Longitudinal analysis & visualization of patient-reported physical function & symptom data
CERSI Collaborators: Amylou Dueck, PhD; Gita Thanarajasingam, MD; Gina Mazza PhD; Minji Lee, PhD; Molly Jeffrey, PhD; Joseph Ross, MD, MHS
FDA Collaborators: Vishal Bhatnagar, MD; Ting-Yu Chen, PhD; Mallorie Fiero, PhD; Paul Kluetz, MD; Meena Murugappan, PharmD, PhD
Project Start Date: October 2022
Project End Date: 08/09/2024
Regulatory Science Challenge
The FDA provided guidelines on what information patients should report during cancer treatments, including how well they can perform daily activities that require physical effort. There hasn't been an agreement on the best way to show this information clearly.
Physical function and side effects of treatment as reported by patients through surveys are important in understanding how well patients tolerate cancer treatment. As such, the FDA Oncology Center of Excellence (OCE) recommended that physical function and side effects be measured by patient surveys during most or all cancer treatment clinical trials. As high-quality information is generated from these trials, improved approaches to analyzing and displaying survey data on physical function and side effects in a way that is valuable to patients, clinicians and decision makers at the FDA are needed. Additionally, little is known about how physical function and side effects change over time in patients with rare cancers, leaving these patients with little to no information about what to expect from their treatments.
This study aimed to better understand physical function and side effects in patients with rare cancers and develop improved ways to communicate patient-reported physical function and side effects for patients, clinicians, and decision makers at the FDA. The content of this project aligns with work by colleagues in the FDA OCE, including the FDA’s Project Patient Voice (PPV), a pilot online platform for patients and clinicians to visualize survey data from cancer trials.
Project Description and Goals
The goals of this project were to create easy-to-understand graphs and charts that show how patients' physical abilities change over time while they are being treated for different types of blood cancers. Information from three clinical trials was used to generate the graphs and charts. We gathered feedback from patients, doctors, and regulators to make sure these visualizations were helpful and clear.
In the study, researchers developed tools to statistically analyze and visualize patient survey data on physical function and side effects from multiple trials in rare cancers including amyloidosis, myeloproliferative neoplasms, carcinoid, sarcoma, and esophageal cancer.
This study aimed to (1) develop novel graphics of physical function and side effect data collected by patient surveys from rare cancer clinical trials and (2) to obtain initial feedback from patient advocates, clinicians/clinical investigators, and decision makers at the FDA on these visualizations. The ultimate goals of this project were to refine multiple depictions over time of physical function and side effect data from patient surveys from rare cancer trials, develop graphical representations that may complement those in FDA’s Project Patient Voice, and obtain preliminary feedback from patients, clinicians, and decision makers at the FDA on their interpretability and ability to reflect information on the tolerability of a cancer treatment.
- US Food and Drug Administration (FDA). Core Patient-Reported Outcomes in Cancer Clinical Trials: Final Guidance for Industry (October 2024) [Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/core-patient-reported-outcomes-cancer-clinical-trials accessed December 17, 2024].
- US Food and Drug Administration (FDA). FDA Project Patient Voice [Available from: https://www.fda.gov/about-fda/oncology-center-excellence/project-patient-voice accessed February 23, 2021].
Research Outcomes
Research Outcome 1 IV. Inform Regulatory Decision Making:
Provide supporting information for outcome 1:
We made new charts that show how patients' physical abilities and symptoms change over time from several clinical trials, including rare cancers. We received feedback from patient advocates, doctors at Mayo Clinic and Yale, and the FDA to see if these charts were easy to understand and useful.
Research Outcome 2 II. Disseminate Scientific Knowledge:
Provide supporting documentation for outcome 2:
We showed our results at an international meeting of quality-of-life researchers. We also wrote a detailed report about our project and sent it to a medical journal to be reviewed by experts.
Our paper, "Longitudinal graphics of patient-reported physical function in patients treated for hematologic malignancies," has been published online by BMC Medical Research Methodology (Open Access).