# Analyzing and Interpreting PRO-CTCAE with CTCAE and Other Clinical Data to Characterize Drug Tolerability

> **NIH NIH U01** · UNIV OF NORTH CAROLINA CHAPEL HILL · 2021 · $469,493

## Abstract

PROJECT ABSTRACT
In cancer clinical trials, it is essential to understand and communicate the negative impact of drugs on patient
participants in order to balance risks and benefits. Symptom side effects such as nausea and diarrhea are
particularly common and impactful on the patient experience. However, historically this information has been
reported by clinicians on behalf of their patients rather than by patients themselves – a process that has been
shown to miss up to half of patients' symptoms. Therefore, the National Cancer Institute (NCI) supported
development of the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse
Events (PRO-CTCAE), a library of questions to enable systematic patient self-reporting of side effect
information. To date, the PRO-CTCAE questions have been developed and tested for validity and feasibility of
use in clinical trials – work that was led by the investigators on this proposal under contracts to the NCI.
However, it is not yet established how best to combine PRO-CTCAE information with traditional measures of
treatment impact such as clinician-reported adverse events, time on treatment, drug dose levels, performance
status, and hospitalizations. The purpose of this proposal is to advance understanding of the role of the PRO-
CTCAE in combination with these traditional measures of treatment impact in assessments of drug tolerability.
In this proposal, advanced statistical methods will be used to analyze data from numerous clinical trials
accessible to the team through the National Clinical Trial Network (cooperative groups) and industry. The
specific aims are to: 1) characterize relationships between the PRO-CTCAE and traditional measures of
treatment impact and tolerability; 2) test and standardize analytic techniques for the PRO-CTCAE; 3) refine
approaches to analyzing missing PRO-CTCAE data; and 4) develop and refine tabular and graphical data
visualizations for longitudinal PRO-CTCAE data. To accomplish these aims, an exceptional multidisciplinary
team has been assembled including scientific, clinical, and patient investigators with extensive experience in
academic and industry clinical research, and longstanding expertise in patient-reported outcomes, adverse
events, biostatistics, psychometrics, clinical trial design/analysis, health disparities, and informatics. This team
has successfully led multiple prior national research consortia. The team will work collaboratively and flexibly
with other investigators and stakeholders through the planned U01 consortium, and will share data, statistical
programming code, and ideas freely. This work is intended to inform best practices for tabulating and reporting
PRO-CTCAE data, and will enable more systematic and patient-centered evaluations of tolerability in cancer
clinical trials.

## Key facts

- **NIH application ID:** 10236375
- **Project number:** 5U01CA233046-04
- **Recipient organization:** UNIV OF NORTH CAROLINA CHAPEL HILL
- **Principal Investigator:** ETHAN M. BASCH
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $469,493
- **Award type:** 5
- **Project period:** 2018-09-30 → 2023-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10236375

## Citation

> US National Institutes of Health, RePORTER application 10236375, Analyzing and Interpreting PRO-CTCAE with CTCAE and Other Clinical Data to Characterize Drug Tolerability (5U01CA233046-04). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10236375. Licensed CC0.

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