Video-on-Demand Library

This content is restricted to members of PSI. If you are already a member please login. To join PSI or to see all the benefits of membership click here.

05 June 2019

Aims: Although patient reported outcomes (PROs) have become important endpoints in randomized controlled trials (RCTs), there is limited consensus on analysis and interpretation, making it difficult to draw robust conclusions based on PRO findings and compare findings across studies. The Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life (SISAQOL) Consortium therefore aims to set recommendations for PRO analysis in cancer RCTS. Methods: The Consortium is comprised of over 40 international experts including PRO researchers and statisticians; representatives from regulatory bodies, academic societies, pharmaceutical industry, cancer institutes and patient organizations. To standardize PRO analysis, well-defined PRO-specific objectives are needed. Following literature reviews, surveys and a consensus panel, a taxonomy of such objectives was established. Essential and desirable statistical properties were selected by the consensus panel as criteria for recommendation. Results: The developed taxonomy of PRO objectives distinguishes confirmatory from descriptive objectives; and superiority from non-inferiority/equivalence hypotheses. Within-treatment and within-individual objectives need to be pre-defined. To identify appropriate statistical methods, two essential and four desirable properties were identified. The essential properties were the ability to (a) perform a comparative test (statistical significance); (b) produce interpretable treatment effect estimates (clinical relevance). For each objective, the consortium evaluated selected statistical methods based on these criteria and ratified the recommendations where consensus was reached. Conclusions: To standardize PRO analysis for cancer RCTs, the SISAQOL consortium proposes a taxonomy of PRO objectives together with appropriate statistical methods. The resulting consensus document will form the first set of PRO analysis guidelines endorsed by a wide consortium in this field. 

Related Documents

Part of Collection(s)

Topic(s)

Upcoming Events