Patient-Reported Outcomes and the Evolution of Adverse Event Reporting in Oncology

Abstract

Adverse event (AE) reporting in oncology has evolved from informal descriptions to a highly systematized process. The Common Terminology Criteria for Adverse Events (CTCAE) is the predominant system for describing the severity of AEs commonly encountered in oncology clinical trials. CTCAE clinical descriptors have been developed empirically during more than 30 years of use. The method of data collection is clinician based. Limitations of the CTC system include potential for incomplete reporting and limited guidance on data analysis and presentation methods. The Medical Dictionary for Regulatory Activities (MedDRA) is a comprehensive medical terminology system used for regulatory reporting and drug labeling. MedDRA does not provide for severity ranking of AEs. CTC-based data presentations are the primary method of AE data reporting used in scientific journals and oncology meetings. Patient-reported outcome instruments (PROs) cover the subjective domain of AEs. Exploratory work suggests PROs can be used with a high degree of patient engagement and compliance. Additional studies are needed to determine how PROs can be used to complement current AE reporting systems. Potential models for integrating PROs into AE reporting are described in this review. AE reporting methods will continue to evolve in response to changing therapies and growing interest in measuring the impact of cancer treatment on health status. Although integration of PROs into AE reporting may ultimately improve the comprehensiveness and quality of collected data, it may also increase the administrative burden and cost of conducting trials. Therefore, care must be used when developing health outcomes and safety data collection plans.

Patient‐reported acute gastrointestinal symptoms during concurrent chemoradiation treatment for rectal cancer

“…18 Complementing physician-reported toxicity and other objective measurements, PROs provide additional symptom information from the patient's perspective that may not be captured fully by using other measurements and that often are underreported by physician assessments. 7,[19][20][21][22][23] Therefore, there is increasing recognition of the value of PROs in the evaluation of the treatment impact on patient QOL, in assessments of treatment tolerability, and as part of the decision-making process by physicians on whether to adopt new therapies for their patients. 7,24,25 This recognition has led to efforts by the National Cancer Institute, working with the cooperative groups, to encourage and facilitate the incorporation of PROs into phase 2, phase 3, and supportive therapy trials.…”

“…We were able to achieve a high completion rate of 95%, and only 2of 77 consecutive patients were excluded because of nonparticipation, probably attributable in part to the low response burden from the short questionnaire. 23,25,26 The instrument appeared to be appropriate for this patient population and treatment, and all items were sensitive to treatment-related changes. Anecdotally, we believe that the PRO measurement did not lengthen clinical visits and that having PROs available facilitated communication and the management of patient symptoms.…”

Stakeholder perspectives on implementing the National Cancer Institute’s patient-reported outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE)

“…Moreover, it was felt that patient-reported adverse event information should be incorporated into published study results and drug labels. These findings reflect an overall movement in clinical research and drug regulatory policy towards favoring patient self-reports for those experiences best known to the patient, such as symptoms [3][4][5][13][14][15][16][17][18]. Of particular note is the FDA's recent guidance for industry on patient-reported outcomes measures in medical product development to support labeling claims, in which patient-reported outcomes (PROs) are advocated for measuring subjective experiences of patients related both to treatment benefit and risk [12,19].…”

“…Initially developed in 1984, it was substantially revised in 2003 to expand anatomic site specificity and include criteria for surgical effects, and was again revised in 2009 to harmonize terminology with the Medical Dictionary for Regulatory Activities [2,3].…”

Patient-reported Outcome Measures in Radiotherapy: Clinical Advances and Research Opportunities in Measurement for Survivorship

“…Systematic monitoring of late effects from radiotherapy is important in determining normal tissue effects and measuring dose response (8). However, consistent assessment is rarely undertaken routinely in the UK.…”