Global rare disease therapeutics market is anticipated to grow at an impressive CAGR in the upcoming five years. Rare diseases are being discovered at an alarming rate. Increasing number of these diseases has drived more research and development for the treatment of these diseases. Thus, driving the market toward the expected growth in the forecast years. Moreover, government across the countries are investing and taking initiatives for the research on the therapeutics and treatments for these diseases thus driving the market growth positively.

The global rare disease therapeutics market is segmented by drug type, therapeutic area, route of administration, distribution channel, and regional distribution. Based on drug type, the market is further segmented into biologics, and organic compounds. Biologics are the dominating segment of the market over the organic compounds on account of effective capacity of the biologics that invokes the quick and effective immune response for the disease. Moreover, research institutes are actively studying the genomics of the diseases and causing factors to get the effective therapeutics for these rare diseases. Furthermore, various rare cancer disorders now have effective biologics like recombinant technology, antibody technology, stem cell therapy and gene therapy to name some that are driving the growth of the market.

COVID-19 effected the global rare disease therapeutics market like the whole world economy. The market suffered a set back in the first half of 2020, owing to the pandemic that spread in all around the world. The countries were observing a complete lockdown and healthcare industry was totally invested in finding the prevention and cure for the COVID-19. Soon the vaccine for the virus was discovered. This discovery did slow down the ongoing studies on the rare disease therapeutics thus setting a drawback for the market. Now, the market has recovered and is expected to show considerable growth in the forecast period.

Some of the major competitors in the market are Novartis AG, Pfizer Inc., F. Hoffmann-La Roche Ltd., AbbVie Inc., Novo Nordisk A/S, Sanofi SA, Bayer AG, Amgen, Inc., Allergan plc., Eisai Co. Ltd., Bristol-Myers Squibb Company, Merck & Co., Inc., Vertex Pharmaceuticals Inc., BioMarin Pharmaceutical Inc., Teva Pharmaceutical Industries Ltd., among others. The companies are focussing on extensive research and developments activities to stay competitive in the market. Other competitive strategies include formation of alliances and partnerships.

Years considered for this report:

Historical Years: 2016 – 2019

Base Year: 2020

Estimated Year: 2021

Forecast Period: 2022 – 2026

Objective of the Study:

  • To analyze and forecast the market size of Global rare disease therapeutics market from 2016 to 2019.
  • To estimate and forecast the market size of Global rare disease therapeutics market from 2020 to 2026 and growth rate until 2026. 
  • To classify and forecast Global rare disease therapeutics market based on drug type, therapeutic area, route of administration, distribution channel, and regional distribution.
  • To identify dominant region or segment in the Global rare disease therapeutics market.
  • To identify drivers and challenges for Global rare disease therapeutics market.
  • To examine competitive developments such as expansions, new product launches, mergers & acquisitions, etc., in Global rare disease therapeutics market.
  • To identify and analyze the profile of leading players operating in Global rare disease therapeutics market.
  • To identify key sustainable strategies adopted by market players in Global rare disease therapeutics market.


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TechSci Research performed both primary as well as exhaustive secondary research for this study. Initially, TechSci Research sourced a list of manufacturers, suppliers, and service providers across the country. Subsequently, TechSci Research conducted primary research surveys with the identified companies. While interviewing, the respondents were also enquired about their competitors. Through this technique, TechSci Research could include the manufactures which could not be identified due to the limitations of secondary research. TechSci Research analyzed the manufactures, distribution channels and presence of all major players across the country.

TechSci Research calculated the market size of Global rare disease therapeutics market using a bottom-up approach, wherein data for various end-user segments was recorded and forecast for the future years. TechSci Research sourced these values from the industry experts and company representatives and externally validated through analysing historical data of these product types and applications for getting an appropriate, overall market size. Various secondary sources such as company websites, news articles, press releases, company annual reports, investor presentations and financial reports were also studied by TechSci Research.

Key Target Audience:

  • Rare disease therapeutics manufacturers, end users, service providers and other stakeholders
  • Distributers and suppliers of rare disease therapeutics and services and other stakeholders
  • Organizations, forums, and alliances related to rare disease therapeutics   
  • Market research and consulting firms

The study is useful in providing answers to several critical questions that are important for the industry stakeholders such as manufacturers, suppliers, end users, etc., besides allowing them in strategizing investments and capitalizing on market opportunities.

Report Scope:

In this report, Global rare disease therapeutics market has been segmented into following categories, in addition to the industry trends which have also been detailed below:

  • Market, By Drug Type:
    • Biologics
    • Organic Compounds
  • Market, By Therapeutic Area:
    • Cancer
    • Blood-related Disorders
    • Central Nervous System (CNS)
    • Respiratory Disorders
    • Musculoskeletal Disorders
    • Cardiovascular Disorders
    • Others
  • Market, By Route of Administration:

o   Oral

o   Injectable

o   Others

  • Market, By Distribution Channel:

o   Hospital Pharmacies

o   Retail Pharmacies

o   Specialty Pharmacies

  • Market, By Region:
    • North America
    • APAC
    • Europe
    • MEA
    • South America

Competitive Landscape

Company Profiles: Detailed analysis of the major companies present in Global rare disease therapeutics market.

Available Customizations:

With the given market data, TechSci Research offers customizations according to a company’s specific needs. The following customization options are available for the report:

Company Information

  • Detailed analysis and profiling of additional market players (up to five).
Global Rare Disease Therapeutics Market is an upcoming report to be released soon. If you wish an early delivery of this report or want to confirm the date of release, please contact us at sales@techsciresearch.com

1.    Product Overview

2.    Research Methodology

3.    Impact of COVID-19 on Global Rare Disease Therapeutics Market

4.    Executive Summary

5.    Voice of Customer

6.    Global Rare Disease Therapeutics Market Outlook

6.1.  Market Size & Forecast

6.1.1.     By Value

6.2.  Market Share & Forecast

6.2.1.     By Drug Type (Biologics, Organic Compounds)

6.2.2.     By Therapeutic Area (Cancer, Blood-related Disorders, Central Nervous System (CNS), Respiratory Disorders, Musculoskeletal Disorders, Cardiovascular Disorders, Others)

6.2.3.     By Route of Administration (Oral, Injectable, Others)

6.2.4.     By Distribution Channel (Hospital Pharmacies, Retail Pharmacies, Specialty Pharmacies)

6.2.5.     By Company (2020)

6.2.6.     By Region (North America, Europe, Asia-Pacific, South America, Middle East & Africa)

6.3.  Product Market Map

7.    North America Rare Disease Therapeutics Market Outlook

7.1.  Market Size & Forecast          

7.1.1.     By Value

7.2.  Market Share & Forecast

7.2.1.     By Drug Type

7.2.2.     By Therapeutic Area

7.2.3.     By Route of Administration

7.2.4.     By Distribution Channel

7.2.5.     By Country (United States; Canada; Mexico)

7.3.  North America: Country Analysis

7.3.1.     United States Rare Disease Therapeutics Market Outlook

7.3.1.1.         Market Size & Forecast

7.3.1.1.1.             By Value

7.3.1.2.         Market Share & Forecast

7.3.1.2.1.             By Drug Type

7.3.1.2.2.             By Therapeutic Area

7.3.1.2.3.             By Route of Administration

7.3.1.2.4.             By Distribution Channel

7.3.2.     Canada Rare Disease Therapeutics Market Outlook

7.3.2.1.         Market Size & Forecast

7.3.2.1.1.             By Value

7.3.2.2.         Market Share & Forecast

7.3.2.2.1.             By Drug Type

7.3.2.2.2.             By Therapeutic Area

7.3.2.2.3.             By Route of Administration

7.3.2.2.4.             By Distribution Channel

7.3.3.     Mexico Rare Disease Therapeutics Market Outlook

7.3.3.1.         Market Size & Forecast

7.3.3.1.1.             By Value

7.3.3.2.         Market Share & Forecast

7.3.3.2.1.             By Drug Type

7.3.3.2.2.             By Therapeutic Area

7.3.3.2.3.             By Route of Administration

7.3.3.2.4.             By Distribution Channel

8.    Europe Rare Disease Therapeutics Market Outlook

8.1.  Market Size & Forecast          

8.1.1.     By Value

8.2.  Market Share & Forecast

8.2.1.     By Drug Type

8.2.2.     By Therapeutic Area

8.2.3.     By Route of Administration

8.2.4.     By Distribution Channel

8.2.5.     By Country (France; Germany; UK; Italy; Spain)

8.3.  Europe: Country Analysis

8.3.1.     France Rare Disease Therapeutics Market Outlook

8.3.1.1.         Market Size & Forecast

8.3.1.1.1.             By Value

8.3.1.2.         Market Share & Forecast

8.3.1.2.1.             By Drug Type

8.3.1.2.2.             By Therapeutic Area

8.3.1.2.3.             By Route of Administration

8.3.1.2.4.             By Distribution Channel

8.3.2.     Germany Rare Disease Therapeutics Market Outlook

8.3.2.1.         Market Size & Forecast

8.3.2.1.1.             By Value

8.3.2.2.         Market Share & Forecast

8.3.2.2.1.             By Drug Type

8.3.2.2.2.             By Therapeutic Area

8.3.2.2.3.             By Route of Administration

8.3.2.2.4.             By Distribution Channel

8.3.3.     United Kingdom Rare Disease Therapeutics Market Outlook

8.3.3.1.         Market Size & Forecast

8.3.3.1.1.             By Value

8.3.3.2.         Market Share & Forecast

8.3.3.2.1.             By Drug Type

8.3.3.2.2.             By Therapeutic Area

8.3.3.2.3.             By Route of Administration

8.3.3.2.4.             By Distribution Channel

8.3.4.     Italy Rare Disease Therapeutics Market Outlook

8.3.4.1.         Market Size & Forecast

8.3.4.1.1.             By Value

8.3.4.2.         Market Share & Forecast

8.3.4.2.1.             By Drug Type

8.3.4.2.2.             By Therapeutic Area

8.3.4.2.3.             By Route of Administration

8.3.4.2.4.             By Distribution Channel

8.3.5.     Spain Rare Disease Therapeutics Market Outlook

8.3.5.1.         Market Size & Forecast

8.3.5.1.1.             By Value

8.3.5.2.         Market Share & Forecast

8.3.5.2.1.             By Drug Type

8.3.5.2.2.             By Therapeutic Area

8.3.5.2.3.             By Route of Administration

8.3.5.2.4.             By Distribution Channel

9.    Asia-Pacific Rare Disease Therapeutics Market Outlook

9.1.  Market Size & Forecast          

9.1.1.     By Value

9.2.  Market Share & Forecast

9.2.1.     By Drug Type

9.2.2.     By Therapeutic Area

9.2.3.     By Route of Administration

9.2.4.     By Distribution Channel

9.2.5.     By Country (China; India; Japan; South Korea; Australia)

8.3  Asia-Pacific: Country Analysis

8.3.1      China Rare Disease Therapeutics Market Outlook

8.3.1.1  Market Size & Forecast

8.3.1.1.1      By Value

8.3.1.2  Market Share & Forecast

8.3.1.2.1      By Drug Type

8.3.1.2.2      By Therapeutic Area

8.3.1.2.3      By Route of Administration

8.3.1.2.4      By Distribution Channel

8.3.2      India Rare Disease Therapeutics Market Outlook

8.3.2.1  Market Size & Forecast

8.3.2.1.1      By Value

8.3.2.2  Market Share & Forecast

8.3.2.2.1      By Drug Type

8.3.2.2.2      By Therapeutic Area

8.3.2.2.3      By Route of Administration

8.3.2.2.4      By Distribution Channel

8.3.3      Japan Rare Disease Therapeutics Market Outlook

8.3.3.1  Market Size & Forecast

8.3.3.1.1      By Value

8.3.3.2  Market Share & Forecast

8.3.3.2.1      By Drug Type

8.3.3.2.2      By Therapeutic Area

8.3.3.2.3      By Route of Administration

8.3.3.2.4      By Distribution Channel

8.3.4      South Korea Rare Disease Therapeutics Market Outlook

8.3.4.1  Market Size & Forecast

8.3.4.1.1      By Value

8.3.4.2  Market Share & Forecast

8.3.4.2.1      By Drug Type

8.3.4.2.2      By Therapeutic Area

8.3.4.2.3      By Route of Administration

8.3.4.2.4      By Distribution Channel

8.3.5      Australia Rare Disease Therapeutics Market Outlook

8.3.5.1  Market Size & Forecast

8.3.5.1.1      By Value

8.3.5.2  Market Share & Forecast

8.3.5.2.1      By Drug Type

8.3.5.2.2      By Therapeutic Area

8.3.5.2.3      By Route of Administration

8.3.5.2.4      By Distribution Channel

10.  Middle East and Africa Rare Disease Therapeutics Market Outlook

10.1.              Market Size & Forecast

10.1.1.  By Value

10.2.              Market Share & Forecast

10.2.1.  By Drug Type

10.2.2.  By Therapeutic Area

10.2.3.  By Route of Administration

10.2.4.  By Distribution Channel

10.2.5.  By Country (UAE; Saudi Arabia; South Africa)

10.3.              MEA: Country Analysis

10.3.1.  South Africa Rare Disease Therapeutics Market Outlook

10.3.1.1.      Market Size & Forecast

10.3.1.1.1.           By Value

10.3.1.2.      Market Share & Forecast

10.3.1.2.1.           By Drug Type

10.3.1.2.2.           By Therapeutic Area

10.3.1.2.3.           By Route of Administration

10.3.1.2.4.           By Distribution Channel

10.3.2.  Saudi Arabia Rare Disease Therapeutics Market Outlook

10.3.2.1.      Market Size & Forecast

10.3.2.1.1.           By Value

10.3.2.2.      Market Share & Forecast

10.3.2.2.1.           By Drug Type

10.3.2.2.2.           By Therapeutic Area

10.3.2.2.3.           By Route of Administration

10.3.2.2.4.           By Distribution Channel

10.3.3.  UAE Rare Disease Therapeutics Market Outlook

10.3.3.1.      Market Size & Forecast

10.3.3.1.1.           By Value

10.3.3.2.      Market Share & Forecast

10.3.3.2.1.           By Drug Type

10.3.3.2.2.           By Therapeutic Area

10.3.3.2.3.           By Route of Administration

10.3.3.2.4.           By Distribution Channel

11.  South America Rare Disease Therapeutics Market Outlook

11.1.              Market Size & Forecast

11.1.1.  By Value

11.2.              Market Share & Forecast

11.2.1.  By Drug Type

11.2.2.  By Therapeutic Area

11.2.3.  By Route of Administration

11.2.4.  By Distribution Channel

11.2.5.  By Country (Brazil; Argentina; Colombia)

11.3.              South America: Country Analysis

11.3.1.  Brazil Rare Disease Therapeutics Market Outlook

11.3.1.1.      Market Size & Forecast

11.3.1.1.1.           By Value

11.3.1.2.      Market Share & Forecast

11.3.1.2.1.           By Drug Type

11.3.1.2.2.           By Therapeutic Area

11.3.1.2.3.           By Route of Administration

11.3.1.2.4.           By Distribution Channel

11.3.2.  Argentina Rare Disease Therapeutics Market Outlook

11.3.2.1.      Market Size & Forecast

11.3.2.1.1.           By Value

11.3.2.2.      Market Share & Forecast

11.3.2.2.1.           By Drug Type

11.3.2.2.2.           By Therapeutic Area

11.3.2.2.3.           By Route of Administration

11.3.2.2.4.           By Distribution Channel

11.3.3.  Colombia Rare Disease Therapeutics Market Outlook

11.3.3.1.      Market Size & Forecast

11.3.3.1.1.           By Value

11.3.3.2.      Market Share & Forecast

11.3.3.2.1.           By Drug Type

11.3.3.2.2.           By Therapeutic Area

11.3.3.2.3.           By Route of Administration

11.3.3.2.4.           By Distribution Channel

12.  Market Dynamics

12.1.              Drivers

12.2.              Challenges

13.  Market Trends & Developments

14.  Competitive Landscape

14.1.              Competition Outlook

14.2.              Global Players Profiled (Leading Companies)

14.2.1.  Novartis AG

14.2.2.  Pfizer Inc.

14.2.3.  F. Hoffmann-La Roche Ltd.

14.2.4.  AbbVie Inc.

14.2.5.  Novo Nordisk A/S

14.2.6.  Sanofi SA

14.2.7.  Bayer AG

14.2.8.  Amgen, Inc.

14.2.9.  Allergan plc.

14.2.10.                Eisai Co. Ltd.

14.2.11.                Bristol-Myers Squibb Company

14.2.12.                Merck & Co., Inc.

14.2.13.                Vertex Pharmaceuticals Inc.

14.2.14.                BioMarin Pharmaceutical Inc.

14.2.15.                Teva Pharmaceutical Industries Ltd.

15.  Strategic Recommendations

16.  About Us & Disclaimer