[Code of Federal Regulations]
[Title 21, Volume 5]
[Revised as of April 1, 2008]
From the U.S. Government Printing Office via GPO Access
[CITE: 21CFR312.34]

[Page 64-65]
 
                        TITLE 21--FOOD AND DRUGS
 
CHAPTER I--FOOD AND DRUG ADMINISTRATION, DEPARTMENT OF 
 
PART 312_INVESTIGATIONAL NEW DRUG APPLICATION--Table of Contents
 
          Subpart B_Investigational New Drug Application (IND)
 
Sec. 312.34  Treatment use of an investigational new drug.

    (a) General. A drug that is not approved for marketing may be under 
clinical investigation for a serious or immediately life-threatening 
disease condition in patients for whom no comparable or satisfactory 
alternative drug or other therapy is available. During the clinical 
investigation of the drug, it may be appropriate to use the drug in the 
treatment of patients not in the clinical trials, in accordance with a 
treatment protocol or treatment IND. The purpose of this section is to 
facilitate the availability of promising new drugs to desperately ill 
patients as early in the drug development process as possible, before 
general marketing begins, and to obtain additional data on the drug's 
safety and effectiveness. In the case of a serious disease, a drug 
ordinarily may be made available for treatment use under this section 
during Phase 3 investigations or after all clinical trials have been 
completed; however, in appropriate circumstances, a drug may be made 
available for treatment use during Phase 2. In the case of an 
immediately life-threatening disease, a drug may be made available for 
treatment use under this section earlier than Phase 3, but ordinarily 
not earlier than Phase 2. For purposes of this section, the ``treatment 
use'' of a drug includes the use of a drug for diagnostic purposes. If a 
protocol for an investigational drug meets the criteria of this section, 
the protocol is to be submitted as a treatment protocol under the 
provisions of this section.
    (b) Criteria. (1) FDA shall permit an investigational drug to be 
used for a treatment use under a treatment protocol or treatment IND if:
    (i) The drug is intended to treat a serious or immediately life-
threatening disease;
    (ii) There is no comparable or satisfactory alternative drug or 
other therapy available to treat that stage of the disease in the 
intended patient population;
    (iii) The drug is under investigation in a controlled clinical trial 
under an IND in effect for the trial, or all clinical trials have been 
completed; and
    (iv) The sponsor of the controlled clinical trial is actively 
pursuing marketing approval of the investigational drug with due 
diligence.
    (2) Serious disease. For a drug intended to treat a serious disease, 
the Commissioner may deny a request for treatment use under a treatment 
protocol or treatment IND if there is insufficient evidence of safety 
and effectiveness to support such use.
    (3) Immediately life-threatening disease. (i) For a drug intended to 
treat an immediately life-threatening disease, the Commissioner may deny 
a request for treatment use of an investigational drug under a treatment 
protocol or treatment IND if the available scientific evidence, taken as 
a whole, fails to provide a reasonable basis for concluding that the 
drug:
    (A) May be effective for its intended use in its intended patient 
population; or
    (B) Would not expose the patients to whom the drug is to be 
administered to an unreasonable and significant additional risk of 
illness or injury.
    (ii) For the purpose of this section, 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.
    (c) Safeguards. Treatment use of an investigational drug is 
conditioned on the sponsor and investigators complying with the 
safeguards of the IND process, including the regulations governing 
informed consent (21 CFR part 50) and institutional review boards (21 
CFR part 56) and the applicable provisions of part 312, including 
distribution of the drug through qualified experts, maintenance of 
adequate manufacturing facilities, and submission of IND safety reports.
    (d) Clinical hold. FDA may place on clinical hold a proposed or 
ongoing

[[Page 65]]

treatment protocol or treatment IND in accordance with Sec. 312.42.

[52 FR 19476, May 22, 1987, as amended at 57 FR 13248, Apr. 15, 1992]