Why Pharma Is Investing in Rare Diseases

The global pharmaceutical industry is pouring billions of dollars into developing treatments for rare diseases, which once drew little interest from major drugmakers but now point the way toward a new era of innovative therapies and big profits.

The investments come as researchers harness recent scientific advances, including the mapping of the human genome, sophisticated and affordable genetic tests and laboratory robots that can screen thousands of compounds per hour in search of the most potent ones. "It's a very, very promising time," said Jimmy Lin, a National Institutes of Health cancer researcher who cofounded the Rare Genomics Institute.

By definition, a rare disease is one that strikes fewer than 200,000 Americans, sometimes only a few dozen. But with 7000 rare diseases known to doctors, and more emerging all the time, nearly 1 in 10 Americans has a rare disease. For most, there is no treatment, let alone a cure. Just getting an accurate diagnosis often requires a medical odyssey, and 30% of children with a rare disease die before age 5 years.

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