Statistical Considerations for Premarketing Risk Assessment

May 16, 2024

About This Course

This course will:

• Describe important statistical considerations in the premarketing assessment of drug safety. The safety profile of a drug evolves over time through gained experience and increased exposure. The use of diligent planning for safety and rigorous assessment of maturing safety data optimizes the ability to characterize the safety profile of a drug
• Cover the importance of planning for the safety assessment of a drug. A focus will be on planning to assess key risks in confirmatory trials to improve the quality and reliability of the safety data collected for these risks. Such a task requires clear specification of the key risks, operational definitions of the risks and plans to ascertain them, and other trial design and conduct considerations to facilitate their assessment at trial completion
• Address statistical considerations in the analysis of safety data, primarily adverse event data. Analysis of adverse outcomes is not a simple calculation of crude proportions of the number of participants experiencing the event. Rather, it requires careful consideration about the approach to analysis, including topics such as handling of treatment discontinuation, using data from multiple trials, defining summary measures of incidence, and choosing statistical methods to estimate the incidence and corresponding uncertainty

Intended Audience

• Pharmaceutical regulatory scientists, clinical reviewers, medical professionals, data scientists, statisticians, statistical programmers, medical writers working on clinical trial safety analyses for new drug marketing applications
• We will not discuss trial data for generic drug applications

Topics Covered

• How trial design can enhance the assessment of safety data
• The importance of tailoring the analysis of safety to align with trial design
• The use of appropriate statistical analysis approaches, including topics such as the choice of metric for estimating risk, handling treatment discontinuation, and integrated analyses

Learning Objectives

• Identify how trial design can enhance the assessment of safety data
• Cite the importance of tailoring the analysis of safety to align with trial design
• Explain appropriate analysis approaches to assess causal relationships between drug and adverse outcomes

Resources

• FDA Guidance Benefit-Risk Assessment for New Drug and Biological Products (October 2023)
• FDA Guidance Premarketing Risk Assessment (March 2005)
• ICH Guidance E1A The Extent of Population Exposure to Assess Clinical Safety: For Drugs Intended for Long-term Treatment of Non-Life-Threatening Conditions (March 1995)
• ICH Guidance E9 Statistical Principles for Clinical Trials (September 1998)

visit the FDA.gov webpage