Global Orphan Drugs Market to Grow at 9.97% During 2025-2033, Propelled by Growing Number of Rare Diseases

December 27, 2024 | Healthcare

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IMARC Group’s latest report, titled “Orphan Drugs Market Report by Drug Type (Biological, Non-Biological), Disease Type (Oncology, Hematology, Neurology, Cardiovascular, and Others), Phase (Phase I, Phase II, Phase III, Phase IV), Top Selling Drugs (Revlimid, Rituxan, Copaxone, Opdivo, Keytruda, Imbruvica, Avonex, Sensipar, Soliris, and Others), Distribution Channel (Hospital Pharmacies, Retail Pharmacies, Online Stores, and Others), and Region 2025-2033,” finds that the global orphan drugs market size reached USD 237.3 Billion in 2024. Orphan drugs refer to medications that are intended for diagnosing, preventing and treating rare, life-threatening diseases or disorders. As they traditionally had a limited market, very few pharmaceutical companies pursued research into these products. Moreover, orphan drug research is dependent on government incentives. However, the growing number of rare diseases lacking treatment, along with improving access to healthcare and spending capacity of individuals, has led to a significant rise in the demand for orphan drugs.

Global Orphan Drugs Market Trends:

At present, incentives for drug development provided by government authorities in various countries are encouraging pharmaceutical companies to develop orphan drugs. For instance, the United States Food and Drug Administration (USFDA) offers benefits such as fast-track approval, protocol assistance, waiver of fees and marketing exclusivity to manufacturers, facilitating the development and commercialization of diagnostic tools and treatments devoted to rare diseases. Furthermore, there has been a rise in the development of orphan biological drugs that can prevent or treat various forms of cancer, tumors and blood disorders, which, in turn, is contributing to the market growth. In 2019, the Center for Drug Evaluation and Research (CDER), a division of the USFDA, approved various new orphan drug therapies for patients with numerous disorders, including tenosynovial giant cell tumor, sickle cell disease, diffuse large B-cell lymphoma, mantle cell lymphoma, acquired thrombotic thrombocytopenic purpura, beta-thalassemia, and acute hepatic porphyria. Looking forward, IMARC Group expects the market value to reach USD 582.5 Billion by 2033, exhibiting a CAGR of 9.97% during 2025-2033.

Market Summary:

Based on the drug type, the market has been bifurcated into biological and non-biological drugs.
On the basis of the disease type, the market has been segregated into oncology, hematology, neurology, cardiology and others.
The market has been categorized based on the phase into phase I, II, III and IV.
The report has covered some of the top-selling drugs, including revlimid, rituxan, copaxone, opdivo, keytruda, imbruvica, avonex, sensipar, soliris and others.
The market has been segmented based on the distribution channel into hospital pharmacies, retail pharmacies, online stores and others.
Region-wise, the market has been divided into North America (the United States and Canada), Asia Pacific (China, Japan, India, South Korea, Australia, Indonesia and others), Europe (Germany, France, the United Kingdom, Italy, Spain, Russia and others), Latin America (Brazil, Mexico and others), and Middle East and Africa.
The competitive landscape of the market has been examined, with some of the key players being AbbVie Inc., Alexion Pharmaceuticals Inc., Amgen Inc., Biogen Inc., Bristol-Myers Squibb Company, F. Hoffmann-La Roche AG, Jazz Pharmaceuticals Plc, Johnson & Johnson Private Services, Inc., Merck & Co. Inc., Novartis AG, Pfizer Inc., Sanofi S.A., Takeda Pharmaceutical Company Limited and Teva Pharmaceutical Industries Ltd.

Report Scope:

Report Features	Details
Base Year of the Analysis	2024
Historical Period	2019-2024
Forecast Period	2025-2033
Units	Billion USD
Segment Coverage	Drug Type, Disease Type, Phase, Top Selling Drugs, Distribution Channel, Region
Region Covered	Asia Pacific, Europe, North America, Latin America, Middle East and Africa
Countries Covered	United States, Canada, Germany, France, United Kingdom, Italy, Spain, Russia, China, Japan, India, South Korea, Australia, Indonesia, Brazil, Mexico
Companies Covered	AbbVie Inc., Alexion Pharmaceuticals Inc., Amgen Inc., Biogen Inc., Bristol-Myers Squibb Company, F. Hoffmann-La Roche AG (Roche Holding AG), Jazz Pharmaceuticals Plc, Johnson & Johnson, Merck & Co. Inc., Novartis AG, Pfizer Inc., Sanofi S.A., Takeda Pharmaceutical Company Limited and Teva Pharmaceutical Industries Ltd.
Customization Scope	10% Free Customization
Post-Sale Analyst Support	10-12 Weeks
Delivery Format	PDF and Excel through Email (We can also provide the editable version of the report in PPT/Word format on special request)

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Global Orphan Drugs Market to Grow at 9.97% During 2025-2033, Propelled by Growing Number of Rare Diseases

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