An increase in the prevalence and misdiagnosis of rare diseases has led to an increase in the sales of orphan drugs.
Wilmington, Delaware, United States, Transparency Market Research Inc. -, Sept. 26, 2024 (GLOBE NEWSWIRE) — The orphan drugs market (희귀의약품 시장) was worth US$ 195.0 billion in 2023. The market is expected to reach US$ 424.0 billion by 2034, growing 6.9% CAGR from 2024 to 2034. Personalized medicine approaches designed to target the genetic abnormalities underlying rare diseases can be created as understanding of these diseases increases.
Pharmaceutical companies are encouraged to invest in orphan drug development by their exclusive market access period, which prevents competitors from entering the market. Due to less competition and payers’ willingness to pay for therapies that target rare diseases, orphan pharmaceuticals often enjoy higher prices and are more likely to be covered by payers.
Orphan drugs are drugs that are intended to treat rare diseases. Rare diseases, sometimes called orphan diseases, affect less than 65 people out of every 100,000. Orphan drug development is supported by similar programs in Australia, the European Union, and Japan, which offer grant programs, tax credits, and regulatory advantages.
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Key Findings of the Market Report
- Based on disease type, pulmonary diseases are expected to create a market for orphan drugs.
- The biologics segment is expected to create a market for orphan drugs.
- Pharmacy services in hospitals made up the largest segment of the market in 2023
- Orphan pharmaceuticals were most popular in North America in 2023.
Global Orphan Drugs Market: Growth Drivers
- Advances in biotechnology, including genomics, proteomics, and precision medicine, have facilitated the identification and understanding of rare diseases at the molecular level.
- To address specific disease mechanisms and improve patient outcomes, pharmaceutical companies can develop orphan drugs that address unmet medical needs in rare diseases.
- Due to technological advances in drug discovery and development, such as high-throughput screening, computational modeling, and gene editing methods such as CRISPR-Cas9, drug targets for uncommon diseases are being discovered and validated more quickly.
- Clinically effective orphan medications are being developed as a result of these developments. In addition, they simplify the process of developing drugs.
- With the globalization of clinical trials, pharmaceutical companies can access a wider range of patients, including rare disorders. Trials that occur across borders are more convenient, have greater generalizability, and are faster to get approved by regulatory agencies.
Global Orphan Drugs Market: Regional Landscape
- Orphan drugs are a significant market in North America. Pharmaceutical companies are incentivized by government programs like the Orphan Drug Act (ODA) in the United States and comparable legislation in other nations to create orphan medications. These incentives encourage funding for developing orphan drugs, including tax breaks, market exclusivity, and research grants.
- Regulatory bodies like Health Canada and the U.S. Food and Drug Administration (FDA) have set up specialized programs and regulatory procedures to speed up the development and approval of orphan medications.
- FDA initiatives such as the Orphan Drug Designation and Accelerated Approval route have made it possible to reduce regulatory procedures for orphan pharmaceuticals.
- A strong research infrastructure comprising eminent universities, research centers, and biotechnology hubs is present in North America. These institutes conduct preclinical and clinical research on orphan pharmaceuticals in partnership with pharmaceutical corporations, which utilize their resources and experience to expedite the medication development process.
- Healthcare costs in the United States are among the highest in the world due to the aging population, expensive prescription drugs, and sophisticated medical technology. The high cost of healthcare provides an environment that fosters the development of cutting-edge treatments for rare diseases.
Global Orphan Drugs Market: Competitive Landscape
As the orphan drugs market expands globally, new orphan drugs are offered by companies specializing in this field.
Key Players
- Novartis AG
- Bristol-Myers Squibb Company
- Celgene Corporation
- F. Hoffmann-La Roche Ltd.
- Pfizer, Inc.
- Sanofi S.A.
- Alexion Pharmaceuticals, Inc.
- Eli Lilly and Company
- Novo Nordisk A/S
- AstraZeneca plc
- Eisai Co., Ltd.
- Daiichi Sankyo Company Limited
- Bayer AG
- GlaxoSmithKline plc
- Merck & Co. Inc.
- Johnson & Johnson
- Biogen Inc.
- Shire plc
- Amgen Inc.
Key Developments
- In July 2023, Novartis acquired the San Diego-based preclinical biotechnology company DTx Pharma for US$ 500 million, the Swiss pharma company. The deal includes an additional US$ 500 million potential payment upon achieving certain milestones.
- In addition to the FALCON platform, Novartis acquired DTx’s proprietary Fatty Acid Ligand Conjugated OligoNucleotide (siRNA) therapeutic to enhance the distribution and absorption of siRNA therapies.
- In February 2024, Alexion, a company owned by AstraZeneca, received funding to develop a drug to treat a potentially fatal blood disorder. Voydeya has received a Breakthrough Therapy designation from the FDA and a Priority Medicines (PRIME) designation from the European Medicines Agency.
- As a result of its Orphan Drug Designation, Voydeya has been approved for the treatment of PNH in the United States, the European Union and Japan as well.
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Global Orphan Drugs Market: Segmentation
Disease Type
- Oncology
- Gastrointestinal
- Pulmonary
- Neurology
- Hematology
- Cardiovascular
- Metabolic Disorders
- Endocrinology
- Infectious Diseases
- Others
Indicator
- Biologic
- Non-biologic
Distribution Channel
- Hospital Pharmacies
- Specialty Pharmacies
- Retail Pharmacies
- Others
Region
- North America
- Europe
- Asia Pacific
- Latin America
- Middle East & Africa
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