Rare Disease Treatment Market Size, Industry Analysis Report, Regional Outlook, Application Potential, Price Trends, Competitive Market Share & Forecast, 2020 - 2026

Report ID: GMI3873

Summary Methodology

Rare Disease Treatment Market size is projected to experience significant growth from 2019 to 2025.

Increasing prevalence of rare diseases across the world will stimulate rare disease treatment market growth in the coming years. According to the National Institute of Health, approximately 30 million Americans (10% of the total population) suffer from one of the 7,000 rare diseases. Also, 50 percent of patients affected by rare disease are children and only 5 percent of rare diseases have treatment. Additionally, rising investment in rare disease treatment sector and introduction of new drugs will boost the market growth in the forthcoming years. For instance, more than 560 drugs are in development for the treatment of rare disease. Therapies in development include treatment for amyotrophic lateral sclerosis, multiple myeloma, cystic fibrosis and enzyme deficiency disorders.

Rising government initiatives for spreading awareness regarding rare diseases along with presence of suitable healthcare infrastructure in developed and developing economies will drive the industry growth. For instance, the Orphan Drug Act 1983 was introduced by the U.S. to facilitate the development of orphan drugs for the treatment of rare diseases such as Amyotrophic Lateral Sclerosis (ALS), cystic fibrosis, muscular dystrophy, etc. Orphan Drug Act and two other laws The Best Pharmaceutical for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) were introduced to encourage the development of medicines for pediatric rare diseases. Additionally, favorable reimbursement policies and regulations for approval of drugs will escalate the growth of rare disease treatment market in the coming years.

However, lack of trained personals and challenges faced by pharmaceutical companies during the time of drug development are major limiting factors for the market growth.

Genetic diseases segment is estimated to dominate the therapeutic category in the foreseeable period. The growth of segment will be attributed to the rising incidence of rare genetic disorders in children. Almost 80 percent of rare diseases are of genetic origin. With increasing prevalence of genetic disorders such as hemophilia, phenylketonuria, cystic fibrosis, autosomal dominant polycystic kidney disease, etc. the demand for treatment will increase, thereby fueling the market growth.

Biologics drug category will hold a significant share over the forecast years. A biologic product or drug is produced from living organism or contains a living organism. These are derived from human, animal or microorganisms. Biologics includes gene therapy, recombinant proteins, and autologous transplants. These are commonly used for the treatment of rare cancers and are becoming increasingly important for other rare disorders. The biological product has the potential to correct the disorder permanently by employing gene therapy. Hence, the above-mentioned advantages will play a significant role in market growth.

Injectables is estimated to dominate the route of administration segment as the majority of medications are dispensed in injectable form. Many drugs including biologics tend to degrade via oral route and are dispensed in injectable form. Also, the bioavailability of an intravenously administered drug is 100%. Thus, for quick action intravenous route is preferable. Hence, above mentioned factors will play significant role in augmenting the business growth.

Specialty pharmacies will foresee significant growth over the forecast period. The growth is primarily due to the availability of orphan drugs in the specialty pharmacies. These are the primary source of most therapies for rare conditions. With the growing prevalence of the rare disease, the role of specialty pharmacies for dispensing the therapies will increase, fueling the market growth in the near future.

U.S. rare disease treatment market will foresee significant growth due to the presence of strong healthcare infrastructure and suitable reimbursement policies. The initiatives adopted by the U.S. FDA for spreading awareness regarding rare diseases along with the approval of new drugs will further escalate the market growth. Additionally, presence of key players and increasing need for drugs owing to rising rare diseases will further boost the business growth in the foreseeable future.

India rare disease treatment market is expected to show robust growth in the coming years owing to a developed healthcare system. The Indian government is undertaking several initiatives and programs to accelerate the diagnosis and treatment options for patients with a rare disease. The initiatives are also encouraging companies to invest in the research and development of orphan drugs in India.

Some of the major key players involved in the global rare disease treatment market are F. Hoffmann-La Roche Ltd, Sanofi, AbbVie Inc., Novartis AG, among others. Companies are investing in research and development for the introduction of new therapies. For instance, in June 2016, the U.S. FDA approved AbbVie’s therapy, Ibrutinib for the treatment of chronic graft-versus-host-disease.  

Rare Disease Treatment Market by Therapeutic Area, 2014-2025 (USD Million)

  • Neurologic diseases
  • Genetic diseases
  • Metabolic diseases
  • Infectious diseases
  • Inflammatory diseases
  • Pulmonary diseases
  • Musculoskeletal diseases
  • Dermatological disorders
  • Endocrine diseases
  • Malignant diseases
  • Blood disorders

Rare Disease Treatment Market by Drug Category, 2014-2025 (USD Million)

  • Biologics
  • Organic compounds

Rare Disease Treatment Market by Route of Administration, 2014-2025 (USD Million)

  • Oral
  • Injectable

Rare Disease Treatment Market by Distribution Channel, 2014-2025 (USD Million)

  • Hospitals
  • Retail pharmacies
  • Specialty pharmacies
  • Others

The above information is provided for the following regions and countries:

  • North America
    • U.S.
    • Canada
  • Europe
    • Germany
    • UK
    • France
    • Italy
    • Spain
  • Asia Pacific
    • Japan
    • China
    • India
  • Latin America
    • Mexico
    • Brazil
  • Middle East and Africa
    • South Africa
    • Saudi Arabia

Company Profiles:

  • F. Hoffmann-La Roche Ltd
  • Sanofi
  • AbbVie Inc.
  • Novartis AG
  • Bayer AG
  • Pfizer Inc
  • Novo Nordisk A/S
  • Bristol-Myers Squibb Company
  • Teva Pharmaceutical Industries Ltd
  • Baxter
  • GlaxoSmithKline plc
  • Boehringer Ingelheim International GmbH
  • Vertex Pharmaceuticals Incorporated
  • AstraZeneca
  • Janssen Global Services, LLC
  • Alexion
  • Biogen International
  • Orchard Therapeutics plc.
  • Shire

What Information does this report contain?

Historical data coverage: 2015 to 2019; Growth Projections: 2020 to 2026.
Expert analysis: industry, governing, innovation and technological trends; factors impacting development; drawbacks, SWOT.
6-7 year performance forecasts: major segments covering applications, top products and geographies.
Competitive landscape reporting: market leaders and important players, competencies and capacities of these companies in terms of production as well as sustainability and prospects.

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