Lessons

Terminology and Overview

Clinical Uses of Biomarkers
Learning Objectives:
  1. Understand the different ways biomarkers are used in oncology clinical practice, including for diagnosis, prognosis, and therapy selection.
  2. Understand that biomarkers can be based on single measurements or summaries of multiple measurements.
The Use of Biomarkers in Clinical Trials
Learning Objectives:
  1. Understand the different ways biomarkers are used in clinical trials, including determining eligibility, stratification factors, PK/PD outcomes, and correlative aims.
  2. Understand the differences between integral, integrated, and exploratory.
  3. Understand how the level of evidence and background information about the assay depends on its use.
Assay terminology
Learning Objectives:
  1. Understand the different types of assay inputs, e.g. type and quantity of specimens, reagents, and SOP.
  2. Understand what an SOP is and why it is important.
  3. Understand the different types of assay outputs and how they are translated into clinically useful biomarkers.
Lesson Template
Learning Objectives:
  1. This is the first objective.
  2. Sentence number two.
  3. Sentence number 3.

Statistical Issues in Analytical Performance Assessment

Identifying and Describing Different Sources of Variation
Learning Objectives:
  1. Understand the differences between pre-analytical, analytical, and biological variation.
  2. Understand statistical properties of the variance, and its decomposition into variance components.
  3. Understand how to identify what components are estimable in a given study.
Describing and comparing measures of location
Learning Objectives:
  1. Understand what the term accuracy may mean with a continuous or dichotomous biomarker.
  2. Understand the statistical properties of bias when there is a reference value.
Statistical issues in design and reporting analytical validation studies
Learning Objectives:
  1. Understand when to report the coefficient of variation and how to interpret it.
  2. Sample size planning for assay validation, including negative and positive controls.
  3. Understand how to describe a study when reporting statistics, including what replicates were performed and how.

Statistical Issues in Biomarker-Based Clinical Trials

Enrichment Designs
Learning Objectives:
  1. Understand the levels of evidence needed to justify an enrichment design.
  2. Understand the limitations of the end results from such a design.
  3. Other pitfalls, including the impact of false positive/negative rates, and partial enrichment.
Integrated Correlative Aims
Learning Objectives:
  1. Understand the planning process for assessing prognostic biomarkers.
  2. Understand the planning process for assessing predictive biomarkers, including when the treatment is efficacious overall and not.
  3. Understand the signature development process.
  4. Understand the expectations regarding the analysis and statistical plans.
Other
Learning Objectives:
  1. Stratification factors, and when imbalance is a concern.
  2. PK/PD outcomes.
  3. Exploratory correlative aims.