Patient-reported outcomes (PROs), including health-related quality of life, symptoms such as pain or fatigue, and health utility, are increasingly assessed in clinical trials as a measure of effectiveness; providing valuable information on the patient perspective. PRO trial data may be used to inform clinical care and decision-making, predict long-term outcomes and influence health-policy; but to do so, as with any trial outcome, they must be collected with rigor. The research published in PLoS One calls for the development of consolidated, easily accessible and internationally endorsed consensus guidelines for protocol developers. This was presented at the 'Improving the Design of Clinical trials with Patient Reported Outcomes: Tips for Protocol Design and Review' workshop at the ISOQOL Annual Conference in Berlin from 15 - 18 October 2014.
Patient-Reported Outcome (PRO) Assessment in Clinical Trials: A Systematic Review of guidance for Trial Protocol Writers provides a systematic review of existing PRO-specific protocol guidance. "We found that guidance was difficult to access, lacked consistency and may be challenging to implement in practice: we identified 162 unique recommendations spread across 54 different publications." said researchers Professor Melanie Calvert and Mr Derek Kyte (University of Birmingham). They explain that in their second publication, Systematic Evaluation of the Patient-Reported Outcome (PRO) Content of Clinical Trial Protocol, "we evaluated, for the first time, the PRO-specific content of 75 NIHR-HTA clinical trial protocols using a specially constructed PRO-specific protocol checklist (the development of which was informed by the systematic review presented above) and also the SPIRIT checklist. We found the PRO content of trial protocols to be poor: information on the PRO rationale/hypothesis, data collection methods, training and management was often incomplete or missing completely. For example, only 8% of protocols included a rationale for PRO assessment."
The collection of PROs is essential for the development of pharmaceutical treatments and the implementation of new healthcare interventions. Trial results tell us what is working, and what is not, from the patient's point of view."
- Mr Derek Kyte and Professor Melanie Calvert, taken from the University of Birmingham Press Release on 16 October, 2014
Both of the papers and an associated blog Going PRO - clinical trials must plan to capture patient-reported outcomes by Professor David Moher, call for the development of consolidated, easily accessible and internationally endorsed consensus guidelines for protocol developers with associated training tools to ensure key PRO information is routinely included in trial protocols with a PRO endpoint to facilitate the rigorous collection and reporting of PRO data, thus maximising its capacity to effectively inform patient care. The press release and Birmingham brief have been picked up by reporters in Germany and China.