Global rare disease therapeutics market is projected to undergo a vigorous expansion in the coming years. A robust product pipeline and upcoming launches are anticipated to drive market growth. In 2021, there were approximately 791 prospective orphan drug candidates undergoing clinical trials for the treatment of rare diseases, according to Pharmaceutical Research and Manufacturers of America. Clinical orphan pharmaceuticals were used to treat 192 genetic disorders, 56 neurological disorders, 54 blood disorders, 51 autoimmune diseases, and 36 infectious diseases. Also, 168 were for rare malignancies and 120 were for rare blood cancers.

Additionally, the Center for Drug Evaluation and Research (CDER) approved about 26 new orphan medications in 2021 for the treatment of rare disorders. Amondys 4, Exkivity, Lumakras, Cytalux, Bylvay, Scemblix, Welireg, Empaveli, fexinidazole, Besremi, Evkeeza, and Zynlonta were a few of the products that were approved. Therefore, it is projected that increased approval and the introduction of novel orphan medications will spur the industry growth.

Increasing Number of Patients with Rare Diseases

The market for treating rare diseases will expand as the number of rare diseases rises. There are about 7,000 rare ailments that have been diagnosed, and about 70% of them have no treatment options, according to a 2020 Journal of Rare Disorders article. Therefore, continued market expansion prospects will arise from research into unmet rare disease treatments in the future. Furthermore, the Global Genes Project estimates that 300 million people globally suffer from uncommon diseases. Due to the increased occurrence of rare conditions, it will be necessary to use specialized medications, which will accelerate the use of therapies for rare diseases.

Approximately 6% to 8% of people in the European Union are thought to be affected by a rare disorder, according to estimates from the European Organization for Rare Diseases (EURORDIS). In order to address the demand for specific therapies for the treatment of rare conditions such as fatal familial insomnia and rare cardiovascular ailments, among others, various research laboratories are concentrating on research and development. Furthermore, between 50% and 60% of rare illnesses start in childhood, and several of these conditions are deadly. Thus, it is anticipated that in the upcoming years, there will be a significant increase in the need for certain therapeutic medications due to the expanding paediatric population suffering from rare ailments. Additionally, due to the surge in incidences of rare diseases, a number of biopharmaceutical companies are funding clinical studies for brand-new medications intended to treat rare diseases.

Initiatives Taken by Governments & Various Market Players

Government rules that encourage product development, such the Orphan Drug Act, which grants the orphan drug classification to potential drug candidates created by pharmaceutical companies, are expected to assist the market's growth. This program advances treatment for orphan diseases through research and development.

For patients with rare diseases afflicted by the COVID-19 pandemic, the National Organization for Rare Disorders (NORD) introduced the "COVID-19 critical relief" program in April 2020. In accordance with this initiative, the NORD offered orphan disease patients and their guardians financial support of up to USD 1,000 annually to meet their non-medical requirements. Therefore, it is projected that the availability of such programs will increase the uptake of rare disease treatment over the forecast period.

Additionally, businesses have launched projects including partnerships and collaborations for the creation, production, and marketing of new treatments in the global market. For example, in August 2020 Sarepta Therapeutics, Inc. and the University of Florida collaborated for the development of innovative genetic therapies for the treatment of individuals with a variety of orphan diseases, such as Duchenne muscular dystrophy (DMD).

Growing Research and Development Strategies

One of the key market driving factors is the rising R&D expenditures made by well-known players for the creation of fresh product offerings for orphan drugs. A significant number of major clinical-stage biopharmaceutical companies and existing market players currently have orphan drug pipeline candidates at various phases of clinical trials due to the increase in public awareness and knowledge of rare diseases. Because rare disorders are more likely to have big pharmacological discoveries that lead to the development of blockbuster drugs than more common diseases, there is an increasing interest in therapies for rare disorders. According to clinicaltrials.gov, there are around 2,615 studies related to rare disease therapeutics which are in different phases of developments across different parts of the globe. In the coming years, this number is further expected to increase, thereby opening new prospects for the market growth.


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Market Segmentation

Global rare disease therapeutics market is segmented by therapeutic area, route of administration, drug type, distribution channel, region, and company. Based on therapeutic area, the market can be segmented into cancer, cardiovascular disorders, musculoskeletal disorders, hematologic disorders, infectious diseases, metabolic disorders, endocrine disorders, and others. On the basis of route of administration, the market can be divided into oral, injectable, and others. Based on drug type, the market can be categorized into biologics, biosimilars, and small molecule. On the basis of distribution channel, the market can be fragmented into hospital pharmacies, specialty pharmacies, and online pharmacies. 

Company Profiles

Some of the major competitors operating in the global rare disease therapeutics market include Novartis AG, Pfizer Inc., F Hoffmann-La Roche Ltd., AbbVie Inc, Novo Nordisk A/S, Sanofi SA, Bayer AG, Amgen Inc, Eisai Co Ltd, and Bristol-Myers Squibb Co.

Attribute

Details

Base Year

2022

Historical Years

2018 – 2021

Estimated Year

2023

Forecast Period

2024 – 2028

Quantitative Units

Revenue in USD Million and CAGR for 2018-2022 and 2023-2028

Report Coverage

Revenue forecast, company share, competitive landscape, growth factors, and trends

Segments Covered

·         Therapeutic Area

·         Route of Administration

·         Drug Type

·         Distribution Channel

Regional Scope

North America; Europe; Asia Pacific; Middle East & Africa; South America

Country Scope

United States; Canada; Mexico; France; Germany; United Kingdom; Italy; Spain; China; India; Japan; South Korea; Australia; South Africa; Saudi Arabia; UAE; Brazil; Argentina; Colombia

Key Companies Profiled

Novartis AG, Pfizer Inc., F Hoffmann-La Roche Ltd., AbbVie Inc, Novo Nordisk A/S, Sanofi SA, Bayer AG, Amgen Inc, Eisai Co Ltd, and Bristol-Myers Squibb Co. 

Customization Scope

10% free report customization with purchase. Addition or alteration to country, regional & segment scope.

Pricing and Purchase Options

Avail customized purchase options to meet your exact research needs. Explore purchase options

Delivery Format

PDF and Excel through Email (We can also provide the editable version of the report in PPT/Word format on special request)

 

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:

  • Rare Disease Therapeutics Market, By Therapeutic Area:
    • Cancer
    • Cardiovascular Disorders
    • Musculoskeletal Disorders
    • Hematologic Disorders
    • Infectious Diseases
    • Metabolic Disorders
    • Endocrine Disorders
    • Others
  • Rare Disease Therapeutics Market, By Route of Administration:

o   Oral

o   Injectable

o   Others

  • Rare Disease Therapeutics Market, By Drug Type:
    • Biologics
    • Biosimilars
    • Small Molecule
  • Rare Disease Therapeutics Market, By Distribution Channel:

o   Hospital Pharmacies

o   Specialty Pharmacies

o   Online Pharmacies

  • Rare Disease Therapeutics Market, By Region:
    • North America
      • United States
      • Canada
      • Mexico
    • Europe
      • Germany
      • France
      • United Kingdom
      • Italy
      • Spain
    • Asia Pacific
      • China
      • India
      • Japan
      • South Korea
      • Australia
    • South America
      • Brazil
      • Argentina
      • Colombia
    • Middle East & Africa
      • Saudi Arabia
      • UAE
      • South Africa

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

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1.    Product Overview

1.1.  Market Definition

1.2.  Scope of the Market

1.2.1.     Markets Covered

1.2.2.     Years Considered for Study

1.2.3.     Key Market Segmentations

2.    Research Methodology

2.1.  Objective of the Study

2.2.  Baseline Methodology

2.3.  Key Industry Partners

2.4.  Major Association and Secondary Sources

2.5.  Forecasting Methodology

2.6.  Data Triangulation & Validation

2.7.  Assumptions and Limitations

3.    Executive Summary

3.1.  Overview of the Market

3.2.  Overview of Key Market Segmentations

3.3.  Overview of Key Market Players

3.4.  Overview of Key Regions/Countries

3.5.  Overview of Market Drivers, Challenges, Trends

4.    Clinical Trial Analysis

4.1.  Ongoing Clinical Trials

4.2.  Completed Clinical Trials

4.3.  Terminated Clinical Trials

4.4.  Breakdown of Pipeline, By Development Phase

4.5.  Breakdown of Pipeline, By Status

4.6.  Breakdown of Pipeline, By Therapeutic Area

4.7.  Breakdown of Pipeline, By Region

4.8.  Clinical Trials Heat Map

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 Therapeutic Area (Cancer, Cardiovascular Disorders, Musculoskeletal Disorders, Hematologic Disorders, Infectious Diseases, Metabolic Disorders, Endocrine Disorders, Others)

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

6.2.3.     By Drug Type (Biologics, Biosimilars, Small Molecule)

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

6.2.5.     By Region

6.2.6.     By Company (2022)

6.3.  Market Map

6.3.1.     By Therapeutic Area

6.3.2.     By Route of Administration

6.3.3.     By Drug Type

6.3.4.     By Distribution Channel

6.3.5.     By Region

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 Therapeutic Area

7.2.2.     By Route of Administration

7.2.3.     By Drug Type

7.2.4.     By Distribution Channel

7.2.5.     By Country

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 Therapeutic Area

7.3.1.2.2.             By Route of Administration

7.3.1.2.3.             By Drug Type

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 Therapeutic Area

7.3.2.2.2.             By Route of Administration

7.3.2.2.3.             By Drug Type

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 Therapeutic Area

7.3.3.2.2.             By Route of Administration

7.3.3.2.3.             By Drug Type

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 Therapeutic Area

8.2.2.     By Route of Administration

8.2.3.     By Drug Type

8.2.4.     By Distribution Channel

8.2.5.     By Country

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 Therapeutic Area

8.3.1.2.2.             By Route of Administration

8.3.1.2.3.             By Drug Type

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 Therapeutic Area

8.3.2.2.2.             By Route of Administration

8.3.2.2.3.             By Drug Type

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 Therapeutic Area

8.3.3.2.2.             By Route of Administration

8.3.3.2.3.             By Drug Type

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 Therapeutic Area

8.3.4.2.2.             By Route of Administration

8.3.4.2.3.             By Drug Type

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 Therapeutic Area

8.3.5.2.2.             By Route of Administration

8.3.5.2.3.             By Drug Type

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 Therapeutic Area

9.2.2.     By Route of Administration

9.2.3.     By Drug Type

9.2.4.     By Distribution Channel

9.2.5.     By Country

9.3.  Asia-Pacific: Country Analysis

9.3.1.     China Rare Disease Therapeutics Market Outlook

9.3.1.1.         Market Size & Forecast

9.3.1.1.1.             By Value

9.3.1.2.         Market Share & Forecast

9.3.1.2.1.             By Therapeutic Area

9.3.1.2.2.             By Route of Administration

9.3.1.2.3.             By Drug Type

9.3.1.2.4.             By Distribution Channel

9.3.2.     India Rare Disease Therapeutics Market Outlook

9.3.2.1.         Market Size & Forecast

9.3.2.1.1.             By Value

9.3.2.2.         Market Share & Forecast

9.3.2.2.1.             By Therapeutic Area

9.3.2.2.2.             By Route of Administration

9.3.2.2.3.             By Drug Type

9.3.2.2.4.             By Distribution Channel

9.3.3.     Japan Rare Disease Therapeutics Market Outlook

9.3.3.1.         Market Size & Forecast

9.3.3.1.1.             By Value

9.3.3.2.         Market Share & Forecast

9.3.3.2.1.             By Therapeutic Area

9.3.3.2.2.             By Route of Administration

9.3.3.2.3.             By Drug Type

9.3.3.2.4.             By Distribution Channel

9.3.4.     South Korea Rare Disease Therapeutics Market Outlook

9.3.4.1.         Market Size & Forecast

9.3.4.1.1.             By Value

9.3.4.2.         Market Share & Forecast

9.3.4.2.1.             By Therapeutic Area

9.3.4.2.2.             By Route of Administration

9.3.4.2.3.             By Drug Type

9.3.4.2.4.             By Distribution Channel

9.3.5.     Australia Rare Disease Therapeutics Market Outlook

9.3.5.1.         Market Size & Forecast

9.3.5.1.1.             By Value

9.3.5.2.         Market Share & Forecast

9.3.5.2.1.             By Therapeutic Area

9.3.5.2.2.             By Route of Administration

9.3.5.2.3.             By Drug Type

9.3.5.2.4.             By Distribution Channel

10.  South America Rare Disease Therapeutics Market Outlook

10.1.              Market Size & Forecast

10.1.1.  By Value

10.2.              Market Share & Forecast

10.2.1.  By Therapeutic Area

10.2.2.  By Route of Administration

10.2.3.  By Drug Type

10.2.4.  By Distribution Channel

10.2.5.  By Country

10.3.              South America: Country Analysis

10.3.1.  Brazil 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 Therapeutic Area

10.3.1.2.2.           By Route of Administration

10.3.1.2.3.           By Drug Type

10.3.1.2.4.           By Distribution Channel

10.3.2.  Argentina 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 Therapeutic Area

10.3.2.2.2.           By Route of Administration

10.3.2.2.3.           By Drug Type

10.3.2.2.4.           By Distribution Channel

10.3.3.  Colombia 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 Therapeutic Area

10.3.3.2.2.           By Route of Administration

10.3.3.2.3.           By Drug Type

10.3.3.2.4.           By Distribution Channel

11.  Middle East and Africa Rare Disease Therapeutics Market Outlook

11.1.              Market Size & Forecast

11.1.1.  By Value

11.2.              Market Share & Forecast

11.2.1.  By Therapeutic Area

11.2.2.  By Route of Administration

11.2.3.  By Drug Type

11.2.4.  By Distribution Channel

11.2.5.  By Country

11.3.              MEA: Country Analysis

11.3.1.  South Africa 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 Therapeutic Area

11.3.1.2.2.           By Route of Administration

11.3.1.2.3.           By Drug Type

11.3.1.2.4.           By Distribution Channel

11.3.2.  Saudi Arabia 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 Therapeutic Area

11.3.2.2.2.           By Route of Administration

11.3.2.2.3.           By Drug Type

11.3.2.2.4.           By Distribution Channel

11.3.3.  UAE 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 Therapeutic Area

11.3.3.2.2.           By Route of Administration

11.3.3.2.3.           By Drug Type

11.3.3.2.4.           By Distribution Channel

12.  Market Dynamics

12.1.              Drivers

12.2.              Challenges

13.