Overview and Scope
Orphan drugs refer to drugs that are specifically designed to treat an uncommon medical problem. It might be characterized as medicines that address public health needs but are not produced by the pharmaceutical industry due to financial constraints. Since a chemical may be used to treat a common condition but not have been designed for another, more rare indication, the indications of a medicine may likewise be called “”””orphan.””””
Sizing and Forecast
The orphan drugs market size has grown rapidly in recent years. It will grow from $188.45 billion in 2023 to $208.04 billion in 2024 at a compound annual growth rate (CAGR) of 10.4%. The growth in the historic period can be attributed to orphan drug act of 1983, rising interest in rare diseases, drug repurposing, advancements in genetic understanding, research funding initiatives.
The orphan drugs market size is expected to see exponential growth in the next few years. It will grow to $481.69 billion in 2028 at a compound annual growth rate (CAGR) of 23.4%. The growth in the forecast period can be attributed to evolving market access policies, value-based healthcare initiatives, global market expansion, patient-centric drug development, targeting rare cancers. Major trends in the forecast period include increasing target identification, accelerated regulatory pathways, advancements in gene therapy, expanded research collaborations, precision medicine approaches.
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Segmentation & Regional Insights
The orphan drugs market covered in this report is segmented –
1) By Therapy Area: Oncology, Blood, Central Nervous System, Endocrine, Cardiovascular, Respiratory, Immunomodulatory
2) By Diseases Type: Oncology, Hematology, Neurology, Cardiovascular, Other Diesease Types
3) By Drug Type: Biological, Non-Biological
4) By Distribution Channel: Hospital Pharmacy, Retail Pharmacy, Online Sales
North America was the largest region in the global orphan drugs market in 2023. Middle East is expected to be the fastest growing region in the orphan drugs market report during the forecast period. The regions covered in the orphan drugs market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa.
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Major Driver Impacting Market Growth
The rising prevalence of rare diseases is a key factor driving the growth of the orphan drugs market. Any disease that affects a small percentage of the population is rare. Rare disease treatments often require a cold chain, a supply chain regulated by the temperature that is not readily accessible in all countries. Orphan diseases or rare diseases occur rarely among people (i.e., 7 out of 10,000). However, globally, the prevalence of rare diseases is increasing in recent years. For instance, in January 2022, according to an article shared by the American Cancer Society, in USA, there are about 54,000 new instances of oropharyngeal or oral cancer each year. 11,230 people lose their lives to oropharyngeal or oral cancer. The average age of most people diagnosed with these cancers is 63, but they can occur in young people. Just over 20% (1 in 5) of cases occur in patients younger than 55. Thus, the rising prevalence of rare diseases is driving the growth of the orphan drugs market.
Key Industry Players
Major companies operating in the orphan drugs market report are Pfizer Inc., Johnson & Johnson Services Inc., F Hoffmann-La Roche Ltd., Farbenfabriken Bayer AG, Novartis AG, Sanofi SA, Bristol-Myers Squibb Co., AstraZeneca plc, GlaxoSmithKline plc, Takeda Pharmaceutical Company Limited, Eli Lilly and Company, Gilead Sciences Inc., Applied Molecular Genetics Inc., Novo Nordisk A/S, Merck & Co. KG, AbbVie Inc., Teva Pharmaceutical Industries Ltd., Regeneron Pharmaceuticals Inc., CSL Limited, Biogen Inc., Celgene Corporation, Vertex Pharmaceuticals Inc., Eisai Co. Ltd., Grifols SA, Alexion Pharmaceuticals Inc., BioMarin Pharmaceutical Inc., Aegerion Pharmaceuticals Inc., Sankyo Co. Ltd., Shire plc, Actelion Pharmaceuticals Ltd.
The orphan drugs market report table of contents includes:
1. Executive Summary
2. Orphan Drugs Market Characteristics
3. Orphan Drugs Market Trends And Strategies
4. Orphan Drugs Market – Macro Economic Scenario
5. Global Orphan Drugs Market Size and Growth
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32. Global Orphan Drugs Market Competitive Benchmarking
33. Global Orphan Drugs Market Competitive Dashboard
34. Key Mergers And Acquisitions In The Orphan Drugs Market
35. Orphan Drugs Market Future Outlook and Potential Analysis
36. Appendix
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