Project Title: Evaluation of a Global Item for Side Effect Bother
External Institution Name: Northwestern University and University of Tasmania
External Collaborators: Co-Principal Investigators: John Devin Peipert, PhD, Northwestern University, Jessica Roydhouse, PhD, Menzies Institute for Medical Research, University of Tasmania
FDA Collaborators: Vishal Bhatnagar, MD Ting-Yu Jeff Chen, PhD, Mallorie Fiero, PhD, Erica Horodniceanu, MPH, Paul Kluetz, MD
Project Start Date: October 2021
Regulatory Science Challenge
Cancer treatments have many side effects. Reports of these side effects are important for determining if these treatments are safe. However, even treatments determined to be safe can have side effects that negatively impact patients and can make it hard to continue with treatment. Understanding how side effects impact patients requires information directly from patients to complement clinician reported side effects in cancer trials.
Clinical trials can collect information about side effects using patient-reported outcome (PRO) questionnaires. One example of a questionnaire item that can be asked to patients in a cancer trial is “I am bothered by side effects of treatment”, with five response options from “Not at all” to “very much”. This single item may be helpful in capturing the overall side effect impact on patients, and the FDA considers this as valuable information when evaluating the benefits and risks of cancer drugs. Improved understanding of the item’s relationship to how patients feel and function can guide use of this item in future cancer trials. This research will help improve the use and interpretation of this core outcome in cancer trials.
Project Description and Goals
This study aims to find out if a single patient-reported item is suitable for understanding the overall side effect impact on patients in cancer trials. Because this item has been included as part of a longer questionnaire in many trials, we will use data from trials that have already been conducted. We will carry out several statistical analyses to achieve our aims.
The study will address four related research questions: First, we will look at how this item relates to patients’ functioning (e.g., ability to walk long distances). Second, we will look at how this item relates to the symptoms that patients report (e.g., diarrhea). Third, we will look at how this item relates to the side effects that clinicians report that patients experience in trials. Fourth, we will look at how to analyze this item in future cancer trials.