Rare Disease Treatment Market size was over USD 224.6 billion in 2024 and is estimated to reach USD 652.9 billion by the end of 2037, expanding at a CAGR of 9.3% during the forecast timeline, i.e., 2025-2037. In 2025, the industry size of rare disease treatment is assessed at USD 245.4 billion. 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.
Growth Drivers
Challenges
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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 652.9 billion |
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Regional Scope |
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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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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 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.
Author Credits: Radhika Pawar
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