Orphan Drugs Market — Global Industry Insights, Trends,
Size, Share, Outlook, and Opportunity Analysis, 2018-
2026
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 There are few drugs that have been withdrawn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.
However, these drugs have shown good results for treatment of those diseases which in
particular do not have any standard drug, owing to drive the orphan drugs market. For
instance, thalidomide used as a hypnotic drug was withdrawn from market when teratogenic
risk was discovered, however, this drug has shown prominent result in treating leprosy.