Rare Disease Drugs; Drugs Intended For the Treatment or Prevention of Rare Diseases
Rare disease drugs are widely used for the treatment of rare
disease, any disease that affects a small percentage of the population. The
Orphan Drug Act defines a rare disease as a disease or condition that impacts
less than 200,000 people in the U.S. Rare diseases are known as orphan diseases
as drug/pharma companies are not interested in adopting them to develop
treatments. Orphan Drug Act created financial incentives to encourage companies
to develop rare diseases drugs. Orphan drug is a pharmaceutical agent developed
to treat rare disease.
According to Coherent
Market Insights the Global
Rare Disease Drugs Market Competitive Analysis.
The exact cause for many rare diseases remains unknown. Rare
disease drugs are those that are intended for the treatment or prevention of a
rare diseases. Drug development in rare diseases is challenging for many reasons,
including the complex biology and the lack of understanding of the natural
history of many rare diseases. The United States Food and Drug Administration
(FDA) has approved hundreds of drugs for rare diseases, but most
life-threatening rare diseases do not have FDA-approved treatments.
While referred to as rare due to the smaller size of
individual patient populations, rare diseases are collectively common and their
burden on society is staggering. There are more than 7,000 rare diseases,
according to the National Institutes of Health (NIH). Moreover, the FDA Office
of Orphan Products Development (OOPD) mission is to advance evaluation and
development of products (drugs, biologics, devices, or medical foods) that
demonstrate promise for both the diagnosis and treatment of rare diseases.
In the United States, FDA has authority to grant orphan-drug
designation to a drug or biological product to prevent, diagnose or treat a
rare disease. In 2020, the FDA approved 32 novel drugs and biologics with
orphan drug designation. Novel drugs approved in 2019 with orphan drug
designation were: Brukinsa, Adakveo, Cablivi, Egaten, Givlaari, Ga 68 DOTATOC, Inrebic,
Polivy, Oxbryta, pretomanid, Rozlytrek, Reblozyl, Scenesse, TissueBlue, Turalio,
Trikafta, Sunosi, Vyondys 53, Vyndaqel, Wakix, and Xpovio.
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