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Trial Design

Compassionate Use — What It Means in Clinical Trials

Plain English Definition

Compassionate use is another name for expanded access. It allows seriously ill patients to receive experimental treatments that have not yet been approved, when they have no other treatment options remaining. The treating doctor must apply to the drug manufacturer and the relevant regulatory authority.

Why It Matters

This term often comes up when patients are told there are no remaining approved treatments. If your doctor mentions compassionate use, it means they are exploring every possible option for your care.

Example

You might hear: "Your doctor has applied for compassionate use of Drug Z." This means a formal request has been made to access a drug that is still being tested.

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