Orphan Drug — What It Means in Clinical Trials

Plain English Definition

An orphan drug is a medication developed to treat a rare disease (one affecting fewer than 200,000 people in the US). Orphan drug designation provides financial incentives to drug companies — including tax credits, reduced fees, and seven years of market exclusivity — to encourage development of treatments for conditions that would otherwise not be profitable to pursue.

Why It Matters

If you have a rare disease, orphan drug trials may be your best path to treatment. These designations encourage companies to invest in conditions that affect small numbers of people.

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