PRO-CTCAE Provides "Missing Patient Voice" in Reporting Cancer Treatment Symptoms

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A prescription drug’s label AE section is almost entirely based on clinicians’ impression of patients’ symptoms, not the patients’ own reports.
A prescription drug’s label AE section is almost entirely based on clinicians’ impression of patients’ symptoms, not the patients’ own reports.

Patients with cancer want to proceed with patient-reported outcomes (PROs) for the adverse events (AEs) they experience when undergoing treatment.

That is the conclusion of an Invited Commentary1 on the first primary validation publication2 for the National Cancer Institute's (NCIs) Patient-Reported Outcomes Common Terminology Criteria for Adverse Events (PRO-CTCAE).

“As clinicians, we often miss side-effect symptoms our patients are experiencing, especially between visits,” Ethan Basch, MD, MSc, the study's principal investigator, told Cancer Therapy Advisor.

“The PRO-CTCAE is a tool that can potentially help us collect this information better, leading to improved communication, better symptom management, and more patient-centered drug development.”

Dr. Basch said that although other PRO-questionnaires are used to assess quality of life, “or to evaluate how much cancer-related symptoms resolve during treatment (eg, does treatment X decrease pain related to bone metastases?), PRO-CTCAE is different from these tools in that it is intended to assess side effects. PRO-CTCAE will likely become the gold standard for patient reporting of side effects in cancer clinical trials, while other PRO tools will continue to be useful for their purposes.”

When patients want to know about potential symptoms they may experience from a prescription drug, they read the label's AE section, little do they realize “that this information, largely collected during clinical trials, is based almost entirely on clinicians' impression of patients' symptoms – not on patients' own firsthand reports of their experiences with the drug,” Dr. Basch, then at Memorial Sloan Kettering Cancer Center, New York, NY, wrote in an article in the The New England Journal of Medicine in 2010 titled, “The Missing Patient Voice in Drug-Safety Reporting.”3

This “patient voice” is especially important since the 2009 U.S. Food and Drug Administration (FDA) final guidance document recommends that applicants for drug labeling claims of treatment benefits incorporate PRO instruments in clinical trials that best measure concepts from a patient perspective.4,5

“Yet there is no current standard measurement approach or instrument for collecting direct patient reports of AEs in clinical research,”5 Dr. Basch and colleagues wrote in 2014.

Currently, investigators document symptomatic AEs in cancer clinical trials using the CTCAE, which has evolved since the National Cancer Institute (NCI) developed “Common Toxicity Criteria” in 1982 for use in adverse drug experience reporting.6 The current version, 4.03, includes 790 discrete AEs; v5.0 is expected to be released in October 2015, with implementation in early 2016.

However, the CTCAE is known to under detect symptomatic AEs.5 For that reason, the NCI issued two contracts, in 2008 and 2010, for creation of a companion PRO measurement system.

Since then, Dr. Basch, now with the Cancer Outcomes Research Program, Lineberger Comprehensive Cancer Center at the University of North Carolina in Chapel Hill, NC, and a group of multidisciplinary investigators and patient representatives have been working to develop and validate the PRO-CTCAE.

RELATED: ASCO Statement on Increasing the Number of Older Patients in Clinical Trials

They first conducted a systematic evaluation of all 790 AEs in the CTCAE item library and identified 78 appropriate for patient self-reporting.5

Next, they created a plain-language English term for each, with one to three items characterizing the frequency, severity, and/or activity interference of the AE.

This resulted in 124 PRO-CTCAE items, from cardiac disorders (pounding/racing heartbeat), to gastrointestinal disorders (bloating, constipation, vomiting), to nervous system disorders (dizziness, headache, problems with concentration).2,5

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