Compassionate Use
A pathway that allows seriously ill patients to access experimental treatments outside of clinical trials when no other options exist.
In Detail
Compassionate use, also known as expanded access for individual patients, is a regulatory pathway that allows seriously ill patients to receive experimental treatments that have not yet been approved by the FDA. This pathway is available when a patient has a serious or immediately life-threatening condition, no comparable or satisfactory alternative therapy is available, and the patient is unable to enroll in a clinical trial (due to geographic limitations, exclusion criteria, or the trial being closed to enrollment). The treating physician must determine that the potential benefit justifies the potential risks. To initiate compassionate use, the physician submits a request to the drug manufacturer, files an IND application with the FDA (which can be done using a streamlined form), and obtains IRB approval. The FDA approves the vast majority of compassionate use requests, historically over 99%, and can provide emergency authorization within 24 hours for life-threatening situations. Under the 2018 Right to Try Act, eligible patients with life-threatening conditions can also request experimental treatments directly from manufacturers without filing a full IND, though this pathway has been used less frequently than the traditional expanded access route. There are important considerations for patients: insurance may not cover the costs of compassionate use treatment, the treatment may have unknown side effects, and receiving an experimental drug through compassionate use does not guarantee it will work. Compassionate use is distinct from expanded access programs, which provide treatment to larger groups of patients simultaneously.
Frequently Asked Questions
What does "Compassionate Use" mean in clinical trials?
A pathway that allows seriously ill patients to access experimental treatments outside of clinical trials when no other options exist.
Why is "compassionate use" important for patients?
Understanding compassionate use helps patients and caregivers navigate clinical trial participation with confidence. It is part of the broader clinical research process that ensures treatments are safe and effective before reaching patients.
Related Terms
Expanded Access
A program that provides an investigational drug to a group of patients with serious conditions outside of a clinical trial, often while the drug is being reviewed for approval.
FDA Approval Process
The regulatory pathway through which the U.S. Food and Drug Administration evaluates and approves new drugs, biologics, and medical devices for public use.
Investigational New Drug (IND)
An application submitted to the FDA before a new drug can be tested in humans, containing preclinical data demonstrating safety and a plan for clinical trials.
Clinical Trial
A research study that tests a medical treatment, drug, device, or intervention in human volunteers to determine whether it is safe and effective.
this entity is one of the U.S. clinical trials and research registries concepts that recurs across this site. The definition above is the technical answer; the paragraphs below add the practical context for how the concept connects to the the NIH ClinicalTrials.gov registry data behind every per-entity page on the site.
In the the NIH ClinicalTrials.gov registry data, this concept shapes one or more of the fields that drive the per-entity grades and rankings on this site. The methodology page describes which fields feed into which output; this glossary entry documents the underlying term.
Source: ClinicalTrials.gov, 2026.