensive evaluation of all the market dynamics that will impact this market

during the forecast period

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2.5 Assumptions

Table 1. Assumptions

Parameter Assumptions

Global Orphan Drugs Market, By Type

Global Orphan Drugs Market, By Therapeutic

Area

Global Orphan Drugs Market. By Region

Market Share Analysis, By Major Players


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3 Executive Summary

Orphan Drugs are the pharmaceutical drugs which can be biologically derived or non-

biologically derived and is majorly used for the treatment of rare diseases. Hence,

these drugs are called as ‘orphan’ drugs. The orphan drug designation is different for

different countries. For Example; a pharmaceutical drug gets an orphan status if the

affected population with a rare disease is less than 200,000 in the U.S. and 5 out of

10,000 in Europe.

The Orphan Drugs market offers many growth opportunities and driving factors as

compared to the restraining factors. It is stated by various research scientists that

development of Orphan Drugs will drastically help the people suffering from rare and

ultra-rare diseases. Thus, large number of key companies such as Novartis, Roche,

Celgene, Amgen, Alexion Pharmaceuticals, and others are in constant efforts to

develop orphan drugs. For which, these companies are rigorously increasing their

research and development expenses on clinical trials to introduce novel Orphan Drugs

which would ultimately drive the market growth. Furthermore, demand for Orphan

Drugs is also expected to increase due to the rise in the incidence of rare disease and

population affected by these diseases. Orphan Drugs have proven a promising option

for the disease diagnosis and mitigation. Other factors such as faster regulatory

approvals, longer market exclusivity, positive result of many orphan drugs during

clinical trials and others drive the market growth to a great extent. However, high costs

of treatment and shift towards stem cell therapy and gene therapy like treatments are

expected to hamper the market growth during the future period.

The major revenue opportunities are expected to come from therapeutic areas such

as Oncology, Metabolic Disease, Infectious Diseases and Neurology. Whereas

oncology will account for XX% of the total revenue generated by 2022, neurology and

metabolic disease will demand a great potential towards Orphan Drugs market.

Biological orphan drugs will continue to dominate in the Orphan Drugs market while

Non-Biological orphan drugs will see increasing growth rate. Areas for investment in

the Orphan Drugs market will focus on improving research and development for the

manufacturing of Orphan Drugs, improving supply chain and reduction in the treatment

price.

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The demand for finished Orphan Drugs products is expected to shift to Asia-Pacific

during the forecast period. The countries such as India, Australia, Japan, and

Germany will continue to be in the race for Orphan Drugs market.

The Global Orphan Drugs market was estimated to be US$ 101.82 billion in 2015. This

market is expected to grow at a CAGR of 11.4% in the forecast period and is expected

to be US$ 214.14 billion in 2022. This report segments the Orphan Drugs market by

type, therapeutic area and regions.

Table 2. Global Orphan Drugs Market Summary

Global Orphan Drugs Market Market Size (US$ billion) CAGR%

(2016-2022) 2016 2022

Total 111.87 214.14 11.4%

Typ

e

Biological Orphan Drugs

Non-Biological Orphan Drugs

Ther

apeu

tic

Are

a

Oncology

Neurology

Hematology

Infectious Disease

Metabolic Disease

Other

Geo

grap

hy

North America

Europe

Asia-Pacific

Rest of the World


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4 Market Dynamics

4.1 Drivers

Drivers Impact on the Market

2014 – 2016 2017 – 2019 2020 – 2022

Increasing prevalence of rare

diseases

●=High ◑=Medium ◔=Low


1. Increasing prevalence of rare diseases

The prevalence of rare diseases has become a challenge for the world as there are

more than 7000 known rare diseases. This count is increasing day by day with 50%

of the affected population of rare diseases being children, and most of the rare

diseases are of genetic origin. Though the definition of rare disease states for ratio

1:10, there are an estimated 350 million people affected with the rare disease in the

world.

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5 Industry Analysis

5.1 Industry Overview

Orphan Drugs Timeline


Table 7. Market Attractiveness for Orphan Drugs Setup

Parameter Regulated Markets Semi-Regulated

Markets

Non-Regulated

Markets

R&D Costs/Production

Costs

Clinical Trials

Capability

Regulatory Rigidity

Attractiveness to

Consumers

●=Favorable ◑=Less Favorable ◔=Not Favorable


U.S publishes the orphan drugs act and becomes the first country to support the development of orphan drugs

Japan passes the legislation for orphan drugs

Australia passes the legislation for orphan drugs

Orphan drugs constituted 22% of total drug sales

EU passes the legislation for orphan drugs

More than 7000 rare disease occurrences

Increased prevalence of rare diseases; 25 million people affected with rare disease in U.S

1983 1993 1997 2000 2010 2012 2015

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6 Orphan Drugs Market, By Type

The type segments of the Orphan Drugs market included in this report are Biological

Orphan Drugs and Non-Biological Orphan Drugs. Biological Orphan Drugs continues

to be the largest drug type in 2015, while Non-Biological Orphan Drugs is expected to

possess great potential during the forecast period.

Table 17. List of 10 Top Marketed Orphan Drugs (2015)

Orphan Drug Orphan Drug Type Company Revenue (US$

millions)

Revlimid Biological Celgene 5,801

Soliris Biological Alexion

Pharmaceuticals

2,590

Rituxan Biological Roche 7,321

Tasigna Non-Biological Novartis 1,632

Advate Biological Baxalta 2,240

Yervoy Biological Bristol-Myers Squibb 1,126

Sprycel Non-Biological Bristol-Myers Squibb 1,620

Pomalyst Non-Biological Celgene 983

Keytruda Biological Merck & Co 566

Alimta Non-Biological Eli Lily 2,493


The table gives the clear indication that majority of the top-selling orphan drugs are

biologics and most of these drugs are used in the treatment of cancers. Revlimid from

Celgene was the top-selling orphan drug with annual revenue of US$ 5.80 billion.

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Global Orphan Drugs Market By Type: Market Size vs. Market Penetration

(2015)


Market Penetration

Ma

rke

t Siz

e

High

Hig

h



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6.1 Share Segmentation

Global Orphan Drugs Segment Share (%), By Type (2016 & 2022)


Biological Orphan Drugs was the largest drug segment in the global orphan drugs

market accounting for XX% market share. The year 2015, also saw the decrease of

non-biological orphan drugs market share as compared to 2014.

Scalar Market Research Analysis predicts that the biological orphan drugs will

continue to dominate during the forecast period.

Table 18. Global Orphan Drugs Market Size (US$ billion), By Type (2014 – 2022)

Type 2014 2015 2016 2017 2018 2019 2020 2021 2022 CAGR%

(2016 – 2022)

Biological

Non-

Biological

Total


Biological orphan drugs account for the largest orphan drug type segment of the

Orphan Drugs market in 2015. This market segment is expected to grow at a CAGR

of XX% from US$ XX billion in 2015 to US$ XX billion in 2022.

64.4%

35.6%

2016

65.7%

34.3%

2022

Biological

Non-Biological

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Global Orphan Drugs Market Size (US$ billion), By Type (2016 & 2022)


72.05

140.79

39.82

73.36

2016 2022

Biological Non-Biological

Mar

ketS

ize

(US$

Bil

lion

)

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7 Orphan Drugs Market, By Therapeutic Area

The application segments of the Orphan Drugs market included in this report are

Oncology, Neurology, Hematology, Infectious Disease, Metabolic Disease, and Other

Diseases. Oncology was the largest therapeutic segment for orphan drugs in 2015,

and it’s also expected to be the fastest growing application segment in the forecast

period.

Market Attractiveness for Orphan Drugs, By Therapeutic (2016)

Therapeutic Area Market Size Growth Rate Overall Attractiveness

Oncology

Neurology

Hematology

Infectious Disease

Metabolic Disease

Other Diseases

●=Significantly Attractive ◑=Attractive ◔=Less

Attractive Source: Scalar Market Research Analysis

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7.1 Share Segmentation

Global Orphan Drugs Segment Share (%), By Therapeutic Area (2016 & 2022)


Oncology was the largest segment in the Global Orphan Drugs market for therapeutic

segment accounting for 35.1% market share and is expected to continue its

dominance by the end of 2022.

Table 21. Global Orphan Drugs Market Size (US$ billion), By Therapeutic Area (2014 –

2022)

Therapeutic 2014 2015 2016 2017 2018 2019 2020 2021 2022 CAGR%

(2016 – 2022)

Oncology

Neurology

Hematology

Infectious

Disease

Metabolic

Disease

Other

Total


35.3%

7.2%

7.1%6.5%

12.0%

31.9%

2016

36.3%

7.3%

7.5%6.5%

13.6%

28.9%

2022Oncology

Neurology

Haematology

Infectious Disease

Metabolic Disease

Others

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Global Orphan Drugs Market Size (US$ billion), By Therapeutic Area (2016 &

2022)


39.47

8.10 7.97 7.2213.46

35.64

77.71

15.61 15.95 13.88

29.06

61.93

Oncology Neurology Haematology Infectious Disease

Metabolic Disease

Others

2016 2022

Mar

ketS

ize

(US$

Bil

lion

)

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8 Orphan Drugs Market, By Geography

The regional segments of the Orphan Drugs market included in this report are North

America (NA), Europe (EU), Asia-Pacific (APAC), and the Rest of the World (ROW).

North America was the largest regional segment in 2015, while Asia-Pacific is

expected to be the fastest-growing regional segment in the forecast period.

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Table 28. Orphan Drugs Market Prospects, by Geography (2016 – 2022)

Geography Growth Prospects Indicators

North America

Europe

Asia-Pacific

Latin America

Middle East &

Africa


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Global Orphan Drugs Market, Segment Share (%), By Geography (2016 &

2022)


Europe was the largest regional segment of the Orphan Drugs market in 2015. The

market had a XX% share of the global market in 2015. However, Europe is expected

to lose its market share to North America by 2022.

Table 29. Global Orphan Drugs Market Size (US$ billion), By Geography (2014 – 2022)

Geography 2014 2015 2016 2017 2018 2019 2020 2021 2022 CAGR%

(2016 – 2022)

North

America

Europe

Asia-Pacific

Rest of

World

Total


Asia-Pacific is expected to be the largest growing regional segment with a CAGR of

XX% during the forecast period.

55.5%24.6%

16.5%

3.4%

2016

54.9%

22.9%

18.2%

4.0%

2022

NA

Europe

APAC

ROW

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8.1 North America

Orphan Drugs Market in North America, Snapshot

Orphan Drugs Market in North America Market Size (US$ billion) CAGR%

(2016-2022) 2016 2022

Total

Typ

e



Ther

apeu

tic

Are

a

Oncology

Neurology

Hematology

Infectious Disease

Metabolic Disease

Other

Reg

ion

s United States

Canada


Orphan Drugs Market in North America, Market Size (US$ billion) (2014-2022)


53.67 56.54 62.06

68.40 75.71

84.18 94.10

105.21

117.63

2014 2015 2016 2017 2018 2019 2020 2021 2022

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Table 31. Orphan Drugs Market in North America, Market Size (US$ billion) (2014 –

2022)

2014 2015 2016 2017 2018 2019 2020 2021 2022 CAGR% (2016-2022)

Market Size


8.1.1 By Regions

Orphan Drugs Market in North America, Segment Share (%) By Regions,

(2016 & 2022)


8.1.1.1 U.S.

Table 32. Orphan Drugs Market in U.S., Market Size (US$ billion), (2014 – 2022)

Country 2014 2015 2016

2017 2018 2019

2020 2021 2022 CAGR% (2016-2022)

United States

Source: Scalar Market Research Analysi

86.7%

13.3%

2016

88.3%

11.7%

2022

United States

Canada

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9 Competitive Landscape

9.1 Market Share Analysis

Market Share (%) of Major Players (2015)


The above chart gives the market share of the top leading players in the Orphan Drugs

market. The companies Novartis, Alexion Pharmaceuticals, Roche, Sanofi, and

Celgene, accounts for almost XX% of the market share in this market. However, these

companies are expected to lose their market share to other major players such as

Pfizer and Eli Lilly in the long run. Novartis holds the number one position and is

expected to remain on the top in the coming years because of its long portfolio of

orphan drugs. Shire is expected to enter in top 5 following its recent acquisition of

Baxalta for US$32 billion in June, 2016.

9.4%

2.5%

6.7%

4.3%

7.9%

3.2%66.0%

Novartis

Alexion Pharmaceuticals

Celgene

Shire

Roche

Sanofi

Others

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11 Scalar 360 Degree Market Perspective

The Global Orphan Drugs market is estimated to grow from US$ 111.87 billion in 2016

to US$ 214.14 billion in 2022, at a CAGR of 11.4% during the forecast period of 2016

– 2022. Biological Orphan Drugs will account for maximum share during the forecast

period whereas the market for Non-Biological Orphan will see a huge potential in the

coming years.

The major revenue opportunities are expected to come from therapeutic areas such

as oncology and metabolic diseases. Oncology will account for 36.29% market share

whereas the market for Orphan Drugs in neurology, Hematology and infectious

diseases will see huge opportunities in the Asia-Pacific regions.

North America will continue to be the region for developments in Orphan Drugs market

whereas Japan will account for major opportunities by 2022. Countries like India,

China, Germany, Australia, and Singapore will also see a huge potential for expansion

of this market in the forecast period.

The leading players in the Orphan drugs market are Novartis, Celgene, Roche, Alexion

Pharmaceuticals, BioMarin Pharmaceuticals, Sanofi, Pfizer, and Amgen. Scalar

Market research study believes that Novartis will continue to dominate the orphan

drugs market by 2022 and could reach a market share of 11%.

Forward Thinking: Orphan Drugs Shows Great Potential for Commercialization

Despite of less population of rare diseases and less patient pool, the

commercialization of the development of orphan drugs has been more

attractive than the non-orphan drug development.

The economic development of orphan drugs development is largely driven by

premium pricing, tax credits, faster rate of regulatory approvals, longer market

exclusivity, and lesser marketing costs.

Also, the rising prevalence of rare diseases in different geographies is attracting

big pharmaceuticals for the development of orphan drugs.

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Table 59. Orphan Drugs Market Snapshot

Criteria Orphan Drugs Market

Business Maturity

Type of Market Players

Number of Market Players

Market Perimeter

R&D Investment

Market Evolutionary Stage

Growth Strategies


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12 Appendix

Table 60. Acronyms

CAGR Compounded Annual Growth Rate

NA North America

EU Europe

APAC Asia-Pacific

MEA Middle East & Africa

LA Latin America

NR Not Reported

R&D Research & Development

EMA European Medicines Agency


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12.1 Related Reports

Healthcare Business Process Outsourcing (BPO) Market, by Provider Service

(Medical Billing, Medical Coding, Medical Transcription and Others), Payer

Services (Member Management Services, Claims Management Services,

Provider Management Services and Others), Pharmaceutical Services

(Research and Development, Manufacturing and Non-Clinical Services) -

Global Revenue, Trends, Growth, Share, Size and Forecast to 2022

Biosimilars Market, by Product Types (Recombinant Glycosylated Proteins,

Recombinant Non-Glycosylated Proteins, Recombinant Peptides), Applications

(Oncology, Blood Disorders, Infectious Diseases, Autoimmune Diseases, and

Others) - Global Revenue, Trends, Growth, Share, Size and Forecast to 2022

Active Pharmaceutical Ingredient Market, By Types (Synthetic Chemical,

Biological), Therapeutic Areas (Cardiovascular Disorders, Metabolic Disorders,

Neurological Disorders, Oncology, NSAIDs, Other Disorders), Manufacturing

Formats (Captive, Contract) - Global Revenue, Trends, Growth, Share, Size

and Forecast to 2022

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99

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