CERSI Collaborators: Amylou Dueck, PhD; Gita Thanarajasingam, MD; Gina Mazza PhD; Minji Lee, PhD; Molly Jeffrey, PhD; Joseph Ross, MD

FDA Collaborators: Vishal Bhatnagar, MD; Ting-Yu Chen, PhD; Mallorie Fiero, PhD; Paul Kluetz, MD; Meena Murugappan, PharmD, PhD

Project Start Date: October 2022

Regulatory Science Challenge

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) has recommended physical function and side effects be measured by patient surveys during most or all cancer treatment clinical trials. 1 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 on their treatments.

This study aims 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. 2

Project Description and Goals

In the proposed study, researchers will develop 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 aims 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 are 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.

 

 

 

• 1US Food and Drug Administration (FDA). Core Patient-Reported Outcomes in Cancer Clinical Trials: Draft Guidance for Industry 2021 [Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/core-patient-reported-outcomes-cancer-clinical-trials accessed May 4 2022].
• 2US 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].