Advanced Superiority, Non-inferiority, or Equivalence Trials

Why Should You Attend:

This advanced webinar is directed toward professionals who want to understand the mechanics involved in design, execution, and analysis of alternate clinical trial designs - Superiority, Non-inferiority, and Equivalence Trials. The presentation will continue to address concerns of those on the side of new drug/device developments that may have safety, ease of administration, or economic or other advantages over current treatment. A discussion of the role of these non-traditional clinical trial designs for indication designation and labeling is included. For those who need to intelligently evaluate clinical study design and outcome, for business, regulatory, or evidence-based medical decisions, this is a discussion about how to evaluate specific design and biostatistical applications within IND/IDE reports and published studies.

The nuts and bolts of study design and analysis for these approaches center on the specific research question, target populations, margins of equivalence or non-inferiority, sample size adjustments, hypothesis testing, and statistical analysis. In this presentation each of these areas will be addressed, first through an examination of the critical assumptions and options available within each category, followed by practical examples and case studies. Techniques for matching the “research question” to the hypothesis tests and statistical evaluation will be demonstrated.

A discussion of target population considerations as they change throughout the drug or device development plan and the relationship between choice of target population and designation of equivalence and non-inferiority margins will be included. Impact on sample-size requirements and simple methods for calculating effect sizes as sample-size decisions change will be explained. In each area, concrete examples or case studies will be used to connect theory to action and outcome regardless of participant level of statistical expertise. Useful references to online sample and effect-size calculators and how to use them appropriately will be included. Finally, the implications for regulatory compliance and peer-review of these study designs will be discussed.

Areas Covered in the Webinar:

• General review of NI, equivalence, and superiority study objectives and designs.
• Distinguishing among clinical questions, research questions, and hypothesis tests.
• Making sure the clinical question, research question, and hypothesis test are in agreement with the purpose of the study.
• Identifying appropriate resources and calculating a meaningful interval of equivalence or acceptable “margin” of inferiority.
• Effect sizes and how they are used in determining hypothesis margins and study sample size.
• Examples and case studies in which these principles are demonstrated.
• FDA guidance and CONSORT considerations.

Learning Objectives:

• Participants will understand the roles of and be able to calculate simple effect sizes and required sample sizes for basic study designs.
• Participants will have references and resources to guide them in determination of margins of equivalence and non-inferiority.
• Participants will achieve a higher level of confidence in their decision making for clinical trial design.