Orphan Drugs Market by product type, and disease type - Global Industry Insights, Trends, 2025

ORPHAN DRUGS MARKET
Global Industry Insights, Trends, Outlook, and
Opportunity Analysis, 2017–2025
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REPORT DESCRIPTION
Orphan Drugs Market Overview
The drugs which are developed for treatment of rare diseases and conditions are referred to as
orphan drugs. The rare diseases such as Tourette syndrome, Huntington’s disease, muscular
dystrophy, myoclonus, and ALS (Lou Gehrig’s disease) are some of those diseases for treatment
of which adequate drugs have not been developed, and hence these diseases are treated by
orphan drugs. Orphan drugs are developed by the pharmaceutical companies for public health
need, and the sales generated by this drugs are very minimal when compared to normal drugs.
The orphan diseases manifest in patient populations representing 6-8% of the global population.
Symptoms of some orphan diseases may appear at birth or in childhood including cystic fibrosis,
infantile spinal muscular atrophy, familial adenomatous polyposis (FAP), and some others.
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Developing novel indications for designated orphan drugs is expected to drive the orphan
drugs market
There are around 5,000 rare diseases listed globally and 80% of those are to genetic origins. The
probability of getting infected by these diseases in adults is 50%.The need for the development
of orphan drugs which will treat such diseases and proactive initiatives undertaken by
government and regulatory authorities are the major factors driving the orphan drugs market.
For instance, the authorities of U.S. FDA for orphan drug development department are providing
the incentives to healthcare and biotechnology industries in order to enhance their research
and development in orphan drugs. There are few drugs that have been withdrawn from the
market due to certain risk developed over prolong administration of these drugs.
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REPORT DESCRIPTION
The increasing prevalence of rare cancer in patients owing to oncological diseases segment to dominate in
the orphan drugs market
The global orphan drugs market is segmented on the basis of disease, by product type and by region
On the basis of disease type,
• Hematologic and Immunologic diseases
• Infectious diseases
• Metabolic diseases
• Neurologic diseases
• Oncologic diseases
• Other orphan diseases
On the basis of product type,
• Biological orphan drugs
• Non-biological orphan drugs
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Numerous collaborative research programs across North America and Europe is owing to
growth of orphan drugs market
Regional segmentation of orphan drugs market by Coherent Market Insights comprises of North
America, Latin America, Europe, Asia Pacific, Middle East, and Africa. North America dominates
the market globally due to factors such as established regulatory and reimbursement framework
and incentives given by U.S. Food and Drug Administration (FDA) for research and development
in orphan drugs sector. The various collaborative research programs across United States and
Europe provides research data on rare diseases to manufacturers, patients and health experts
which gives timely medical care, thus boost the orphan drug market. Whereas the prevalence of
genetic diseases and growth of population are some factors which might allow the orphan drugs
manufacturers to invest in the Asia Pacific region.
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Initiatives taken by government to increase the investment in orphan drugs development driving
the growth of orphan drugs market.
Key players operating the orphan drugs market are Acorda Therapeutics, Actelion Pharmaceuticals
Ltd., Amgen, Alexion Pharmaceuticals, Baxter international, Bayer healthcare pharmaceuticals,
Biomarin Pharmaceuticals, Novartis, F.Hoffmann-La Roche AG, Johnson and Johnson and many
others. The increase in investments, and some initiatives taken by government helping the orphan
drug manufacturers. For instance, U.S. government provides 50% of tax credit on the research and
development expenses spent on the orphan drugs development by manufacturers. However the
high cost of the orphan drugs and generation of small sales from it is amongst those factors which is
lowering the interest of companies for market.
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The drugs which are developed for treatment of rare diseases and conditions are referred to as orphan drugs. The rare diseases such as Tourette syndrome, Huntington’s disease, muscular dystrophy, myoclonus, and ALS (Lou Gehrig’s disease) are some of those diseases for treatment of which adequate drugs have not been developed, and hence these diseases are treated by orphan drugs. Orphan drugs are developed by the pharmaceutical companies for public health need, and the sales generated by this drugs are very minimal when compared to normal drugs. The orphan diseases manifest in patient populations representing 6-8% of the global population. Symptoms of some orphan diseases may appear at birth or in childhood including cystic fibrosis, infantile spinal muscular atrophy, familial adenomatous polyposis (FAP), and some others. Request to download and view Sample Report: https://www.coherentmarketinsights.com/insight/request-sample/500