Orphan Drugs Market Report 2017-2023

  • September, 2017
  • Domain: Healthcare - Pharmaceuticals
Pages: 125
Tables: 41
Charts: 39
Regions/Countries: 4
Companies: 12
Enquiry Hours: 10
  • Get Free 10% Customization in this Report

Global Orphan Drugs Market Orphan Drugs Market-Global drivers, restraints, opportunities, trends and forecasts to 2023 By Drug Types (Biologic, Non-Biologic), Application (Oncology, Metabolic Diseases, Haematology, Infectious Diseases, Neurology, Others) Region (North America, Europe, Asia-Pacific and Rest of the world) - Forecast to 2023


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.


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:

1     Industry Outlook  

1.1        Industry Overview

1.2        Industry Trends

2     Report Outline  

2.1        Report Scope

2.2        Report Summary

2.3        Research Methodology

2.4        Report Assumptions

3     Market Snapshot 

3.1        Market Definition – Infoholic Research

3.2        Segmented Addressable Market (SAM)

3.2.1     Advantages of Development of Orphan Drug

3.2.2     Trends of orphan drug market

3.3        Related Addressable Markets (RAM)

3.3.1     Oncology (Cancer) Drugs

3.3.2     Active pharmaceutical ingredients (APIs)

3.3.3     Over the counter drugs (OTC)

4     Market Outlook  

4.1        Overview

4.2        Regulatory Framework for orphan drugs

4.3        Funding Scenario for orphan drugs

4.4        Market Segmentation

4.5        Porter 5(Five) Forces

4.6        PEST Analysis

5     Market Characteristics 

5.1        Market Dynamics

5.1.1     Drivers    Conducive government legislation    Marketing exclusivity for orphan drugs    Growing unmet need for treatment of orphan diseases

5.1.2     Opportunities    Growing Novel indications for known orphan drugs    Untapped emerging market

5.1.3     Restraints    High cost of treatment    Lack of Adequate patients for clinical trials

5.1.4     DRO – Impact Analysis

5.1.5     Key Stakeholders

6     Drug Details: Market Size and Analysis

6.1        Overview

7     Types: Market Size and Analysis 

7.1        Overview

7.1.1     Biologics

7.1.2     Non-Biologics

8     Application: Market Size and Analysis 

8.1        Overview

8.1.1     Oncology

8.1.2     Metabolic Diseases

8.1.3     Hematology

8.1.4     Infectious Diseases

8.1.5     Neurology

8.1.6     Others

9     Pipeline Molecules: Market Size and Analysis 

9.1        Overview

10   Regions: Market Size and Analysis 

10.1     Overview

10.2     North America

10.2.1  Overview

10.2.2  US

10.2.3  Canada

10.3     Europe

10.3.1  Overview UK Germany France

10.4     APAC

10.4.1  Overview  India Japan China

10.5     Rest of the World

10.5.1  Overview Brazil Mexico Africa Middle East

11   Competitive Landscape  

12   Vendor Profiles 

12.1     Novartis AG

12.1.1  Overview

12.1.2  Business Unit

12.1.3  Geographic Presence

12.1.4  Business Focus

12.1.5  SWOT Analysis

12.1.6  Business Strategy

12.2     F.Hoffmann-La Roche Ltd

12.2.1  Overview

12.2.2  Business Unit

12.2.3  Geographic Presence

12.2.4  Business Focus

12.2.5  SWOT Analysis

12.2.6  Business Strategy

12.3     Celgene Corporation

12.3.1  Overview

12.3.2  Geographic Presence

12.3.3  Business Focus

12.3.4  SWOT Analysis

12.3.5  Business Strategy

12.4     Bristol-Myers Squibb

12.4.1  Overview

12.4.2  Geographic Presence

12.4.3  Business Focus

12.4.4  SWOT Analysis

12.4.5  Business Strategy

12.5     Shire PLC

12.5.1  Overview

12.5.2  Business Unit

12.5.3  Geographic Presence

12.5.4  Business Focus

12.5.5  SWOT Analysis

12.5.6  Business Strategy

13   Companies to Watch For 

13.1     Pfizer Inc.,

13.1.1  Overview

13.1.2  Highlights

13.2     Sanofi

13.2.1  Overview

13.3     Bayer AG

13.3.1  Overview

13.4     Alexion Pharmaceutical Inc.

13.4.1  Overview

13.5     Biogen Pharmaceuticals.

13.5.1  Overview

13.6     Eli Lilly and Company.

13.6.1  Overview

13.7     Amgen Inc.

13.7.1  Overview



Research Framework

Infoholic research works on a holistic 360° approach in order to deliver high quality, validated and reliable information in our market reports. The Market estimation and forecasting involves following steps:

  • Data Collation (Primary & Secondary)
  • In-house Estimation (Based on proprietary data bases and Models)
  • Market Triangulation
  • Forecasting

Market related information is congregated from both primary and secondary sources.

Primary sources

involved participants from all global stakeholders such as Solution providers, service providers, Industry associations, thought leaders etc. across levels such as CXOs, VPs and managers. Plus, our in-house industry experts having decades of industry experience contribute their consulting and advisory services.

Secondary sources

include public sources such as regulatory frameworks, government IT spending, government demographic indicators, industry association statistics, and company publications along with paid sources such as Factiva, OneSource, Bloomberg among others.

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