Justification for Use of a Placebo Or Sham Procedure

Lead Researcher: HS #

Study Title:

APPENDIX L

JUSTIFICATION FOR USE OF A PLACEBO OR SHAM PROCEDURE,

OR PLACEBO WASHOUT PERIOD

University of California, Irvine

Institutional Review Board

Please refer to the guidelines on the HRP Website on the use of Placebo.

The IRB must consider whether the use of placebo unfairly denies subjects in the placebo group medical benefit. The issue becomes more important when the use of placebo could involve the risk of irreversible harm or major discomfort. The IRB must assess potential subject harm (physical, psychological, social, etc.) and the scientific validity of the study and determine whether the use of a placebo is ethically appropriate. Your response to the following questions will help the IRB determine whether use of a placebo is appropriate.

USE OF PLACEBO OR SHAM PROCEDURE:

1. Does a proven standard treatment/therapy exist to treat the disease/condition being studied? Standard treatment/therapy may be a type of active treatment to treat the disease/condition; supportive care (e.g., treatment to alleviate symptoms but without specific activity to treat the disease/condition).

No Yes

Explain your answer:

2. Is the standard treatment/therapy considered to be effective?

No Yes

Explain your answer:

3. Provide compelling and scientifically sound methodological reasons for use of placebo or sham procedure (e.g., the toxicity of the therapy is such that patients commonly refuse it).

4. Explain whether standard therapy is given to mitigate permanent harms (e.g., psychological harm, disfigurement or other serious adverse sequelae) or whether it is given to treat symptoms that constitute inconvenience or discomfort only.

5. Does the disease/condition being treated have the potential to progress to a higher risk condition if not actively treated (i.e., if assigned to placebo)?

No Yes

Explain your answer:

6. Explain whether the natural fluctuation of the disease/condition is significant enough to necessitate the use of placebo or sham comparison to determine if the observed changes are due to treatment or natural history.

7. Explain whether it would be possible to predict the placebo or sham response rate in this study with a reasonable degree of accuracy.

8. Clarify whether subjects in the placebo group would be exposed to an increased risk of death, severe morbidity or disability, severe discomfort, or other long-term negative effects.

9. Explain how the study design will minimize risks to subjects:

10.  If vulnerable populations are included in the study, justify their participation and detail how subjects will be adequately protected.

USE OF PLACEBO WASHOUT PERIOD:

1. Provide compelling and scientifically sound methodological reasons for use of placebo-washout:

2.  Explain whether the disease/condition being treated has the potential to progress to a higher risk condition if not actively treated?

3. Indicate the duration of the washout period:

4. Describe the risks specific to the placebo-washout phase of the study:

5. Explain how the study design will minimize risks to subjects:

6. Describe the increased subject monitoring that will occur during the washout period and detail any rescue plans for subjects whose disease/condition worsens.

7. If vulnerable populations (children, pregnant women, cognitively impaired, etc.) are included in the study, please justify their participation. NOTE: Completion of a vulnerable population appendix is also required.