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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 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:
Market Breakup by Route of Administration:
Market Breakup by Phase Type:
Market Breakup by Patient Type
Market Breakup by Drug Type
Market Breakup by Distribution Channel
Market Breakup by Region
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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.
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.
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.
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.
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.
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:
REPORT FEATURES | DETAILS |
Base Year | 2023 |
Historical Period | 2017-2023 |
Forecast Period | 2024-2032 |
Scope of the Report |
Historical and Forecast Trends, Industry Drivers and Constraints, Historical and Forecast Market Analysis by Segment:
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Breakup by Therapeutic Area |
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Breakup by Route of Administration |
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Breakup by Phase Type |
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Breakup by Patient Type |
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Breakup by Drug Type |
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Breakup by Distribution Channel |
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Breakup by Region |
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Market Dynamics |
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Supplier Landscape |
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Companies Covered |
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*At Expert Market Research, we strive to always give you current and accurate information. The numbers depicted in the description are indicative and may differ from the actual numbers in the final EMR report.
1 Preface
1.1 Objectives of the Study
1.2 Key Assumptions
1.3 Report Coverage – Key Segmentation
2 Research Methodology
3 Executive Summary
4 Rare Diseases Overview
4.1 Guidelines and Stages
4.2 Pathophysiology
4.3 Screening and Diagnosis
4.4 Treatment Pathway
5 Patient Profile
5.1 Patient Profile Overview
5.2 Patient Psychology and Emotional Impact Factors
5.3 Risk Assessment and Treatment Success Rate
6 Current Scenario Evaluation and Regulatory Framework
6.1 Emerging Therapies and Clinical Trials
6.2 Patent Landscape
6.2.1 Patent Overview
6.2.1.1 Patent Status and Expiry
6.2.1.2 Timelines from Drug Development to Commercial Launch
6.2.1.3 New Drug Application
6.2.1.3.1 Documentation and Approval Process
6.3 Cost of Treatment
6.4 Regulatory Framework
6.4.1 Regulatory Overview
6.4.1.1 US FDA
6.4.1.2 EU EMA
6.4.1.3 INDIA CDSCO
6.4.1.4 JAPAN PMDA
6.4.1.5 Others
7 Challenges and Unmet Needs
7.1 Treatment Pathway Challenges
7.2 Compliance and Drop-Out Analysis
7.3 Awareness and Prevention Gaps
8 Global Rare Diseases Treatment Market
8.1 Global Rare Diseases Treatment Market Overview
8.2 Global Rare Diseases Treatment Market Analysis
8.2.1 Market Overview
8.2.1.1 Global Rare Diseases Treatment Market Historical Value (2017-2023)
8.2.1.2 Global Rare Diseases Treatment Market Forecast Value (2024-2032)
8.2.2 Global Rare Diseases Treatment Market by Therapeutic Area
8.2.2.1 Market Overview
8.2.2.1.1 Oncological Disorders
8.2.2.1.2 Neurological Disorders
8.2.2.1.3 Cardiovascular Disorders
8.2.2.1.4 Musculoskeletal Disorders
8.2.2.1.5 Hematological Disorders
8.2.2.1.6 Infectious Disorders
8.2.2.1.7 Metabolic Disorders
8.2.2.1.8 Endocrine Disorders
8.2.2.1.9 Others
8.2.3 Global Rare Diseases Treatment Market by Route of Administration
8.2.3.1 Market Overview
8.2.3.1.1 Oral
8.2.3.1.2 Injectables
8.2.3.1.3 Others
8.2.4 Global Rare Diseases Treatment Market by Phase Type
8.2.4.1 Market Overview
8.2.4.1.1 Preclinical Phase
8.2.4.1.2 Phase I
8.2.4.1.3 Phase II
8.2.4.1.4 Phase III
8.2.4.1.5 Phase IV
8.2.5 Global Rare Diseases Treatment Market by Patient Type
8.2.5.1 Market Overview
8.2.5.1.1 Neonates
8.2.5.1.2 Pediatrics
8.2.5.1.3 Adults
8.2.6 Global Rare Diseases Treatment Market by Drug Type
8.2.6.1 Market Overview
8.2.6.1.1 Biologics
8.2.6.1.2 Non-Biologics
8.2.6.1.3 Others
8.2.7 Global Rare Diseases Treatment Market by Distribution Channel
8.2.7.1 Market Overview
8.2.7.1.1 Hospital Pharmacies
8.2.7.1.2 Retail Pharmacies
8.2.7.1.3 Online Pharmacies
8.2.8 Global Rare Diseases Treatment Market by Region
8.2.8.1 Market Overview
8.2.8.1.1 North America
8.2.8.1.2 Europe
8.2.8.1.3 Asia Pacific
8.2.8.1.4 Middle East and Africa
8.2.8.1.5 Latin America
9 North America Rare Disease Treatment Market
9.1 Market Share by Country
9.2 United States of America
9.2.1 Historical Trend (2017-2023)
9.2.2 Forecast Trend (2024-2032)
9.3 Canada
9.3.1 Historical Trend (2017-2023)
9.3.2 Forecast Trend (2024-2032)
10 Europe Rare Disease Treatment Market
10.1 Market Share by Country
10.2 United Kingdom
10.2.1 Historical Trend (2017-2023)
10.2.2 Forecast Trend (2024-2032)
10.3 Germany
10.3.1 Historical Trend (2017-2023)
10.3.2 Forecast Trend (2024-2032)
10.4 France
10.4.1 Historical Trend (2017-2023)
10.4.2 Forecast Trend (2024-2032)
10.5 Italy
10.5.1 Historical Trend (2017-2023)
10.5.2 Forecast Trend (2024-2032)
10.6 Others
11 Asia Pacific Rare Disease Treatment Market
11.1 Market Share by Country
11.2 China
11.2.1 Historical Trend (2017-2023)
11.2.2 Forecast Trend (2024-2032)
11.3 Japan
11.3.1 Historical Trend (2017-2023)
11.3.2 Forecast Trend (2024-2032)
11.4 India
11.4.1 Historical Trend (2017-2023)
11.4.2 Forecast Trend (2024-2032)
11.5 ASEAN
11.5.1 Historical Trend (2017-2023)
11.5.2 Forecast Trend (2024-2032)
11.6 Australia
11.6.1 Historical Trend (2017-2023)
11.6.2 Forecast Trend (2024-2032)
11.7 Others
12 Latin America Rare Disease Treatment Market
12.1 Market Share by Country
12.2 Brazil
12.2.1 Historical Trend (2017-2023)
12.2.2 Forecast Trend (2024-2032)
12.3 Argentina
12.3.1 Historical Trend (2017-2023)
12.3.2 Forecast Trend (2024-2032)
12.4 Mexico
12.4.1 Historical Trend (2017-2023)
12.4.2 Forecast Trend (2024-2032)
12.5 Others
13 Middle East and Africa Rare Disease Treatment Market
13.1 Market Share by Country
13.2 Saudi Arabia
13.2.1 Historical Trend (2017-2023)
13.2.2 Forecast Trend (2024-2032)
13.3 United Arab Emirates
13.3.1 Historical Trend (2017-2023)
13.3.2 Forecast Trend (2024-2032)
13.4 Nigeria
13.4.1 Historical Trend (2017-2023)
13.4.2 Forecast Trend (2024-2032)
13.5 South Africa
13.5.1 Historical Trend (2017-2023)
13.5.2 Forecast Trend (2024-2032)
13.6 Others
14 Global Rare Diseases Treatment Market Dynamics
14.1 Market Drivers and Constraints
14.2 SWOT Analysis
14.3 Porter’s Five Forces Model
14.4 Key Demand Indicators
14.5 Key Price Indicators
14.6 Industry Events, Initiatives, and Trends
14.7 Value Chain Analysis
15 Supplier Landscape
15.1 F. Hoffmann-La Roche Ltd
15.1.1 Company Overview
15.1.2 Product Portfolio
15.1.3 Demographic Reach and Achievements
15.1.4 Mergers and Acquisitions
15.1.5 Certifications
15.2 Pfizer, Inc.
15.2.1 Company Overview
15.2.2 Product Portfolio
15.2.3 Demographic Reach and Achievements
15.2.4 Mergers and Acquisitions
15.2.5 Certifications
15.3 PTC Therapeutics
15.3.1 Company Overview
15.3.2 Product Portfolio
15.3.3 Demographic Reach and Achievements
15.3.4 Mergers and Acquisitions
15.3.5 Certifications
15.4 AstraZeneca
15.4.1 Company Overview
15.4.2 Product Portfolio
15.4.3 Demographic Reach and Achievements
15.4.4 Mergers and Acquisitions
15.4.5 Certifications
15.5 Novartis AG
15.5.1 Company Overview
15.5.2 Product Portfolio
15.5.3 Demographic Reach and Achievements
15.5.4 Mergers and Acquisitions
15.5.5 Certifications
15.6 Bristol-Myers Squibb Company
15.6.1 Company Overview
15.6.2 Product Portfolio
15.6.3 Demographic Reach and Achievements
15.6.4 Mergers and Acquisitions
15.6.5 Certifications
15.7 Takeda Pharmaceutical Company Limited
15.7.1 Company Overview
15.7.2 Product Portfolio
15.7.3 Demographic Reach and Achievements
15.7.4 Mergers and Acquisitions
15.7.5 Certifications
15.8 Bayer AG
15.8.1 Company Overview
15.8.2 Product Portfolio
15.8.3 Demographic Reach and Achievements
15.8.4 Mergers and Acquisitions
15.8.5 Certifications
15.9 AbbVie Inc.
15.9.1 Company Overview
15.9.2 Product Portfolio
15.9.3 Demographic Reach and Achievements
15.9.4 Mergers and Acquisitions
15.9.5 Certifications
15.10 Merck & Co., Inc.
15.10.1 Company Overview
15.10.2 Product Portfolio
15.10.3 Demographic Reach and Achievements
15.10.4 Mergers and Acquisitions
15.10.5 Certifications
15.11 Teva Pharmaceutical Industries Ltd.
15.11.1 Company Overview
15.11.2 Product Portfolio
15.11.3 Demographic Reach and Achievements
15.11.4 Mergers and Acquisitions
15.11.5 Certifications
16 Global Rare Diseases Treatment Market- Drug Distribution Model (Additional Insight)
16.1 Overview
16.2 Potential Distributors
16.3 Key Parameters for Distribution Partner Assessment
17 Payment Methods (Additional Insight)
17.1 Government Funded
17.2 Private Insurance
17.3 Out-of-Pocket
*Additional insights provided are customizable as per client requirements.
The market is anticipated to grow at a CAGR of 12.7% during the forecast period of 2024-2032.
The market is primarily driven by the increasing disease burden, technological advancements, increased investments, and novel product launches and approvals.
North America is anticipated to hold a maximum share in the market during the forecast period.
Based on the therapeutic area, the market is categorised into oncological disorders, neurological disorders, cardiovascular disorders, musculoskeletal disorders, haematological disorders, infectious disorders, metabolic disorders, endocrine disorders, and others.
Based on the route of administration, the market is segmented into oral, injectables and others.
Based on the drug type, the market is bifurcated into biologics, non-biologics, and others.
Based on the distribution channel, the market is divided into hospital pharmacies, retail pharmacies, online pharmacies, and others.
The different regions in the market are North America, Europe, Asia Pacific, Latin America and the Middle East and Africa.
The key companies involved in the market are 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., among others.
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