Rare Disease Therapeutics Global Market Report 2023: Increasing Number of Patients with Rare Diseases Bolsters Growth
Dublin, April 12, 2023 (GLOBE NEWSWIRE) -- The "Rare Disease Therapeutics Market- Global Industry Size, Share, Trends, Competition, Opportunity and Forecast, 2018-2028 Segmented By Therapeutic Area, By Route of Administration, By Drug Type, By Distribution Channel, By Region and Competition" report has been added to ResearchAndMarkets.com's offering.
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, approximately 791 prospective orphan drug candidates were 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 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). 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 pediatric population suffering from rare ailments. Additionally, due to the surge in incidences of rare diseases, several 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 as 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.
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 on 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 development across different parts of the globe. In the coming years, this number is further expected to increase, thereby opening new prospects for market growth.
Report Scope:
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:
Oral
Injectable
Others
Rare Disease Therapeutics Market, By Drug Type:
Biologics
Biosimilars
Small Molecule
Rare Disease Therapeutics Market, By Distribution Channel:
Hospital Pharmacies
Specialty Pharmacies
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
Key Topics Covered:
1. Product Overview
2. Research Methodology
3. Executive Summary
4. Clinical Trial Analysis
5. Voice of Customer
6. Global Rare Disease Therapeutics Market Outlook
7. North America Rare Disease Therapeutics Market Outlook
8. Europe Rare Disease Therapeutics Market Outlook
9. Asia-Pacific Rare Disease Therapeutics Market Outlook
10. South America Rare Disease Therapeutics Market Outlook
11. Middle East and Africa Rare Disease Therapeutics Market Outlook
12. Market Dynamics
13. Market Trends & Developments
14. Competitive Landscape
15. Strategic Recommendations
A selection of companies mentioned in this report includes
Novartis AG
Pfizer Inc.
F Hoffmann-La Roche Ltd.
AbbVie Inc
Novo Nordisk A/S
Sanofi SA
Bayer AG
Amgen Inc
Eisai Co Ltd
Bristol-Myers Squibb Co
For more information about this report visit https://www.researchandmarkets.com/r/cbmt0g
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