Abstract

Background

Patient-reported outcome (PRO) data, such as health-related quality of life and symptoms, are increasingly being captured in cancer randomized clinical trials (RCTs) to provide valuable information on treatment risks, benefits and tolerability. Our literature review in various cancer fields showed little consensus about analysis, interpretation and reporting of these data, hindering comparability of results across trials. The Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL) Consortium was convened to set recommendations for PRO analysis in cancer RCTs.

Method

The Consortium consists of 40 international experts including PRO researchers and statisticians, representatives from regulatory bodies, academic societies, pharmaceutical industry, cancer institutes and patient organizations. Subgroups were formed to focus on four priorities: (a) specification of well-defined PRO research objectives, (b) recommendations for appropriate statistical PRO analysis methods, (c) standardization of statistical terminology, and (d) development of guidelines for analyzing missing data. Methods used included literature review, surveys and expert discussions. Recommendations were ratified through consensus voting in a final meeting.

Results

A taxonomy of research objectives was established. Appropriate statistical methods, with the exception of summary measures, were proposed. Consensus was reached on the taxonomy of research objectives and statistical methods, along with a definition of missing data and two rates to report missing data occurrence. While some statements concerning handling missing data or statistical analyses are to be discussed, many statements were ratified for each of the priorities.

Conclusion

A robust first set of PRO analyses recommendations was developed in a joint process with diverse international stakeholders. Addressing the needs and requirements of these stakeholders provides a strong foundation for widespread endorsement of these recommendations. Ultimately, harmonization of current research practices will enhance interpretability and impact of PRO data in cancer RCTs.

This publication is a SISAQOL group authorship reflecting solely the views of the individual members.