In rare circumstances, patients who meet certain conditions may have access to these highly restricted drugs either under the Expanded Access/Compassionate program or with the Single Patient Access.
Expanded access: Patients not able to enroll in a clinical trial, such as Phase III, may receive the drug from the sponsoring drug company. The FDA generally approves these drugs under the Early Access Program as it least (somewhat) has shown to be effective for some people. It is however up to the discretion of the drug company to supply the drug.
Single patient access
Patients who don’t qualify for either clinical trials or an expanded access program (if one exists) may be able to get the unapproved new drug by applying for single patient access.
In this case, the patient’s doctor must be in agreement and first inquire with the drug company if the drug can be used for the patient and request the drug company to supply the drug. If the company agrees, the patient’s doctor requests the drug company to ask the FDA to approve the drug for use by this one patient.
The FDA requires the doctor to send information about the patient, why the request is being made, the proposed treatment plan, and a signed informed consent from the patient.