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In 1983, the Orphan Drug Act was passed to give drug makers incentives to create medicines for rare diseases, which are defined as maladies that affect fewer than 200,000 people. The incentives include tax credits and seven years of marketing exclusivity. Since then, more than 400 orphan drugs have been approved by the Food and Drug Administration. Last year, though, 41 percent of all FDA approvals were for orphan drugs. And sales of orphan medicines, which carry high price tags, are forecast this year to total $107 billion.

In a new paper in the American Journal of Clinical Oncology, a team of researchers argues that drug makers are exploiting loopholes that allow them to widen the market for such drugs and distorting the original purpose of the law. We spoke with Martin Makary, a cancer surgeon and professor of health policy at Johns Hopkins School of Medicine, who is one of the authors, about the need for reform. This is an edited version of the conversation …


Pharmalot: What’s the problem with the Orphan Drug Act?

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