Drugs that are being tested but are not yet approved are called investigational (or experimental) drugs and patients can get access in two ways:
- They can participate in the drug testing process, known as a clinical trial,
- They can be given the drug as part of a special program, more formally known as expanded access.
In general, to obtain access to a drug that is in testing but has not yet been approved, a patient must meet the entrance criteria and volunteer to participate in a clinical study of the drug.
Sometimes, people with serious or immediately life-threatening illnesses cannot wait until a drug that could possibly help them stay alive is approved. In some of these cases, FDA authorizes the manufacturer to provide patients with access to a drug that is still being tested for safety and effectiveness but is not yet approved.
FDA does not create expanded access programs; these programs are created by the drug’s manufacturer, sometimes at the request of a doctor whose patient is in urgent need of the medication. If there is no established expanded access program in effect for which the patient qualifies, FDA can consider a request for expanded access on a case-by-case basis.
