Lectures and seminars Sfo Epi Seminar in Biostatistics: Using EHR to Extend Understanding of Treatment Effectiveness Beyond Clinical Trials

Speakers:

Professor Rebecca Hubbard PhD, Division of Biostatistics, Perelman School of Medicine, University of Pennsylvania and

Dr. Ronac Mamtani MD, Assistant Professor, Division of Hematology/Oncology, Abramson Cancer Center, Perelman School of Medicine, University of Pennsylvania 

Abstract:

Randomized clinical trials (RCTs) are considered the gold standard for evaluating efficacy of novel treatments but suffer from limitations. Some treatments cannot be ethically or feasibly allocated via random assignment. Moreover, vulnerable populations are under-represented in RCTs, raising concerns about equity and the external validity of results. Clinical trial treatment settings may not reflect care and outcomes as they are experienced in routine practice. Real-world data (RWD) sources, including electronic health records (EHR), have the potential to address many of these concerns. However, limitations of RWD necessitate careful attention to study design and application of appropriate statistical methods. In this talk, we will discuss sources of RWD, their strengths and limitations, and their potential to supplement RCT evidence on treatment efficacy. We present two novel statistical developments aimed at extending evidence on treatment efficacy beyond clinical trials: (1) a method for addressing data quality issues in EHR data to obtain valid treatment effectiveness estimates in the presence of differential outcome misclassification without requiring specification of the measurement error models and (2) an approach to combining clinical trial and EHR-derived effectiveness estimates. The overarching objective of this presentation is to introduce data sources, study design considerations, and statistical challenges arising in the analysis of RWD. Through judicious application of these methods RWD have the potential to aid clinical decision making through the creation of evidence on treatment effectiveness that would not be possible using RCTs alone.

Zoom Link: https://ki-se.zoom.us/j/65923678214

*Webinar format: unless you are on the panel you will be muted and with no video. Participants can raise their hands during the Q&A to be recognized to ask questions.

If you have any questions, please contact Erin Gabriel.