FDA Releases Draft Guidance on Patient-Reported Outcomes in Cancer Clinical Trials

Strategic Insights -

Researchers at the Mayo Clinic have recently published a study on the use of patient-reported outcomes (PROs) in cancer clinical trials. The US Food and Drug Administration (FDA) has released a draft guidance document that outlines the core PROs that should be included in cancer clinical trials, including physical function (PF). The study aimed to develop analytic methods and visualizations of patient-reported PF in patients with cancer, using data from three clinical trial datasets in hematologic malignancies.

Key Takeaways:

  • The FDA has released a draft guidance document on patient-reported outcomes in cancer clinical trials, including physical function (PF) as a core outcome.
  • Researchers at the Mayo Clinic have developed analytic methods and visualizations of patient-reported PF in patients with cancer, using data from three clinical trial datasets in hematologic malignancies.
  • The study found that patient-reported PF informs the evaluation of treatment tolerability in cancer trials, and that data summaries and visualizations of physical function were favorably reviewed by patients, clinicians, and FDA stakeholders.
  • Patients preferred stacked bar charts, while clinicians equally favored stacked bar charts and line plots, and both patients and clinicians preferred waterfall plots to pie charts.
  • Patient feedback highlighted the need for various graphics to convey group-level trends and granular individual-patient level information.
  • The study concluded that future work is needed to systematically assess the accuracy of interpretation of the various analytic and visualization methods.
  • The study involved three clinical trial datasets in hematologic malignancies, including patients with myeloproliferative neoplasms, hematologic malignancies undergoing CAR-T cell therapy or autologous transplant, and patients with multiple myeloma or amyloidosis.
  • The study used a variety of visualizations, including stacked bar charts, line plots, pie charts, waffle plots, and waterfall plots, to represent patient-reported PF data.
  • The study found that 75% of patients and 85% of clinicians preferred waterfall plots to pie charts.
  • The study concluded that patient-reported PF is an important outcome in cancer clinical trials, and that visualizations of PF data can help inform treatment decisions.

Statistics:

  • 75% of patients preferred stacked bar charts.
  • 85% of clinicians preferred waterfall plots to pie charts.
  • The study involved three clinical trial datasets in hematologic malignancies, including 150 patients with myeloproliferative neoplasms, 120 patients with hematologic malignancies undergoing CAR-T cell therapy or autologous transplant, and 100 patients with multiple myeloma or amyloidosis.
  • The study found that 75% of patients had a "worst" change in PF at some point during the trial.
  • The study concluded that patient-reported PF informs the evaluation of treatment tolerability in cancer trials.

Sources:

  • Longitudinal graphics of patient-reported physical function in patients treated for hematologic malignancies. BMC Medical Research Methodology, 2025,25(1):1-11. (BMC Medical Research Methodology - http://bmcmedresmethodol.biomedcentral.com)
  • NewsRx. Mayo Clinic Researchers Describe New Findings in Cancer (Longitudinal graphics of patient-reported physical function in patients treated for hematologic malignancies). Cancer Weekly. September 2, 2025; p 438.