Compassionate use refers to the use of an unapproved drug to treat a patient, outside of a clinical trial setting, when no other viable treatment options are available. Compassionate use can also be referred to as expanded access, managed access, or named patient supply. This can be an important treatment option for certain patients as it provides access to potentially life-saving medicines, before they become commercially available.

Because this treatment option is not appropriate for all patients, the bullets below explain what conditions must be met. Compassionate use may be a viable option for a patient when all of the following apply:

  • The patient is suffering from a serious or life-threatening disease or condition. The patients considered for compassionate use are usually suffering from a rare disease, a rare form of cancer, or a debilitating or degenerative condition.
  • There is no satisfactory alternative therapy. If there is an alternative, the patient must have tried this therapy and their physician must have deemed it ineffective for that patient.
  • The patient cannot be enrolled into an active clinical trial. This could be because a patient does not live near to a clinical trial site, or it could be because the patient is ineligible for the clinical trial.
  • The potential patient benefit outweighs the potential risks of treatment. This must be determined by the treating physician and then verified by the drug manufacturer and, in most cases, the FDA.
  • Providing the investigational drug through compassionate use will not interfere with clinical trials that could support a medical product’s development or marketing approval.

If these conditions are met, a patient would be able to consider compassionate use. However, a patient cannot receive a drug through compassionate use without the consent and support of their treating physician. Furthermore, patients and physicians cannot receive a drug through compassionate use without the approval of the drug manufacturer. The drug manufacturer has the final say when it comes to providing compassionate use access to its products.