Rare Disease Treatment Market - Historic Data (2019-2024), Global Trends 2025, Growth Forecasts 2037
Rare Disease Treatment Market in 2025 is evaluated at USD 241.31 billion. The global market size was estimated at over USD 224.6 billion in 2024 and is anticipated to grow at a CAGR of 9.3%, with projections indicating it will exceed USD 713.63 billion by 2037. North America is likely to secure a dominant industry presence, impelled by the presence of several prominent players and a wide number of clinical trials.
The rare disease treatment market is largely driven by advancements in biotechnology, increasing investment in orphan drug research, and greater government incentives for developing treatments for rare conditions. More than 400 million people globally suffer from a rare disease. 1 in every 10 people have a rare disease, and 1 in every 2 patients diagnosed with a rare disease is a child. Over 10,000 distinct types of rare diseases exist in the world, and 8 in every 10 cases of rare diseases are genetic by nature.
These diseases often lack effective treatments due to high costs and complex drug development processes. However, regulatory support such as the U.S. Orphan Drug Act has spurred pharmaceutical companies to explore more in the market. For instance, in October 2021, U.S. FDA awarded 11 new clinical trial research grants, valued at USD 25 million through the FDA’s Congressionally-funded Orphan Products Grants Program. Rare disease treatment market companies are increasing their focus on gene and cell therapies, which offer promising one-time treatments for certain rare diseases. Additionally, digital health tools and personalized medicine approaches are gaining momentum as they allow for more targeted and efficient treatment options.
Rare Disease Treatment Sector: Growth Drivers and Challenges
Growth Drivers
- Growing R&D activities for novel therapeutics drugs: It is a key driver in the rare disease treatment market as companies and research institutions focus on developing targeted and advanced solutions such as gene therapies and biologics. These innovative approaches aim to provide more effective treatments with fewer side effects, addressing high unmet needs within the rare disease segment. Enhanced funding, technological advancements, and supportive regulatory pathways are further accelerating R&D efforts, contributing to market growth.
- Government incentives & orphan drug policies: These play a crucial role in driving the rare disease treatment market. These policies, including tax credits, grants, extended market exclusivity, and expedited approval processes, encourage pharmaceutical companies to invest in the costly and complex development of rare disease treatments. By reducing financial, and regulatory barriers, governments worldwide make it more feasible for companies to bring innovative therapies to market, ultimately improving access to life-saving treatments for patients with rare conditions.
Challenges
- Ethical & regulatory issues in gene therapy: These therapies involve altering genetic material to treat or prevent diseases, raising ethical concerns about long-term effects, genetic modifications in future generations, and potential misuse of genetic editing technologies. Regulatory bodies also face challenges in creating frameworks that ensure safety while allowing for innovation. Lengthy approval processes and ethical scrutiny can delay or complicate the development and rare disease treatment market availability of gene therapies.
- Limited clinical data: Rare diseases inherently have smaller patient populations, making it difficult to gather large-scale clinical data or conduct extensive trials. This lack of robust data can hinder regulatory approvals, increase the perceived risk for both developers and investors and limit physicians’ confidence in prescribing new therapies. Consequently, the scarcity of clinical data can slow down the growth and adoption of novel therapies in the rare disease treatment market.
Rare Disease Treatment Market: Key Insights
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Base Year |
2024 |
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Forecast Year |
2025-2037 |
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CAGR |
9.3% |
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Base Year Market Size (2024) |
USD 224.6 billion |
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Forecast Year Market Size (2037) |
USD 713.63 billion |
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Regional Scope |
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Rare Disease Treatment Segmentation
Therapeutic Area (Cancer, Blood-related disorders, Central nervous system, Respiratory disorders)
Based on the therapeutic area, the cancer segment is expected to register a considerable share in 2037 in the rare disease treatment market. A wide number of cancers are classified as rare owing to their low incidence rates and specific genetic mutations. Significant research investments, targeted treatments, immunotherapies, and an urgent need for effective solutions in rare cancers drive growth in this segment. Additionally, regulatory incentives and patient advocacy have spurred pharmaceutical companies to prioritize rare cancer treatments, further reinforcing cancer’s leading position in the rare disease therapeutic area. For instance, in April 2024, the President of India launched CAR-T cell therapy, India’s first home-grown gene therapy, aiming to fight cancer.
Route of Administration (Oral, Injectable)
Based on the route of administration, the oral segment is expected to capture around 55.7% rare disease treatment market share by the end of 2037. The growth is majorly attributed to their ease of use, convenience, and patient-friendly profile, especially for chronic conditions that require long-term management. Patients and healthcare providers often favor oral medications over injectables as they are easier to self-administer and typically involve fewer hospital visits. Recent advancements in oral drug delivery technology have also enabled more effective treatment of complex rare diseases. For instance, in September 2024, the U.S. FDA announced the approval of Miplyffa (arimoclomol), an oral medication for Niemann-Pick disease, type C.
Our in-depth analysis of the global market includes the following segments:
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Drug Type |
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Patient |
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Therapeutic Area |
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Route of Administration |
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Customize this ReportRare Disease Treatment Industry - Regional Synopsis
North America Market Analysis
As per the National Organization for Rare Disorders (NORD), more than 10,000 rare diseases together affect over 30 million Americans. Federal agencies including the NIH and FDA assign funding for rare and orphan disease R&D to support clinical trials in the region. In September 2024, Travere Therapeutics, Inc., received U.S. FDA approval for FILSPARI which aims to reduce the declination rate in kidney functioning among adults suffering from primary IgAN who are at risk of disease progression. In addition, U.S. reimbursement policies such as Medicare coverage and other coverage by private insurance companies, impact the commercial viability of these treatments positively.
The U.S. rare disease treatment market is growing majorly due to the presence of several prominent players. Owing to their presence, the country is witnessing a wide number of clinical trials, followed by medication and therapy launches as a positive outcome for the trials. In July 2022, the U.S. FDA approved Pfizer Inc.’s crizotinib for adult and pediatric patients of 1 year and above, suffering from ALK-positive IMT. These approvals from the regulatory bodies are further projected to lead to several product launches, further enhancing the rare disease treatment market growth in the upcoming years.
According to an article posted by the Government of Canada in March 2023, 1 out of 12 people in Canada suffer from a rare disease. It further states that the Minister of Health declared supportive measures of the first-ever National Strategy for Drugs for Rare Diseases. The government offered an investment of up to USD 1.5 billion over three years. This support from the government is projected to boost the market growth significantly in Canada.
APAC Market Statistics
APAC rare disease treatment market is witnessing a significant rise. As per a report dated July 2024, the Asia Pacific region is home to 60% of the global population. It has registered the highest number of people residing with a rare disease, with estimates indicating more than 258 million individuals. Noteworthy progress to advance access is underway across the APAC region, both in the higher-income and lower-income countries, and more technologically advanced countries such as Australia. In February 2020, the Australia government launched the country’s first National Strategic Action Plan for Rare Diseases. These factors are boosting the expansion of the market in several untouched areas in the region as well.
India rare disease treatment market is projected to witness notable growth owing to supporting policies from the local government for rare and orphan diseases. In March 2021, the Ministry of Health & Family Welfare announced the launch of the National Policy for Rare Diseases (NPRD). The policy includes 63 rare diseases on the reference of the Central Technical Committee for Rare Diseases (CTCRD). So far, 12 Centres of Excellence (CoEs) have been identified, including premier Government tertiary hospitals offering diagnostics, prevention, and treatment facilities for rare diseases. Per person monetary funding of up to Rs. 50 lakhs are offered for the treatment at the notified CoEs for Rare Diseases.
China rare disease treatment market is anticipated to garner a considerable share. According to the CEIBS, in February 2023, among the 7,000 worldwide confirmed rare diseases, over 1,400 cases have been diagnosed in China. NLM stated that in September 2023, the National Health Commission of China officially announced the Second List of Rare Diseases, comprising 86 rare diseases. The demand for therapies in the country is hence increasing. In November 2022, AstraZeneca introduced Soliris (eculizumab), through Alexion, for the treatment of PNH and aHUS in China for adults and children.
Companies Dominating the Rare Disease Treatment Landscape
- AbbVie Inc.
- Company Overview
- Business Strategy
- Key Product Offerings
- Financial Performance
- Key Performance Indicators
- Risk Analysis
- Recent Development
- Regional Presence
- SWOT Analysis
- Alexion Pharmaceuticals, Inc.
- Amgen Inc
- AstraZeneca PLC
- Baxter International
- Bayer AG
- Bristol-Myers Squibb
- Eli Lilly and Company
- F. Hoffmann La Roche Ltd
- GSK plc
- Johnson & Johnson
- Merck & Co. Inc.
- Pharmacyclics LLC
- Seagen Inc.
- Takeda Pharmaceutical Company Limited.
- Vertex Pharmaceutical, Inc.
Companies in the rare disease treatment market are majorly focusing on securing U.S. FDA orphan drug approvals for benefits such as tax credits and market exclusivity. In September 2024, UCB received U.S. FDA approval for BIMZELX for treating adults with active psoriatic arthritis (PsA) active non-radiographic axSpA with objective signs of inflammation, and active ankylosing spondylitis. They also pursue partnerships and acquisitions to broaden their pipelines and invest heavily in R&D, especially in gene and cell therapies. Furthermore, collaborations with patient advocacy groups help raise awareness and tailor treatments to meet specific patient needs.
In the News
- In September 2024, Chugai Pharmaceutical Co., Ltd. received approval from the MHLW Evrysdi Dry Syrup 60 mg (risdiplam) for an additional indication of pre-symptomatic SMA.
- In December 2023, Novartis received U.S. FDA approval for Fabhalta(iptacopan), which is a Factor B inhibitor. It is the first oral monotherapy for the treatment of adults with PNH.
Author Credits: Radhika Pawar
- Report ID: 6667
- Published Date: Apr 18, 2025
- Report Format: PDF, PPT
