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UM Policies & Procedures - HSRO/IRB
1. Preface
2. Background Topics
3. Authorities and Responsibilities
4. Conflict of Interest
5. Institutional Review Boards
6. IRB Meetings
7. General Principles for IRB Reviews
8. Definition of IRB Review Types
9. IRB Review of Initial Studies
10. IRB Review of Continuing Studies
11. Amendments
12. Closing Studies and Final Reports
13. Suspension, Termination and Administrative Closure of IRB Approved Research
14. Unanticipated Problems and Adverse Events
15. Study Violations
16. Compliance Audits
17. Data Safety Monitoring Boards
18. Ancillary Committees
19. External IRB's
20. Policies Specific to Certain Types of Research
21. Informed Consent
22. Participant Recruitment Methods, Advertising Materials and Recruitment-Relevant Payment Arrangements
23. Vulnerable Populations
24. Privacy, Security, Confidentiality and HIPAA
25. Subcontracts/Agreements for UM-Initiated Studies that Engage or Involve non-UM Institutions or Investigators
26. International Research
27. Emergency Use
28. Record Retention
Other Policies
UChart Forms & Guidance
HSRO Forms & Documents
University Fees
Ancillary Committee Information
eProst User Guide
Ethics, Federal Regulations & Guidance
Western IRB (WIRB)
About the HSRO

Basic Folder Information

20.3 Off-Label Use of Marketed Drugs, Biologics or Medical Devices

Approval Date

Review Responsibility:

IRB Policy and Procedure Committee

Current Approval Date:

February 22, 2007

20.3 Off-Label Use of Marketed Drugs, Biologics or Medical Devices

Questions often arise as to whether the "off-label” use of marketed drugs, biologics and medical devices constitutes research which requires IRB approval. 

Physicians may sometimes use legally available drugs, biologics or devices for other than their FDA-approved indications when they determine that such "off-label" use is in the best interests of the patient.  "Off-label" use should be consistent with good medical practice and the best interests of the patients and should be the based on firm scientific rationale and sound medical evidence.  Physicians should be well informed about the product and maintain records of the product's use and effects. 

It is sometimes difficult to determine whether "off-label" use of drugs, biologics or devices is for therapeutic or research purposes or both because there is often little evidence of the safety and efficacy of the drugs, biologics or medical devices for the proposed use.  The key to determining whether the "off-label" use is therapeutic or research is the physician's intent.  If the intent is investigational (i.e. the seeking of generalizable knowledge of the drug, biologic or device's safety or efficacy or if the "off-label" use is intended to prove or disprove a hypothesis or to answer a question, then the use should be considered research requiring the submission of an IND (Investigational New Drug Application) or IDE (Investigational Device Exemption) from the FDA (unless the FDA specifies in writing that an IND or IDE is not required) and IRB approval for the study [c.f. 21 CFR 312.3(b)].  

Submission of an IND or IDE and IRB review/approval are not required when the 'off-label' use of a marketed drug, biologic or device is not intended for research but rather is intended only for the practice of medicine or when all of the following six conditions for the "off-label" use are met [21 CFR 312.2(b)(1)]:

1)  It is not intended to be reported to the FDA in support of a new indication for  use or to support any other significant change in the labeling for the drug 

2)  It is not intended to support a significant change in the advertising for the product

3)  It does not involve a route of administration or dosage level, use in a subject population, or other factor that significantly increases the risks (or decreases the acceptability of the risks) associated with the use of the drug product

4)  It is conducted in compliance with the requirements for IRB review and informed consent [21 CFR parts 56 and 50, respectively]

5)  It is conducted in compliance with the requirements concerning the promotion and sale of drugs [21 CFR 312.7]

6)  It does not intend to invoke 21 CFR 50.24 which is the regulation on waiving informed consent requirements for emergency research [NOTE – Regulations on the permissible, one-time emergency use of a test article with notification to the IRB within 5 days are 21 CFR 56.102(d) and 21 CFR 56.104(c)]

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