Compassionate Use

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.