The use of drugs under investigational new drug status is important because it allows manufacturers to generate data to determine safety and effectiveness in marketing. However, FDA has established programs to allow patients with an immediately life-threatening disease “early access” to new treatments. The FDA defines “immediately life-threatening” as a stage of a disease in which there is a reasonable likelihood that death will occur within a matter of months, or in which premature death is likely without early treatment. For example, advanced cases of AIDS and cancer are considered to be immediately life-threatening diseases.
Since patients who have exhausted standard therapeutic options may be willing to accept additional risks and potentially dangerous side effects from drug products still under study, these programs allow patients access to investigational drugs.
A patient’s health-care provider should contact the drug manufacturer for information about product availability for a specific patient if the health-care provider believes this treatment may be of benefit to their patient. However, FDA is not permitted to disclose any information regarding investigational drugs, or ask sponsors to provide investigational drugs to physicians.
