The short answer
Expanded access, or compassionate use, is a way for a person with a serious illness to try an unapproved treatment outside a clinical trial when no other options remain and they cannot join a trial.
Expanded access lets some patients use an unapproved treatment outside a trial.
It is also called compassionate use.
It is meant for serious conditions with no good alternatives.
It usually requires that the person cannot join a suitable trial.
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A path outside a trial
Expanded access is often called compassionate use. It is a way for a person with a serious illness to try a treatment that is not yet approved, without joining a clinical trial. It exists for situations where the usual options have run out.
Who it is for
Expanded access is generally meant for people with a serious or life-threatening condition. It applies when someone cannot take part in a suitable clinical trial, and when no satisfactory approved treatment is left to try. It is not a substitute for a trial when one is available.
Who must agree
Several parties have to line up. Your own licensed doctor must request it and be willing to oversee and monitor the treatment. The company that makes the investigational treatment must agree to supply it. It is not required to, and the FDA cannot make it. A company may say no, or may not have enough of the drug to go around. The FDA must authorize the use, and it clears the large majority of requests it receives. An institutional review board, the ethics panel that watches over research, also has to approve. You then have a consent talk about the possible risks and benefits before treatment starts.
How it differs from a trial
A clinical trial is a study. It is designed to answer research questions under a controlled plan. Expanded access is about giving a treatment to a patient who needs it. Collecting research data is not the main point. The FDA also weighs whether the likely benefit to you justifies the risk, and whether the use would get in the way of trials still under way. If you think it might apply to you, your oncologist is the person to start the conversation with.
Sources
Common questions
What is expanded access?
Expanded access, sometimes called compassionate use, is a pathway that allows a person with a serious illness to receive an investigational treatment outside a clinical trial when they have no comparable options.
Who qualifies?
It is generally for people with a serious or life-threatening condition who cannot take part in a clinical trial and have no satisfactory approved treatment available.
Who has to agree?
The treating doctor must request it, the company that makes the treatment must agree to provide it, and the FDA must authorize it.
Is it the same as a clinical trial?
No. A trial is a study designed to answer research questions. Expanded access is about providing treatment to a patient, not primarily about gathering research data.
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Written by: Cancer ExplainedSources last checked: 2026-07-14 what this meansLast updated: 2026-08-11Next planned review: 2027-01-14
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Cancer Explained does not originate medical claims. Every page restates guidance already published by the National Cancer Institute, the CDC, the USPSTF and the FDA, in plain language, with the source cited so you can check the original yourself. AI does the translating and organizing; automated checks test claims, citations, clarity and safety before anything publishes, and this is translation and navigation, not clinical judgment. Nothing here is personal medical advice, and no page can account for your particular situation.
Editorial status: Source checked — This page was written with AI assistance and then compared with the sources listed on it, looking for statements those sources do not back up. That check is not a sentence-by-sentence record and can miss errors. It is not a medical review, and it does not confirm the page is complete or right for your situation.
Human medical review: not completed. Pages here are not signed off by a clinician before they publish. That is not an oversight we are quietly working around: we restate published guidance and cite it, so the authority belongs to the source rather than to us, and every page names where its claims come from — you can verify us instead of trusting us. Where a volunteer clinician has reviewed a page, their name and credentials appear on it; where no name appears, no clinician has checked it. We are glad to have reviewers and are recruiting them, and we do not hold pages back waiting for one. Use this site to understand your situation and to ask better questions of the people treating you.
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