Description: Comparative effectiveness research (CER) is increasingly important for generating real-world evidence to inform clinicians, patients, regulators, and other decision makers. The Prescription Drug User Fee Act reauthorization (PDUFA VI) and the 21st Century Cures Act in the US and adaptive pathways in the EU all have implications for the use of real-world evidence to inform regulatory decision making. This course will describe CER methods for generating evidence using real-world data, focusing specifically on observed biases and investigator errors that are avoidable in CER. The course will include a tutorial on creating design diagrams, which can assist in identifying biases and errors in CER studies. Participants will apply the design diagram to a real-world CER study.
Objectives: Objective 1: Understand biases that commonly occur in comparative effectiveness evaluations and strategies for overcoming these biases Objective 2: Critically apply a design diagram to a published CER case study
Outline: Presentation 1: Principles and thoughts on comparative effectiveness research of medications (Sebastian Schneeweiss): This lecture will provide an overview of pharmacoepidemiologic approaches to CER. Special attention will be given to challenges and solutions facing the analysis of observational real-world data.
Presentation 2: Design diagram tutorial (Shirley Wang): This session will introduce and walk through the application of a design diagram template to visually represent a CER study. Such diagrams readily convey the fundamental elements of a CER study design and can assist with diagnosing biases and errors in CER studies.
Participants will be asked to read a CER case study article (Patorno E, et al. BMJ 2018;360:k119. doi: 10.1136/bmj.k119) after watching the pre-recorded sessions and to create a design diagram corresponding to the study. Participants will have the opportunity to submit questions and discussion points in advance of the live discussion. During the live discussion, course faculty will walk through the design diagram for the case study and address questions raised by participants.