Abstract
BackgroundEvidence suggests there are inconsistencies in patient-reported outcome (PRO) assessment and reporting in clinical trials, which may limit the use of these data to inform patient care. For trials with a PRO endpoint, routine inclusion of key PRO information in the protocol may help improve trial conduct and the reporting and appraisal of PRO results; however, it is currently unclear exactly what PRO-specific information should be included. The aim of this review was to summarize the current PRO-specific guidance for clinical trial protocol developers.Methods and FindingsWe searched the MEDLINE, EMBASE, CINHAL and Cochrane Library databases (inception to February 2013) for PRO-specific guidance regarding trial protocol development. Further guidance documents were identified via Google, Google scholar, requests to members of the UK Clinical Research Collaboration registered clinical trials units and international experts. Two independent investigators undertook title/abstract screening, full text review and data extraction, with a third involved in the event of disagreement. 21,175 citations were screened and 54 met the inclusion criteria. Guidance documents were difficult to access: electronic database searches identified just 8 documents, with the remaining 46 sourced elsewhere (5 from citation tracking, 27 from hand searching, 7 from the grey literature review and 7 from experts). 162 unique PRO-specific protocol recommendations were extracted from included documents. A further 10 PRO recommendations were identified relating to supporting trial documentation. Only 5/162 (3%) recommendations appeared in ≥50% of guidance documents reviewed, indicating a lack of consistency.ConclusionsPRO-specific protocol guidelines were difficult to access, lacked consistency and may be challenging to implement in practice. There is a need to develop easily accessible consensus-driven PRO protocol guidance. Guidance should be aimed at ensuring key PRO information is routinely included in appropriate trial protocols, in order to facilitate rigorous collection/reporting of PRO data, to effectively inform patient care.
Highlights
Patient-reported outcomes (PROs), including health-related quality of life (HRQL), symptoms such as pain or fatigue, and health utility, are increasingly assessed in clinical trials as a measure of effectiveness.[1,2,3] patient-reported outcome (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
Guidance should be aimed at ensuring key PRO information is routinely included in appropriate trial protocols, in order to facilitate rigorous collection/reporting of PRO data, to effectively inform patient care
To help ensure optimal PRO data collection, PRO-specific components should be considered during clinical trial design and clearly documented in the trial protocol. [6,7] The trial protocol is the cornerstone of a well-conducted trial, and should provide specific instruction on how to conduct all aspects of the study
Summary
Patient-reported outcomes (PROs), including health-related quality of life (HRQL), symptoms such as pain or fatigue, and health utility, are increasingly assessed in clinical trials as a measure of effectiveness.[1,2,3] 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 aim of this systematic review was to summarize current PRO-specific guidance for clinical trial protocol developers. Eleven unique recommendations related to the inclusion of PROs in the introductory sections of the protocol These focused on aspects surrounding: PRO specific background information (n = 2), for instance, the need to describe the PRO population of interest; specification of the PRO rationale (n = 5), for example, justifying the relevance of PRO assessment in the disease and population under investigation; or outlining the PRO hypothesis and objectives (n = 4). Evidence suggests there are inconsistencies in patient-reported outcome (PRO) assessment and reporting in clinical trials, which may limit the use of these data to inform patient care. The aim of this review was to summarize the current PRO-specific guidance for clinical trial protocol developers
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