Patient-Reported Outcomes in Cancer Trials to Be Standardized

Pam Harrison

February 04, 2020

Recommendations have been made to standardize the analysis of patient-reported outcomes (PROs) in randomized controlled trials (RCTs) involving patients with cancer in order to ensure that quality-of-life results reflect the real risks, benefits, and tolerability of a given treatment, notes an international consortium of experts.

"The use of patient-reported outcomes in cancer clinical trials allows the patient voice to be incorporated in the evaluation of risks and benefits of cancer therapies," write lead author Corneel Coens, PhD, European Organisation for Research and Treatment of Cancer (EORTC) in Brussels, Belgium, and multicenter colleagues.

"The current...recommendations represent an important first step towards generating international consensus-based standards for PRO analysis in cancer RCTs," they conclude.

The recommendations were published online January 30 in The Lancet Oncology.

Patients' Own Reports Predict Survival

The authors belong to the Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints (SISAQOL) Data consortium, and they include PRO experts, statisticians, regulators, and representatives from international academic societies, industry, cancer institutes, and patient organizations.

Led by Andrew Bottomley, PhD, assistant director of EORTC, the consortium developed a "blueprint" for achieving consistent, standardized PRO reporting across cancer RTCs.

As they explain in a statement, patients' own reporting of quality-of-life endpoints is an important means by which to better assess the real value of a given treatment vs its risks.

Indeed, in was recently reported that PROs independently predict overall survival in cancer RCTs.

However, in a critical review of the literature, consortium members determined that there was not yet any consensus on how to optimally analyze PROs in cancer RCTs, thus limiting interpretation of results.

It is important that PRO findings are analyzed consistently across RCTs to ensure that they can "meaningfully" and "reliably" inform decisions about the safety of the treatment being analyzed, as well guide decisions about which is the best treatment for a given cancer. In addition, this information can also inform policy decisions that concern the use of that treatment, especially in the current era of skyrocketing cancer costs, the authors write. 

At a recommendations meeting held in 2018, four working groups from the consortium presented 43 statements; almost three quarters of them were ratified.

Standardized Objectives

Generating robust PRO conclusions across cancer clinical trials involves more than simply agreeing on — and using — standardized objectives, the consortium members point out.

"It also entails thoughtful trial planning and design with meaningful involvement of patient representatives from the beginning of the process," they suggest.

It also entails both the collection of high-quality data and transparent reporting of that data.

"We believe this set of recommendations will support clinical researchers, trialists, and statisticians to improve the conceptualization and design of PRO studies, the quality of statistical analysis, and the clinical interpretation of PROs in cancer clinical trials," the authors observe.

Bottomley himself suggested that although their standardized guidelines were developed specifically for cancer, "we think that the majority of them could be used elsewhere," he said. "Quality of life is important for everyone and we hope that researchers and patients across other disease areas will adapt them to their particular fields."

Commenting on the overall initiative, Kathy Oliver, chair, International Brain Tumour Alliance, Surrey, United Kingdom, and a member of the consortium, noted that the SISAQOL's efforts will help ensure that matters of importance to patients — and what profoundly affects them — are taken into consideration when making treatment decisions that ultimately can affect outcomes, and indeed patient survival.

Coens has disclosed no relevant financial relationships. Bottomley  reports grants to EORTC from Boehringer Ingelheim International and Genentech, as well as grants from Merck. Several coauthors have declared relationships with industry. The full list can be found with the original article.

The Lancet Oncology. Published online January 30, 2020. Abstract

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