Vancouver, Canada — The global orphan drug market size was USD 154.20 Billion in 2022 and is expected to register a rapid revenue CAGR of 12.3 % during the forecast period. Rising frequency of uncommon illnesses and increasing number of governments providing regulatory incentives for development of orphan drugs are major factors driving market revenue growth.
A federal law known as the Orphan Disease Act (ODA) addresses rare disorders, sometimes known as orphan diseases, that are either extremely rare or impact fewer than 200,000 Americans (less than 5 per 10,000 in the general population). Similarly, in Europe, an orphan medicine is one that addresses a disease or condition that affects fewer than 5 people in 10,000 (Orphan Medicine Regulation 141/2000).
The rapid growth of the pharmaceutical sector and increasing prevalence of rare diseases are also expected to drive revenue growth of the market. In recent years, there has been a global increase in the occurrence of rare diseases among people. To address this problem, both industrialized and developing countries have created laws that encourage the creation of medications for uncommon illnesses and ensure that these medications are readily available.
In May 2022, 30 million Americans, or 1 in 10 of the population, suffer from a rare disease, and there are about 7,000 identified rare diseases, according to the Genetic and Rare Diseases (GARD) Information Centre. Moreover, according to data updated by ’Global Genes ‘in September 2021, rare conditions affect about 400 million individuals worldwide. Thus, chances for breakthrough orphan medications are being created by rising Research & Development (R&D) spending.
Market Segment Insights
Type Insights:
On the basis of drug type, the orphan drug market is segmented into biologics and non- biologics. The biologics segment accounted for largest market share in the global orphan drug market in 2022 because of rising number of products that are categorized as biologics. The U.S. Food and Drug Administration (FDA) estimates that as of 1983, the organization’s Office of Orphan Products Development (COPD) has created and distributed over 600 medications and biologic products to treat uncommon illnesses.
Therapy Insights:
On the basis of therapy, the global orphan drug market is segmented into oncology, hematology, neurology, infectious diseases, metabolic disorders, endocrinology, immunology and others. The oncology segment is expected to register moderately fast market growth rate in the global orphan drug market during the forecast period. This is due to increasing number of major players in the industry concentrating on producing oncology medications, which are widely used to treat cancer. For instance, in June 2022, the U.S. Food and Drug Administration (FDA) granted Evorpacept Orphan Drug Designation (ODD) to ALX Oncology Holdings Inc. so that it could be used to treat individuals with acute myeloid leukemia.
Regional Insights:
North America accounted for largest market share in the global orphan drug market in 2022 This is because increasing number of governments supporting patients with rare diseases through money and grants for research due to high occurrence of these conditions in the U.S. The fact that a drug designated as an orphan drug in the U.S. has seven years of marketing exclusivity after receiving FDA approval for a particular indication, tax credits, and user fee waivers.
In addition, the Genetic and Rare Diseases Information Centre (GARD) updated their data in May 2022, estimating that around 30 million Americans and 3.1 million Canadians, respectively, suffer from rare diseases. These figures are based on the Canadian Organisation for Rare Disorders (CORD) 2021 update.
Asia Pacific accounted for third-largest market share in the global orphan drug market in 2022 due to increasing government programs and regulations that encourage orphan drug Research & Development (R&D). In addition, strong healthcare infrastructure and rising product uptake are other factors driving market revenue growth of this region during the forecast period. According to the Japanese Ministry of Health, Labour, and Welfare, an orphan medicine is one that is prescribed to treat an illness, for which there are less than 50,000 prevalent cases.
Major Companies and Competitive Landscape
The global orphan drug market is fragmented, with large and medium-sized players accounting for the majority of market revenue. Major players are deploying various strategies, entering into mergers & acquisitions, strategic agreements & contracts, developing, testing, and introducing more effective products in the market.
Some of the major companies included in the global orphan drug market report are:
- Pfizer Inc.
- AbbVie Inc.
- Novartis AG
- Amryt Pharma plc
- Amgen Inc.
- GSK plc.
- JOHNSON & JOHNSON
- Sanofi
- F. Hoffmann-La Roche Ltd
- Takeda Pharmaceutical Company Limited.
- Teva Pharmaceutical Industries Ltd.
- Bristol-Myers Squibb Company
- Biogen
- Merck & Co., Inc.
- Jazz Pharmaceuticals, Inc,
Strategic Development
- On 24 April 24, 2023, Genentech, a member of the Roche Group announced three new partnerships focused on improving brain health outcomes. These industry-leading initiatives are designed to quantify and assess the complexity, economic impact and patient and care partner burden of brain health conditions and generate robust data around the nuanced dynamics of neurologic care.
- On January 16, 2023, CARsgen Therapeutics Holdings Limited collaborated with Huadong Medicine (Hangzhou) Co., Ltd. for the commercialization of CARsgen’s BCMA CAR-T product CT053 in mainland China. Under the terms of the agreement, CARsgen will receive an upfront payment of around USD 28 million and is eligible to receive regulatory and commercial milestone payments up to USD 140 million.
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