<< Return to IRB Policies and Forms



IRB Guidelines for Investigational New Drug Studies

Cooperative Research


DEPT: IRB POLICY #: 8012.24



The use of investigational new drugs (INDs) shall be in accordance with state and federal laws and regulations and other policies adopted by Cottage Health System.



Cottage Health System Institutional Review Board (CHS IRB) oversees the use of all investigational agents and complies with FDA regulations regarding such use.  Except where special exceptions have been made, the use of an investigational drug may only take place under an IRB-approved research trial.  The Department of Pharmacy follows written policy regarding the proper dispensing of such products within the institution.



Investigational New Drug -  A new drug or biological drug that is used in a clinical investigation.  The term also includes a biological product that is used in vitro for diagnostic purposes.  The terms "investigational drug" and "investigational new drug" are deemed to be synonymous (21 CFR 312.3).

Orphan Drug - A drug intended for use in a rare disease or condition as defined in section 526 of the Federal Food Drug and Cosmetic Act.


This policy currently reflects:

  • 21 CFR 312 - Investigational New Drug Application
  • 21 CFR 316 - Orphan Drug Regulations
  • 21 CFR 56 - Institutional Review Boards
  • Guidance for Industry, Investigators, and Reviewers:  Exploratory IND Studies, Food and Drug Administration Center for Drug Evaluation and Research (CDER), January 2006
  • SBCH Pharmacy Policy #12.60 - Investigational Medications



General Guidelines

  1. Investigators wishing to conduct research using an investigational new drug, may not do so until a research application has been reviewed and fully approved by the IRB.
  2. The research application must include proof of the FDA's approval of the IND.  Acceptable documentation includes:
    a.   The FDA approval letter; or
    b.   Justification from the sponsor can that the agent is exempt from such a requirement.
  3. Investigational new drug trials almost exclusively require Full Board IRB review and approval.
  4. As with any other research conducted under the purview of the IRB, the investigation of a new drug requires IRB approval to continue via the continuing review requirement of 365 days or less.  IRB continuing reviews of IND studies require Full Board review in most cases, unless special exceptions apply.

Emergency Use of a Test Article

Under the Code of Federal Regulations Title 21 Part 56.104(c) use of investigational agents for emergency situations is allowed under the following circumstances:
a.   A patient is faced with a life-threatening situation; and
b.   All treatment methods have been exhausted; and
c.   No additional approved treatment methods are available; and
d.   Due to the nature of the situation, there is insufficient time available to seek IRB approval prior to the use of the investigational drug.
Complete instructions, as well as the application for the Emergency Use of a Test Article are available from the IRB department.  See also IRB policy #8012.08 - Emergency Use of a Test Article


Compassionate Use and Expanded Access Drugs
Generally, the unusual step of authorizing use of an investigational drug or device for what is primarily a treatment purpose is warranted only for patients with serious diseases or conditions who are without satisfactory therapeutic alternatives.  If the requirement for "emergency use" are not met, the patient may be enrolled under a compassionate use protocol, if allowed by the manufacturer.


As with a research protocol, compassionate use cases must be reviewed and approved by a majority of IRB members at a convened meeting before the subject is permitted to enter the study.  Although these protocols often are not viewed as research, federal regulators still consider these patients to be research subjects and such distribution must be reviewed by local IRBs.


In addition to Full Board approval, compassionate use situations require approval from the Director of Pharmacy when the investigational agent will be a drug handled by a CHS pharmacy.


Complete instructions, as well as the application for the Compassionate Use of a Test Article are available from the IRB department.  See also IRB policy #8012.19 - Guidelines for the Compassionate Use of Investigational Agents.


Exploratory IND Studies

The phrase Exploratory IND study is intended to describe a clinical trial that:

  • Is conducted early in Phase 1;
  • Involves very limited human exposure; and
  • Has no therapeutic or diagnostic intent (e.g., screening studies, microdose studies).


Exploratory IND studies are conducted prior to the traditional dose escalation, safety, and tolerance studies that ordinarily initiate a clinical drug development program.  The duration of dosing in an exploratory IND study is expected to be limited (e.g.: 7 days).


Specific information surrounding exploratory IND studies can be obtained from Guidance for Industry, Investigators, and Reviewers:  Exploratory IND Studies, Food and Drug Administration Center for Drug Evaluation and Research (CDER), January 2006, or by calling the FDA's Center for Drug Evaluation and Research.


Orphan Drugs
An orphan-drug designation requires:

  • That the number of people affected by the disease or condition for which the drug product is indicated is fewer than 200,000 persons ("prevalence" is defined as the number of persons in the United States who have been diagnosed as having the disease or condition at the time of the submission of the request for orphan-drug designation); or
  • There is no reasonable expectation that the sales of the drug will be sufficient to offset the costs of developing the drug for the U.S. market and the costs of making the drug available in the United States.

In order to designate a product as an orphan drug, the inventor/manufacturer must be able establish a medically plausible basis for expecting the drug to be effective in the rare disease. This is best supported by clinical trials of the drug in the rare disease or condition.  However, in absence of human data, the application for orphan drug designation may be satisfactorily supported with compelling preclinical data that uses the active moiety of the proposed orphan drug in a relevant animal model for the rare human disease. Pharmtox data, which describes the safety of the drug in animals, does not provide efficacy data, so is not useful in supporting the scientific rationale section of the orphan drug designation.


IRB Oversight:

  • Human clinical trials being conducted in support of the orphan drug designation must take place under the purview of an IRB in the same manner as other clinical trial investigations.
  • The treatment use of orphan drugs which have not yet been designated for marketing by the FDA requires prospective IRB review and approval and informed consent (21 CFR 316.40 and 312.34).




RECOMMENDED BY: Leilani Price DATE: 11/11


APPROVED BY: 5/30/02 DATE: ..

DATE REVISED: 3/06, 5/11