Treatment Investigational New Drug

Treatment Investigational New Drugs (Federal Register, May 22, 1987) are used to make promising new drugs available to desperately ill patients as early in the drug development process as possible. FDA will permit an investigational drug to be used under a treatment IND if there is preliminary evidence of drug efficacy and the drug is intended to treat a serious or life-threatening disease, or if there is no comparable alternative drug or therapy available to treat that stage of the disease in the intended patient population. In addition, these patients are not eligible to be in the definitive clinical trials, which must be well underway, if not almost finished.

Examples of such tissues are bone, skin, corneas, ligaments, tendons, dura mater, heart valves, hematopoietic stem/progenitor cells derived from peripheral and cord blood, oocytes and semen.

An immediately life-threatening disease means 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, herpes simplex encephalitis, and subarachnoid hemorrhage are all considered to be immediately life-threatening diseases. Treatment INDs are made available to patients before general marketing begins, typically during Phase 3 studies. Treatment INDs also allow FDA to obtain additional data on the drug's safety and effectiveness.