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Orphan Drugs: Small Is the New Big


"Big Pharma is used to thinking big, but more and more companies are turning to the rare diseases category and to conditions that affect patient groups with populations less than than some small towns."

Medical Marketing & Media Deborah Weinstein

Unsurprisingly, Big Pharma is used to thinking big. But Corcept and a host of other companies are beginning to focus on health stories that grab less boldfaced treatment. They are turning to the rare-diseases category and to conditions that affect patient groups with populations smaller than some small towns.

Inducements to find and develop drugs for so few people include incentives that go beyond the seven years of patent status under the 1983 Orphan Drug Act. These include government grants that can help defray the costs of clinical trials. The awards range from $200,000 to $400,000 per year depending on the trial phase.

Recent developments suggest progress, albeit uneven. In 2011, 14 new drugs, new formulations and products with new manufacturers were approved that had garnered FDA orphan drug status (including 10 new NMEs and BLAs), a head-over-heels improvement from 2010's total of zero, and a 367% improvement over 2009, when just three such drugs were sanctioned. . .View Full Article

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