New guidelines for statistical analysis of Quality of Life endpoints in cancer randomised controlled trials

International Standards for the Analysis of Quality of Life and Patient Reported Outcomes Endpoints in Cancer Randomised Controlled Trials

By Coens et al., in The Lancet Oncology 2020; 21(2):pe83-e96. www.thelancet.com/journals/lanonc/home

Prof Madeleine King | Chair, Quality of Life Office, University of Sydney

Patient-reported outcome (PRO) data, such as symptoms, functioning and other health-related quality of life endpoints are often included in cancer randomised controlled trials (RCTs) to provide valuable evidence on risks, benefits, safety and tolerability of treatment. PRO findings can and should inform patients, providers, payers and regulatory decision-makers. Ideally, PRO findings would be analysed, interpreted and reported in ways that are both easily understood and methodologically appropriate and robust. However, in reality, the opposite is true – PRO data are analysed with a confusing array of methods and presented and interpreted in many different ways. Standards and clear guidelines are needed.

The SISAQOL Consortium – Setting International Standards in Analysing Patient-Reported Outcomes and Quality of Life Endpoints Data – is setting this straight.

This paper reports the first set of consensus recommendations for PRO analysis in cancer RCTs. These address four key priorities:

1) A taxonomy of PRO objectives for RCTs

2) Appropriate statistical methods for each PRO objective type

3) Standardised statistical terminology for missing PRO data

4) Appropriate ways to manage missing data

This represents an important first step towards generating international consensus-based standards for PRO analysis in cancer RCTs.