Orphan drugs - Statistics & Facts

Published by Matej Mikulic, May 30, 2018
A rare disease is characterized by the number of people that it affects in comparison to the general population. Based on European standards, a disease is considered rare if it impacts one person per 2,000. There is an estimated 8,000 different types of rare diseases and many are considered chronic and life-threatening conditions. It is also possible that a rare disease in one region may be common in a different region of the world.

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In the following 4 chapters, you will quickly find the 17 most important statistics relating to "Orphan drugs".


Rare diseases have long been under-researched and many doctors and politicians were unaware that these conditions existed. At the time, both health policies and research that target rare diseases were limited. Since then, the number of treatment has risen significantly, which has made it easier for patients living with these diseases to live more comfortably. Nevertheless, patients living with rare diseases may have to spend significantly more on treatments than those with more common diseases due to the high prices of orphan drugs.

Pharmaceutical medications that are used to treat rare diseases are known as orphan drugs. In the United States, the Orphan Drug Act of 1983 started to incentivize the production of orphan drugs. In 2017, there were 459 orphan drug designations accepted, while ten years before, there were only 119 such designations. Roche produces one of the most successful pharmaceutical drugs for rare diseases, Rituxan. This drug is primarily used for the treatment of rheumatoid arthritis. Combined, Roche and Novartis, Switzerland-based pharmaceutical companies, have been able to generate almost 23 billion U.S. dollars in orphan drug revenues worldwide in 2017.

Orphan drugs

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