Rare Disease Treatment Market – By Drug Type (Biologics, Non-biologics), Therapeutic Area (Cancer, Respiratory Disorder, Cardiovascular Disorder), Patient (Adult, Pediatric), Route of Administration (Oral, Injectable) – Global Forecast (2024 ­ 2032)

Rare Disease Treatment Market – By Drug Type (Biologics, Non-biologics), Therapeutic Area (Cancer, Respiratory Disorder, Cardiovascular Disorder), Patient (Adult, Pediatric), Route of Administration (Oral, Injectable) – Global Forecast (2024 ­ 2032)

Rare Disease Treatment Market Size

Rare Disease Treatment Market size was valued at USD 205.3 billion in 2023 growing at a CAGR of 9% between 2024 and 2032, driven by factors such the increasing prevalence of cancer and other rare diseases across the globe.

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For instance, as per the article published in National Library of Medicine in December 2023, it has been reported that in the European Union, there are approximately 30 million people living with a rare disease; in Germany there are an estimated 4 million. It is estimated that 300 million people are affected globally. Thus, the rapid increase in the prevalence of rare disease is the major factor that increase the demand for novel drug therapies to treat such disease. Moreover, the increase in awareness about rare diseases grows among healthcare providers, patients, and regulatory bodies. This awareness often leads to increase funding for research and development that propel the market growth.

Rare disease also known as orphan disease is caused due to genetic alterations and can be defined as the disease that affects a small percentage of the population. There are about 7,000 rare diseases. The cause of many rare diseases is unknown, but they are often caused by changes in a person’s genes or chromosomes. Also, these diseases are often more difficult to diagnose and treat than the more common diseases. Developing treatments and medications for rare diseases is often non-viable or difficult as the target population is very low. Thus, various government often provide research grants and funding for drug or treatment development.
 

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Rare Disease Treatment Market Trends

• The pharmaceutical industry is continuously involved in R&D activities for the development of novel drug therapies to treat rare diseases and commercialization of medicines for the treatment of uncommon diseases. Due to increase in funding for drug discovery and R&D activity, there are now significantly more novel molecular pharmaceuticals commercialized each year. More than 800 orphan drug indications have been approved from 1983 and 2019.
• Moreover, the target population base has also contributed to this success and has reinstated its capability to drive investment, inform research, and influence policymakers. For instance, as per the report published by U.S. FDA in January 2024, more than half (28 of 55, or 51%) of the novel drugs approved in 2023, these drugs were approved to prevent, diagnose, or treat a rare disease or condition, for which they received an orphan-drug designation. Therefore, the number of approved orphan indications is surging rapidly than the number of drugs, as some therapeutics are indicated for multiple conditions. Thus, increasing launch of novel drugs and treatment regimens for rare disease treatment will spur the market growth.

Rare Disease Treatment Market Analysis

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Based on the drug type, the global market is classified into biologics and non-biologics. The biologics segment dominated the market with revenue of USD 166.9 billion in 2023.

• The demand for biologics in rare disease treatment increases due to several benefits it possesses such as high specificity, low toxicity, extreme sensitivity to physical conditions, etc. Also, technological advancements in pharmaceutical and biotechnology industry allow large-scale syntheses of biologics in a cost-effective manner. In conditions of safety, biologics are identified to cause less severe adverse events.
• Furthermore, the cost of several biologic drugs is covered by the medical insurance, thus enabling patients to access effective treatments.

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Based on the therapeutic area, the global rare disease treatment market is classified into cancer, blood-related disorders, central nervous system, respiratory disorders, musculoskeletal disorders, cardiovascular disorders, and other therapeutic area. The cancer segment dominated the market with market share of 48.9% in 2023.

• Growing adoption of biological therapy including gene therapy, cytokine therapy, monoclonal antibodies, targeted drug therapy, immunoconjugates for treating the cancer will boost market growth in the future. Also, the growing emphasis on biologics to treat the rare diseases with high compatibility, specificity and low toxicity will witness steady growth in the future.
• Furthermore, various market players are focusing on development of novel products for treating these rare diseases that will propel the growth significantly.

Based on the patient, the global rare disease treatment market is classified into adult and pediatric. The adult segment is expected to exhibit CAGR of 8.7% during the analysis period.

• The adult segment contributed maximum share owing to increasing adult and geriatric population pool suffering from rare disorders. As per the report published by National Institute of Health in November 2023, it has been reported that an estimated 25 to 30 million Americans are affected with rare disease. In addition, Undiagnosed Disease Program (UDP) has received nearly 10,000 inquiries, reviewed more than 3,000 applications, and admitted about 900 patients to the NIH Clinical Center for comprehensive weeklong evaluations. Thus, the increase in the number of people diagnosed with rare disease and the growing demand to develop therapeutic drugs for adult patient population is substantially increasing in the recent past years that will further propel the market growth positively.  

Based on route of administration, the rare disease treatment market is classified into oral and injectable. The oral segment is expected to reach USD 235.1 billion by end of 2032.

• The high market revenue is attributed to various advantages of the oral route of administration such as non-invasiveness, the convenience of drug administration, and patient compliance among others.  According to the research article published in 2021, around 60% of approved small-molecule drugs available commercially are administered through the oral route.  Furthermore, oral therapies offer a higher rate of bioavailability at an affordable cost. Thus, the easy availability of oral dosage in store is another factor that will contribute the market growth for this segment.  

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In 2023, North America secured a substantial market share of 48.8% in the global rare disease treatment market and is expected to dominate throughout the forecast period.

• This is attributable to numerous variables including the presence of key market players, an increase in the number of rare diseases, and rising awareness regarding the benefits of precision medicine among others.
• Also, federal agencies such as the National Institutes of Health (NIH) and the Food and Drug Administration (FDA), allocate funding for rare disease research and development to support basic and translational research, clinical trials, and infrastructure development, thereby driving innovation in rare disease treatments.
• Furthermore, reimbursement policies in the U.S., including coverage by Medicare and private insurance companies, influence the commercial viability of rare disease treatments. Thus, the aforementioned factors contributed to North America's dominance in the market.

Germany to experience the highest growth rate in the market, outstripping other countries in Europe in the assessment period.

• Germany has a relatively high prevalence of rare diseases in the Europe region, with an estimated 4 million people affected. This large patient population creates a significant market opportunity for pharmaceutical companies developing rare disease treatments.
• Further, Germany has a well-developed clinical research infrastructure, including academic medical centers, research hospitals, and clinical trial networks. Thus, the availability of research infrastructure facilitates clinical trials for rare disease treatments, thereby accelerating their development and commercialization which in turn help in the market growth.

The India rare disease treatment market is expected to grow with a significant CAGR of 11.5% during the forecast period.

• The increase in awareness pertaining to rare diseases among healthcare providers, patients, and the public in India will leads to higher demand for treatment options, thereby supplementing the market growth.
• Additionally, Indian pharmaceutical companies often collaborate with international partners, including academic institutions, research organizations, and multinational pharmaceutical companies, to conduct clinical trials and develop rare disease treatments. These partnerships facilitate knowledge sharing, technology transfer, and resource pooling, thus fostering the country level market growth.

Rare Disease Treatment Market Share

The competitive landscape of the market is characterized by the presence of established pharmaceutical companies that manufacture and conduct intense R&D activities to provide rare disease treatment vying for market share. Key players are engaging in strategic initiatives such as collaboration, product launch, investment, and partnerships to strengthen their product portfolio. The companies are actively adopting a multi-pronged approaches to address the rising demand for rare disease treatment.

Rare Disease Treatment Market Companies

Prominent players operating in the rare disease treatment industry are as mentioned below

• AbbVie Inc.
• Alexion Pharmaceuticals, Inc.
• Amgen Inc
• AstraZeneca PLC
• Baxter International
• Bayer AG
• Biogen
• Bristol-Myers Squibb
• Eli Lilly and Company
• F. Hoffmann La Roche Ltd
• GSK plc
• Johnson & Johnson
• Merck & Co. Inc.
• Novartis AG
• Novo Nordisk
• Pfizer, Inc.
• Pharmacyclics LLC
• Sanofi SA
• Seagen Inc.
• Takeda Pharmaceutical Company Limited.
• Vertex Pharmaceutical, Inc.

Rare Disease Treatment Industry News

• In March 2024, AstraZeneca announced that it has entered into a definitive agreement to acquire Amolyt Pharma, a clinical-stage biotechnology company focused on developing novel treatments for rare endocrine diseases. This acquisition aims to expand its bone metabolism franchise with the notable addition of eneboparatide (AZP-3601), a Phase III investigational therapeutic peptide with a novel mechanism of action designed to meet key therapeutic goals for hypoparathyroidism. This strategy is expected to enhance their business relation and pipeline portfolio.
• In January 2024, Sanofi and Inhibrx, Inc. a publicly traded clinical-stage biopharmaceutical company focused on developing a broad pipeline of novel biologic therapeutic candidates, have entered into a definitive agreement under which Sanofi has agreed to acquire Inhibrx. This acquisition will provide the addition of INBRX-101 as a high potential asset to rare disease portfolio of Sanofi.

The rare disease treatment market research report includes an in-depth coverage of the industry with estimates & forecast in terms of revenue in USD Million from 2021 - 2032 for the following segments

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Market, By Drug Type

• Biologics
• Non-biologics

Market, By Therapeutic Area

• Cancer
• Blood-related disorders
• Central nervous system
• Respiratory disorders
• Musculoskeletal disorders
• Cardiovascular disorders     
• Other therapeutic areas

Market, By Patient

• Adult
• Pediatric

Market, By Route of Administration

• Oral
• Injectable

The above information is provided for the following regions and countries

• North America
  • U.S.
  • Canada
• Europe
  • Germany
  • UK
  • France
  • Italy
  • Spain
  • Poland
  • Switzerland
  • Sweden
  • Rest of Europe
• Asia Pacific
  • Japan
  • China
  • India
  • Australia
  • South Korea
  • Indonesia
  • Thailand
  • Vietnam
  • Rest of Asia Pacific
• Latin America
  • Brazil
  • Mexico
  • Argentina
  • Chile
  • Colombia
  • Peru
  • Rest of Latin America
• Middle East and Africa
  • South Africa
  • Saudia Arabia
  • UAE
  • Israel
  • Iran
  • Egypt
  • Rest of Middle East and Africa