Global Rare Diseases Treatment Market Report and Forecast 2024-2032

Global Rare Diseases Treatment Market Outlook

The global rare diseases treatment market is expected to witness significant growth during the forecast period of 2024-2032. The increasing prevalence of rare diseases worldwide, coupled with the growing investment in research and development activities, is driving the growth of the market. The global rare diseases treatment market is likely to grow at a CAGR of 12.7 % during the forecast period.

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Rare Diseases Insights and Segmentation

Rare diseases refer to the set of diseases that have a lower prevalence rate as compared to other prevalent diseases among the general population. At present, according to NPRD Policy 2021, nearly 6000-8000 rare diseases exist globally with new disorders being added to the list every year.

Although rare diseases are rare, however when assessed collectively, they account for nearly 6-8% of the total population. Rare diseases can be acute or chronic; some are easily treatable, while the treatment for some rare diseases is yet to be discovered.

Each nation has its pre-defined standards for classifying any disease as rare or common. According to Orphan Act, a disease is set to be rare, when it is impacting less than 200,000 people in the United States. European Union declares any disease as rare if it is affecting less than 1 in 2000 people. Rare diseases are mainly prevalent in children, accounting for almost 50% of new cases in children, followed by children of age one to five and five to fifteen years. 

GARD, Genetic and Rare Diseases, is an international program developed to support and assist people suffering from rare diseases and their families. GARD provides easy, reliable, and detailed information for every rare disease, in English and Spanish.

There are several acts and regulations formed by the regulatory bodies of different countries to manage the disease burden, spread awareness, and ensure the effectiveness of therapeutic interventions. 

In the United States, the Rare Diseases Orphan Product Development Act is formulated specifically for managing rare diseases. The United Kingdom government also implemented The UK Strategy for Rare Diseases, to ensure that no patient with a rare disease is left behind. 

Organisations and concerned authorities across the world are working and exploring to find innovative ways to assist patients suffering from rare diseases.

According to the rare diseases treatment market research report, the market can be categorised into the following segments:

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Market Breakup by Therapeutic Area:

• Oncological Disorders

• Neurological Disorders

• Cardiovascular Disorders

• Musculoskeletal Disorders

• Hematological Disorders

• Infectious Disorders

• Metabolic Disorders

• Endocrine Disorders

• Others

Market Breakup by Route of Administration:

• Oral

• Injectables

• Others

Market Breakup by Phase Type:

• Preclinical Phase

• Phase I

• Phase II

• Phase III

• Phase IV

Market Breakup by Patient Type

• Neonates

• Pediatrics

• Adults

Market Breakup by Drug Type

• Biologics

• Non-Biologics

• Others

Market Breakup by Distribution Channel

• Hospital Pharmacies

• Retail Pharmacies

• Online Pharmacies

Market Breakup by Region

• North America
  • United States of America
  • Canada
• Europe
  • United Kingdom
  • Germany
  • France
  • Italy
  • Others
• Asia Pacific
  • China
  • Japan
  • India
  • ASEAN
  • Australia
  • Others
• Latin America
  • Brazil
  • Argentina
  • Mexico
  • Others
• Middle East and Africa
  • Saudi Arabia
  • United Arab Emirates
  • Nigeria
  • South Africa
  • Others

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Market Scenario

The market is primarily driven by the increasing prevalence and incidence of rare diseases. This is expected to grow further with the increasing population and other risk factors. According to National Center for Advancing Translational Sciences, in the United States, more than 30 million people are estimated to get diagnosed with rare diseases in 2023.

The market growth is also accelerated by favouring government initiatives and measures, such as the National Policy for Rare Disorders, Orphan Drug Act, and other emerging acts and policies. These measures assist in raising awareness, stimulating novel product development and advancements in the present therapeutic system.

The implementation of certain programs by regulatory bodies has also stimulated the development of the rare diseases treatment market. For example, National Organization for Rare Disorders (NORD) has also launched a COVID-19 critical relief program, for COVID-affected patients living with rare diseases. The program provides USD 1000 annually to orphan disease patients and their families or caretakers for managing their clinical and non-clinical requirements.

Moreover, with extensive research and innovation, there is a strong robust pipeline, with many therapeutic products in the development stage. The increasing number of mergers and acquisitions among the key market players is also expected to bolster the market growth. 

For example, Sarepta Therapeutics collaborated with the University of Florida for discovering novel therapeutic products for the treatment of rare diseases.

Market Segment Insights: Therapeutic Area and Region Wise

Oncological disorders hold a significant share in the therapeutic area segment owing to the greater prevalence of different types of cancer and the rising recurrence rate of cancer indications. Among the regions, North America is anticipated to account for a major share of the rare diseases treatment market due to the higher patient pool, advanced healthcare infrastructure, and favouring government initiatives.

Therapeutic Landscape

In the last few decades, with technological advancements and a better understanding of the disease mechanism, there has been the availability and discovery of several treatment modalities that have proved to be beneficial in disease management. The treatment and prevention of rare disorders depend on the disease and affected area and are done at multiple levels.

Drugs Available for Different Therapeutic Areas

The treatment for oncological disorders is primarily done with the help of the targeted drug treatment that specifically targets the cancerous cells. Monoclonal antibodies and angiotensin inhibitors are some examples of targeted drug therapies.

Several categories of drugs are used for the management of neurological disorders including anticonvulsants, tranquillizers, sedatives, analgesics, and psychotropic agents. The common drugs used for the treatment of cardiac disorders are ACE inhibitors, aldosterone inhibitors, angiotensin II receptor blockers, beta-blockers, calcium channel blockers, vasodilators, and cholesterol-lowering agents.

Non-steroidal anti-inflammatory drugs, prescription medications, and acetaminophen have been found effective for the treatment of musculoskeletal disorders. Haematological or blood disorders are generally treated using thrombolytics, anti-coagulants, and antiplatelet agents. Infectious disorders include a wide range of diseases and include antibiotics, antivirals, antifungals, and anti-parasitics. 

Metabolic disorders affect the metabolic system of the body and can be managed by statins, metformin, farnesoid X receptor agonists and PPAR agonists. The treatment of endocrine disorders is done through antithyroid drugs and with the help of radioactive iodine treatment.

There is no approved treatment regimen for the management of rare disorders, however, improved diagnostic tools and available standard therapies have provided therapeutic effects in managing the disease.

Clinical Trials Landscape

The introduction of artificial intelligence has led to the invention of novel therapies by major pharmaceutical companies. Atopic dermatitis is a rare disorder, its treatment is under Phase II clinical trials and was developed using artificial intelligence and machine learning technology.

Drug repurposing, another novel process is incorporated for identifying the other novel applications for the existing drug. Thalidomide is a safe and effective treatment for the inflammation of leprosy and multiple myeloma. Tocilizumab is another such drug, which was found for the treatment of rheumatoid arthritis and is presently used for the management of the rare form of lung disease.

Another drug, Relyvrio, is primarily used to treat patients with amyotrophic lateral sclerosis and is being approved by the US FDA (Food and Drug Administration) for the treatment of Lou Gehrig’s disease is a rare disease that affects the nerve cells controlling voluntary muscles.

Recently, United States FDA has also accepted the drug approval of Bluebird Bio’s gene therapy Zyntelgo for the management of beta thalassemia, a rare hematological disorder. Another gene therapy Skysona, of the same company has been approved for the treatment of a rare disorder, cerebral adrenoleukodystrophy.

Due to the scientific and technical advancements in addition to the favouring government regulations, major pharmaceutical companies are actively investing in the discovery of novel innovative treatments that are anticipated to boost the rare diseases treatment market growth during the forecast period.

Competitive Landscape

The report gives an in-depth analysis of the key players involved in the rare diseases treatment market, sponsors manufacturing the therapies, and putting them through trials to get FDA approvals. The companies included in the market are as follows:

• F. Hoffmann-La Roche Ltd

• Pfizer, Inc.

• PTC Therapeutics 

• AstraZeneca

• Novartis AG

• Bristol-Myers Squibb Company 

• Takeda Pharmaceutical Company Limited 

• Bayer AG

• AbbVie Inc.

• Merck & Co., Inc. 

• Teva Pharmaceutical Industries Ltd.

Key Highlights of the Report