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Biomarker-based methods for determining noncompliance in a prevention trial
Donna K Pauler1, Kathryn B Gower, Phyllis J Goodman
1Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA. donnap@crab.org
Controlled Clinical Trials
|December 31, 2002
Summary
Simple biomarker monitoring rules effectively track participant compliance in large prevention trials like the Prostate Cancer Prevention Trial (PCPT). These methods are as effective as complex ones for ensuring adherence to treatments such as finasteride.
Area of Science:
- Clinical Trials
- Pharmacology
- Biomarker Research
Background:
- The Prostate Cancer Prevention Trial (PCPT) evaluated finasteride for prostate cancer prevention in 18,000 men.
- Participant compliance is crucial in long-term prevention trials.
- Biomarkers like 5 alpha-dihydrotestosterone (DHT) are explored for monitoring compliance.
Purpose of the Study:
- To develop and compare biomarker-based rules for monitoring compliance in prevention trials.
- To assess the effectiveness of simple versus complex compliance monitoring rules.
- To utilize DHT levels from the PCPT substudy as a case study.
Main Methods:
- Developed three biomarker-based compliance monitoring rules.
- Compared rules using receiver operating characteristic (ROC) curves.
- Applied methods to DHT levels in the PCPT substudy (pill counts as gold standard) and a simulation study.
Main Results:
- Simple absolute threshold monitoring rules demonstrated performance comparable to more complex rules.
- Complex rules utilizing the entire biomarker history did not offer significant advantages over simpler methods.
- Biomarker monitoring, specifically DHT, can effectively track compliance in large-scale trials.
Conclusions:
- Simple biomarker-based monitoring rules are efficient and effective for assessing compliance in prevention trials.
- Biomarker monitoring provides a valuable tool for ensuring adherence and data integrity in clinical research.
- Absolute threshold rules offer a practical approach to compliance monitoring, simplifying trial management.