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  1. Advancing Regulatory Science

Qualitative techniques to define meaningful within-patient change in symptoms of advanced cancer patients

CERSI Collaborators: Molly Jeffery, PhD; Minji Lee, PhD, MS; Kathleen Yost, PhD; Amylou Dueck, PhD; Gita Thanarajasingam, MD; Gladys Asiedu, PhD

FDA Collaborators: Vishal Bhatnagar, MD; Paul Kluetz, MD; Erica Horodniceanu, MPH; Selena Daniels, PharmD, PhD; Elizabeth Duke, MD

Project Start Date: September 27, 2022

Regulatory Science Challenge

Patients receiving cancer treatment often experience changes in cancer symptoms or treatment side effects, such as fatigue, nausea, or pain. Questionnaires called “patient-reported outcome measures” are commonly used to collect information from patients about their symptoms and side effects throughout the course of treatment and even after treatment is completed. It is important for cancer care teams and regulators, such as the Food and Drug Administration, to understand how to interpret the information from these questionnaires. Specifically, they need to know whether the amount of change (improvement or worsening) in cancer symptoms or treatment side effects is meaningful to patients with cancer. With a better understanding of meaningful change, cancer care teams and regulators might be able to better evaluate the impact of different cancer treatments on their patients’ well-being and it could help cancer care teams to support patients more effectively during their treatment. Defining the concept of meaningful change and determining how much change is considered meaningful are challenging. The research in this area is limited; perspective of patients are rarely taken into consideration. Qualitative research can be used to obtain a deeper understanding of the patient experience by generating in-depth information about the experiences, perspectives, priorities, preferences, and feelings of patients, in their own words. This study will apply qualitative research methods to select groups of patients receiving cancer treatment to gain insight into the definition and measurement of meaningful change in cancer symptoms and treatment side effects.

Project Description and Goals

This study investigates how best to communicate the complex concept of meaningful change in cancer symptoms or treatment side effects to patients and how best to incorporate patients’ perspectives when establishing when a change becomes meaningful to them. We will use multiple qualitative methods (such as semi-structured interviews and focus groups) to develop and test our methods for eliciting patient perspectives for defining meaningful change in selected patient-reported outcome measures. Specifically, we will focus on fatigue severity, fatigue interference with daily activities, and the degree to which one is bothered by side effects of treatment.

Analyses of interview and focus group data will focus on patients’ interpretation of meaningful change and how it translates to the change in actual questionnaire score in selected patient-reported outcome measures. The outcome of this project will be to (1) define meaningful change from patient perspectives, (2) understand the amount of change in selected patient-reported outcome measures that patients consider meaningful, and (3) evaluate a new method for engaging patients in studying meaningful change. Different treatment options exist for patients living with prostate cancer, thus they are able to have a choice based on their perspective and preference. The goal of this research study is to assess patients’ perspectives on the benefits and risks associated with HIFU. Eligible prostate cancer patients were identified and recruited from the University of Maryland Medical Center and surveyed about their opinions on prostate ablation options. The survey tool was developed by the FDA research team based on a literature review, interviews with prostate cancer patients, and clinical and regulatory knowledge of HIFU. The information obtained from this work with patients may help inform the FDA’s decision making for new prostate ablation devices.

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