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The Active Ingredient features expert insights and commentary from leaders of ZS Associates' global pharmaceuticals practice.

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Take Your Orphan Drug Portfolio to the Next Level

Posted by Matt Gill on Mon, Oct 07, 2013

Pharmaceutical companies can no longer overlook the potential of orphan drug markets.

As pipelines contract and competition squeezes bottom lines, orphan drug markets—disease markets comprising less than 200,000 patients in the United States—have become an attractive means to expand portfolios.

There are many allures to orphan drug markets: Pharma companies entering orphan drug markets enjoy extended patent exclusivity and special tax benefits, while these drugs often fly under payers’ radar, making them less subject to reimbursement restrictions.

However, results haven’t always matched the potential in orphan drug markets. The "rules" for orphan drug commercialization are far different from those for treatments addressing a broad patient base.

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Topics: Matt Gill, orphan drugs

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