Global Rare Disease Therapeutics Market Report 2023: Players Include AbbVie, Novo Nordisk, Sanofi, Bayer, Amgen and Eisai - ResearchAndMarkets.com

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The global rare disease therapeutics market is projected to undergo a vigorous expansion in the coming years.

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Wednesday, April 5, 2023
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Biotechnology, Pharmaceutical, Health, Orphan Drug Act of 1983, CDER, COVID-19, Therapy, Disease, NORD, Bone marrow, DSM-IV codes, Pharmacy, Loncastuximab tesirine, Infection, National Organization for Rare Disorders, USD, Competition, Market, Malignancy, Administration, EURORDIS, Rare, Patient, Laboratory, European Organization for Quality, European, Center for Drug Evaluation and Research, Route of administration, Knowledge, Pharmaceutical Research and Manufacturers of America, Global Genes, D, Pharmaceutical industry, Research, Mobocertinib
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EUROPEAN UNION
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Summary
Key Points: 
  • The global rare disease therapeutics market is projected to undergo a vigorous expansion in the coming years.
  • The market for treating rare diseases will expand as the number of rare diseases rises.
  • Therefore, continued market expansion prospects will arise from research into unmet rare disease treatments in the future.
  • 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.


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.

The 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.

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.

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. Through this initiative, the NORD offered orphan disease patients and their guardian's 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.

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.

Companies Mentioned

  • 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

Report Scope:

In this report, global rare disease therapeutics market has been segmented into the 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:

  • 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

For more information about this report visit https://www.researchandmarkets.com/r/ez2n7p

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