Rare Disease Treatment Market Size to Worth USD 587.08 Billion by 2034

The rare disease treatment market size is calculated at USD 217.93 billion in 2025 and is expected to reach around USD 587.08 billion by 2034, growing at a CAGR of 11.64% for the forecasted period.

Ottawa, Sept. 30, 2025 (GLOBE NEWSWIRE) -- The global rare disease treatment market size was valued at USD 195.21 billion in 2024 and is predicted to hit around USD 587.08 billion by 2034, rising at a 11.64% CAGR, a study published by Towards Healthcare a sister firm of Precedence Research. The global rare disease treatment market is driven by the increasing innovations and growing government support.

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Key Takeaways

North America held a major revenue of 63% share of the market in 2024.
Asia-Pacific is expected to witness the fastest growth in the rare disease treatment market during the forecast period.
By therapeutic area, the cancer segment dominated the market in 2024.
By therapeutic area, the musculoskeletal conditions segment is expected to grow significantly during the forecast period.
By drug, the biologics segment dominated the market in 2024.
By drug, the biosimilars segment is expected to grow significantly during the forecast period.
By route of administration, the injectable segment dominated the market in 2024.
By route of administration, the oral segment is expected to grow significantly during the forecast period.
By distribution channel, the specialty pharmacy segment dominated the market in 2024.
By distribution channel, the hospital pharmacy segment is expected to be the fastest-growing during the forecast period.

What are the Rare Disease Treatments?

The rare disease treatment market is expanding due to increasing supportive government policies, scientific advances, and growing investments. The rare disease treatments encompass therapies to diagnose, manage, or treat rare diseases. The treatment options present consist of orphan drugs, gene therapies, RNA therapies, and enzyme replacement therapies. At the same time, there is growth in the demand for precision medicine and biologics.

Market Scope:

Metric	Details
Market Size in 2025	USD 217.93 Billion
Projected Market Size in 2034	USD 587.08 Billion
CAGR (2025 - 2034)	11.64	%
Leading Region	North America share by 63%
Market Segmentation	By Therapeutic Area, By Drug, By Route of Administration, By Distribution Channel, By Region
Top Key Players	Arrowhead Pharmaceuticals, Biogen, Inc., Eterna Therapeutics, EveryONE Medicines, Evotec, Genethon, Novartis AG, Pfizer, Reata Pharmaceuticals, Inc., Stoke Therapeutics, Inc., SynaptixBio, Takeda Pharmaceuticals

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What are the Major Growth Drivers Rare Disease Treatment Market?

The growing diagnosis of rare diseases is the major driver in the market, increasing the use of various treatment options. The growing awareness is also increasing their genetic testing, which is enhancing the demand and use of advanced therapies. Additionally, growth in biologics, healthcare investments, rising rare diseases, growing R&D, and technological advancements are other market drivers.

What are the Key Drifts in the Rare Disease Treatment Market?

The market has been expanding due to the growing investments, grants, funding, and government initiatives to enhance the development, innovations, and use of various rare disease treatment options.

In September 2025, a total of £200,000 investment was secured to support people suffering from musculoskeletal conditions by a Bristol social enterprise. The investment will be used to expand their services using AI technologies to promote in-person, community-led, and virtual exercise sessions.
In September 2025, $10-million grant was received from the Weston Family Foundation by The Neuro, McGill University’s Dr. Pedro Rosa-Neto, where it will be used to accelerate the project, enhance existing infrastructure at the McConnell Brain Imaging Centre (BIC), and acquire a new Panorama GS tomograph.
In September 2025, a total of $60m was secured in the Series B funding round of China’s Epigenic Therapeutics, which will be used to support the development of its epigenetic inactivator, EPI-003 and EPI-001, for the treatment of chronic hepatitis B and hypercholesterolemia, respectively.
In December 2024, 974 crores for the financial years 2024–25 and 2025–26 was allocated as directed by the Delhi High Court to establish the National Fund for Rare Diseases (NFRD). Similarly, for 2026–27 and 2027–28 same amount will be allocated.

What is the Significant Challenge in the Rare Disease Treatment Market?

High treatment cost acts as the major limitation in the use of rare disease treatments. This, in turn, limits their adoption and use, affecting the patient outcomes. Moreover, the limited volume of patients, manufacturing complexities, regulatory limitations, and diagnostic challenges act as other market challenges.

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Regional Analysis

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Why did North America Dominate the Rare Disease Treatment Market in 2024?

North America dominated the market in 2024 with 63%, due to growth in the healthcare investment backed by government incentives. The growing R&D and adoption of advanced technologies are also increasing the development of rare disease treatments. Moreover, the presence of reimbursement policies also enhanced the patient outcome.

What Made the Asia Pacific the Fastest Growing Region in the Rare Disease Treatment Market in 2024?

The Asia Pacific is estimated to be the fastest-growing region in the market during the upcoming years, driven by expanding healthcare and growing awareness. This is increasing the rare disease early diagnosis, promoting their treatment. Growing R&D, reimbursement policies, and government support are also promoting their use, enhancing the market growth.

Segmental Insights

By therapeutic area analysis

Why Did the Cancer Segment Dominate in the Rare Disease Treatment Market in 2024?

By therapeutic area, the cancer segment led the market in 2024, due to its unmet needs. This, in turn, increased the demand for targeted treatment options, as well as enhanced the research and development, which were supported by various investments. At the same time, growing incidences also increased the use of other treatment options.

By therapeutic area, the musculoskeletal conditions segment is expected to show lucrative growth during the predicted time. Due to growing advances in genetic testing, their early detection is increasing, promoting their early treatment. Additionally, the growing interest in the development of gene therapies for their treatment is also driving the market.

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By drug analysis

Which Drug Type Segment Held the Dominating Share of the Rare Disease Treatment Market in 2024?

By drug, the biologics segment held the dominating share of the market in 2024, driven by the targeted action. Therefore, due to their enhanced specificity, their use in the treatment of various rare diseases has increased to deal with their complex mechanisms. Furthermore, the biotechnological companies also contributed by enhancing the development of various therapies.

By drug, the biosimilars segment is expected to show significant growth during the predicted time. Due to their affordability, their use is increasing for the treatment of a variety of rare diseases. At the same time, the expiration of patents is attracting companies to develop their cheaper versions.

By route of administration analysis

What Made Injectable the Dominant Segment in the Rare Disease Treatment Market in 2024?

By route of administration, the injectable segment led the market in 2024, as they are ideal route for various biologics. It also provided fast and targeted action, as well as enhanced the drug bioavailability. Moreover, their long duration of actions also enhanced their use and patient adherence to the treatment.

By route of administration, the oral segment is expected to show lucrative growth during the upcoming years. Their development is increasing as they are enhancing patient convenience. Oral small molecules are also being developed, which is promoting home-based treatment.

By distribution channel analysis

How the Specialty Pharmacy Segment Dominated the Rare Disease Treatment Market in 2024?

By distribution channel, the specialty pharmacy segment held the largest share of the market in 2024, driven by its special storage and handling of the rare drugs. Moreover, they also provide personalized services, education, and insurance policies. This increased their use, contributing to the market growth.

By distribution channel, the hospital pharmacy segment is expected to show the highest growth during the upcoming years. The presence of various rare disease treatments, along with their proper handling and distribution, is enhancing their use. They also provide help during emergency conditions.

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The global clinical trial services market is valued at USD 60.7 billion in 2024 and is expected to increase to USD 66.07 billion in 2025. By 2034, the market is projected to reach USD 141.85 billion, registering a CAGR of 8.85%.

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The global veterinary infectious disease diagnostics market is estimated at USD 2.68 billion in 2024 and is expected to rise to USD 2.89 billion in 2025. By 2034, the market is projected to reach USD 5.76 billion, growing at a CAGR of 7.95%.

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Recent Developments in the Rare Disease Treatment Market

In September 2025, once-daily injection, Forzinity for a rare, genetic disease, that is Barth syndrome, developed by Stealth Biotherapeutics, was approved by the U.S. FDA.
In September 2025, the FDA nod was received for Palsonify, developed by Crinetics, which is recommended as the first-line treatment for adults with acromegaly, where surgery was not effective or surgery was not an option.
In September 2025, AYUSH-MANN, which is an initiative to enhance public awareness on the role of integrative approaches in the treatment of neurological disorders and mental health, was launched by the collaboration between the Ministry of AYUSH, Government of India, and the National Institute of Mental Health and Neurosciences (NIMHANS).

Rare Disease Treatment Market Key Players List

Eterna Therapeutics
Arrowhead Pharmaceuticals
EveryONE Medicines
Biogen, Inc.
Novartis AG
Evotec
Pfizer
Stoke Therapeutics, Inc.
Genethon
Takeda Pharmaceuticals
Reata Pharmaceuticals, Inc.
SynaptixBio

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Segments Covered in The Report

By Therapeutic Area

Cancer
Musculoskeletal Conditions
Neurological Conditions
Cardiovascular Conditions
Hematologic Disorders
Infectious Diseases
Metabolic Disorders
Endocrine Disorders
Others

By Drug

Biologics
Biosimilars
Small Molecules

By Route of Administration

Oral
Injectable
Others

By Distribution Channel

Hospital Pharmacy
Specialty Pharmacy
Online Pharmacy

By Region

North America

U.S.
Canada

Asia Pacific

China
Japan
India
South Korea
Thailand

Europe

Germany
UK
France
Italy
Spain
Sweden
Denmark
Norway

Latin America

Brazil Mexico
Argentina

Middle East and Africa (MEA)

South Africa
UAE
Saudi Arabia
Kuwait

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Towards Healthcare is a leading global provider of technological solutions, clinical research services, and advanced analytics, with a strong emphasis on life science research. Dedicated to advancing innovation in the life sciences sector, we build strategic partnerships that generate actionable insights and transformative breakthroughs. As a global strategy consulting firm, we empower life science leaders to gain a competitive edge, drive research excellence, and accelerate sustainable growth.

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