Patient-reported Outcomes in Randomised Controlled Trials of Prostate Cancer: Methodological Quality and Impact on Clinical Decision Making

Abstract

Context Patient-reported outcomes (PRO) data from randomised controlled trials (RCTs) are increasingly used to inform patient-centred care as well as clinical and health policy decisions. Objective The main objective of this study was to investigate the methodological quality of PRO assessment in RCTs of prostate cancer (PCa) and to estimate the likely impact of these studies on clinical decision making. Evidence acquisition A systematic literature search of studies was undertaken on main electronic databases to retrieve articles published between January 2004 and March 2012. RCTs were evaluated on a predetermined extraction form, including (1) basic trial demographics and clinical and PRO characteristics; (2) level of PRO reporting based on the recently published recommendations by the International Society for Quality of Life Research; and (3) bias, assessed using the Cochrane Risk of Bias tool. Studies were systematically analysed to evaluate their relevance for supporting clinical decision making. Evidence synthesis Sixty-five RCTs enrolling a total of 22 071 patients were evaluated, with 31 (48%) in patients with nonmetastatic disease. When a PRO difference between treatments was found, it related in most cases to symptoms only (n = 29, 58%). Although the extent of missing data was generally documented (72% of RCTs), few reported details on statistical handling of this data (18%) and reasons for dropout (35%). Improvements in key methodological aspects over time were found. Thirteen (20%) RCTs were judged as likely to be robust in informing clinical decision making. Higher-quality PRO studies were generally associated with those RCTs that had higher internal validity. Conclusions Including PRO in RCTs of PCa patients is critical for better evaluating the treatment effectiveness of new therapeutic approaches. Marked improvements in PRO quality reporting over time were found, and it is estimated that at least one-fifth of PRO RCTs have provided sufficient details to allow health policy makers and physicians to make critical appraisals of results. Patient summary In this report, we have investigated the methodological quality of PCa trials that have included a PRO assessment. We conclude that including PRO is critical to better evaluating the treatment effectiveness of new therapeutic approaches from the patient's perspective. Also, at least one-fifth of PRO RCTs in PCa have provided sufficient details to allow health policy makers and physicians to make a critical appraisal of results.

Preliminary evidence on the uptake, use and benefits of the CONSORT-PRO extension

“…Similar to past reviews [19,21,23], we found that reporting of PRO endpoints in a dedicated publication was a predictor of more complete reporting. Whilst detailed secondary PRO publications should be encouraged as they allow for presentation of additional analyses, the principal PRO findings should be reported in accordance with CONSORT-PRO and in the main RCT publication to facilitate interpretation of PRO results within the context of other endpoints, and to provide the patients' perspective to complement other trial information.…”

“…Again, these are knowledge transfer concerns requiring intervention to improve reporting practices and to ensure PRO results are interpreted accurately so they can appropriately inform patient care. Recent reviews confirm that reporting of PRO endpoints remains unsatisfactory overall; particularly regarding the reporting of PRO hypotheses, methodology, missing data, and generalisability of results [19][20][21][22][23][24][25][26]. Failing to report this information is wasteful as it limits the potential for readers to appraise the effect of interventions on patient health status, and the potential for PRO systematic reviews to impact clinical recommendations and health policy [27,28].…”

“…It may also decrease clinicians' confidence in the value of PRO data [29]. These aforementioned reviews [19][20][21][22][23][24] predominately include RCTs published before CONSORT-PRO. We expect that adherence to CONSORT-PRO will improve with time, as awareness and uptake increases.…”

Standardization of Patient-centered Outcomes: Less Is More

“…A key trend in US health care has been more focus on patient-reported outcomes (PROs), reflecting those aspects of outcome that are best obtained directly from patients, such as functional, psychosocial, and healthrelated quality of life. Importantly, PROs represent a metric applicable across varying procedures and provider types and, applied with quality-adjusted life-year data, enable comparative, effective research studies that inform valuebased health care models.So, how are we doing with collecting PRO data in prostate cancer (PCa) management?During the past decade, >22 000 PCa patients have been enrolled in 65 clinical trials with a PRO component [2]. Approximately 48% of these patients studied had localized PCa.…”

“…During the past decade, >22 000 PCa patients have been enrolled in 65 clinical trials with a PRO component [2]. Approximately 48% of these patients studied had localized PCa.…”