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Targeting Rare Diseases

Posted on March 20th, 2017 by in Pharma R&D


What is known as a “rare” disease might affect anywhere between a couple hundred to a couple hundred thousand people here in the U.S. And it’s estimated that as much as 10% of the nation’s population has one of the thousands of known rare diseases, most of which are genetic. Clearly rare diseases aren’t so rare at all, and that is why they have become a growing market – one that is in fact growing even faster than the market for traditional drugs, and which has some 400 drugs currently in development.

Recent strides in precision medicine have helped make it possible for scientists to target rare diseases, and advocacy groups have been urging researchers in that direction. Sensing that the era of “blockbuster” drugs is coming to an end, pharmaceutical companies have become more willing to direct their R&D departments to look at orphan drugs for new possibilities. It doesn’t hurt either that the combination of technological advancement and a friendlier regulatory environment is making it easier to do research on rare diseases.

Already, orphan drugs are being repurposed to treat rare diseases ranging from uncommon cancers to enzyme deficiency disorders, and continued advances will surely bring more therapies to help people suffering from rare conditions.

To learn more about the challenges and opportunities in this area, check out the article Focus on Rare Diseases in PharmaVOICE.


All opinions shared in this post are the author’s own.

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