Principles and Practice of Clinical Research

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Principles and Practice of Clinical Research
Data and Safety Monitoring Boards
Frederick L. Ferris III, MD
December 7, 2004
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Data and Safety Monitoring Boards
Why?

• Participant Safety

• Policy Review

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Data and Safety Monitoring Boards
New Treatment for Lung Cancer

• Totally New Compound

• Possible Liver Toxicity in Animals

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Data and Safety Monitoring Boards
New Treatment for Lung Cancer
Ten Patients Enrolled 6 Months FU

• Placebo Group

Two Dead from Lung Cancer

• Treated Group

None Dead from Lung Cancer
Two Dead from Liver Failure
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Data and Safety Monitoring Boards
Who?

• Independent of Sponsor/Investigators

• Appointed by Sponsor

• Members Without Conflict of Interest

• Different Areas of Expertise

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Data and Safety Monitoring Boards
What? Study Monitoring

• Review Protocols and Procedures

• Review Study Design

• Monitor Ongoing Quality

• Monitor Patient Accrual and Drop-out

• Monitor Clinic and Patient Compliance

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Data and Safety Monitoring Boards
What? Data Monitoring

• Identify Major Response Variables

• Identify Possible Adverse Outcomes

• Develop Stopping Guidelines

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Sequential Study Monitoring
OBrian - Flemming
OBrian - Flemming
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Reject Null Hypothesis
Reject Null Hypothesis
4
3
2
Continue Study
Accept Null Hypothesis
1
Test Statistic
0
-1
Continue Study
-2
-3
Reject Null Hypothesis
-4
-5
40
20
60
80
0
Number of Events
9
Data and Safety Monitoring Boards
Study Outcomes

• Planned Termination

• Harm Early Termination

• Early Benefit Early Termination

• Futility Early Termination

• Study Extension with Changes