The use of patient-reported outcomes instruments in registered clinical trials: Evidence from ClinicalTrials.gov

Abstract

Background-Investigators of clinical trials in which the list of outcomes include patient-reported outcomes (PROs) -usually labeled quality of life (QoL) -have a large number of instruments from which to choose. The extent and manner in which PRO instruments are used in clinical trials can be assessed using data from clinical trial registries. Most medical journals now require a clinical trial be registered before its results are considered for publication. This requirement is intended to discourage publication bias, such as the reporting of tests of hypotheses different from those stipulated at the start of the trial and selective reporting of partial results.Purpose-To assess the usage of PRO instruments in registered trials by various trial characteristics and to determine if the instruments are adequately identified in the registry.Methods-A local copy of the ClinicalTrials.gov database was made in September 2007. The outcomes of all interventional trials registered since September 2004 were assessed for usage of a PRO instrument. Odds ratios of PRO usage were estimated by a logistic regression model.Results-Of 17,704 interventional trials, 2,481 (14.0%) used at least one PRO instrument. However, less than half of those trials (41.0%) identified the instrument to be used. PRO usage is positively associated with phase (III), randomization type (randomized), intervention type (behavior) and sponsorship type (university/research organization).Conclusions-PRO instruments are used in a significant percentage but minority of clinical trials. Trial registries should require that all PRO instruments be identified, including the concepts or outcomes they are intended to measure.

Cardiovascular Health: The Importance of Measuring Patient-Reported Health Status

“…However, patient health status surveys remain underused as metrics in clinical studies. 15 In addition, patient health status is a strong, independent predictor of other health outcomes, including mortality, cardiovascular events, hospitalization, and costs of care. [16][17][18] As such, patient health status surveys not only measure health outcomes, they also help predict outcomes and quantify patient risk above and beyond traditional risk variables such as patient demographics, medical history, and physiological and anatomic tests.…”

Erratum to: Psychometric evaluation of the Cystic Fibrosis Questionnaire-Revised in a national, US sample

“…Efforts to develop reliable and valid PRO instruments have been very successful, resulting in numerous well-validated tools and a growing empirical and clinical database [11]. PROs are particularly important for patients with chronic diseases, such as cystic fibrosis (CF), whose daily lives are significantly impacted by their illness.…”

Clinical outcomes in psoriatic arthritis: A systematic literature review

“…Instruments for measuring PROs are easy to administer and some, like the Health Assessment Questionnaire (HAQ), have been proven to be reliable, valid, and sensitive to change (11). There are several ways to explore PROs: some are qualitative (12), whereas others are quantitative such as by assessing the frequency of different outcome measures (4,5).…”