Development of a guidance for including patient-reported outcomes (PROs) in post-approval clinical trials of oncology drugs for comparative effectiveness research (CER).

Basch, Ethan; Abernethy, A. P.; Mullins, C. Daniel; Tiglao, M. R.; Tunis, Sean

doi:10.1200/jco.2011.29.15_suppl.6000

Cited by 6 publications

(19 citation statements)

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“…Other authors have since produced guidelines that also address aspects of this area, although most are limited by a lack of systematic development or stakeholder involvement [18,19,21,24-33]. More recently, the US Food and Drug Administration [8] and the Centre for Medical Technology Policy [34] have published guidance documents containing recommendations geared towards the management of missing data and aimed at optimising the implementation of PROs such as HRQL. Moreover, these guidelines specifically promote the training and education of both trial researchers and participants as a key element of clinical trial quality control.…”

Section: Discussionmentioning

confidence: 99%

“…Ideally, current guidance documents should be supplanted with internationally endorsed consensus guidelines, specifically tailored to the promotion of best practice in PRO data-collection. In the meantime, trialists should utilise existing PRO and HRQL-specific data-collection recommendations [8,18,19,21,23-34] to inform the trial design process, alongside more general high quality protocol guidelines such as the SPIRIT 2013 statement [10,41]. Trial management teams have a responsibility to provide local site personal with protocol content and supporting trial documentation, alongside trial training and education, which aids optimal collection of both standard and ‘concerning’ PRO data, whilst minimising the risk of bias.…”

Section: Discussionmentioning

confidence: 99%

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Inconsistencies in Quality of Life Data Collection in Clinical Trials: A Potential Source of Bias? Interviews with Research Nurses and Trialists

et al. 2013

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BackgroundPatient-reported outcomes (PROs), such as health-related quality of life (HRQL) are increasingly used to evaluate treatment effectiveness in clinical trials, are valued by patients, and may inform important decisions in the clinical setting. It is of concern, therefore, that preliminary evidence, gained from group discussions at UK-wide Medical Research Council (MRC) quality of life training days, suggests there are inconsistent standards of HRQL data collection in trials and appropriate training and education is often lacking. Our objective was to investigate these reports, to determine if they represented isolated experiences, or were indicative of a potentially wider problem.Methods And FindingsWe undertook a qualitative study, conducting 26 semi-structured interviews with research nurses, data managers, trial coordinators and research facilitators involved in the collection and entry of HRQL data in clinical trials, across one primary care NHS trust, two secondary care NHS trusts and two clinical trials units in the UK. We used conventional content analysis to analyze and interpret our data. Our study participants reported (1) inconsistent standards in HRQL measurement, both between, and within, trials, which appeared to risk the introduction of bias; (2), difficulties in dealing with HRQL data that raised concern for the well-being of the trial participant, which in some instances led to the delivery of non-protocol driven co-interventions, (3), a frequent lack of HRQL protocol content and appropriate training and education of trial staff, and (4) that HRQL data collection could be associated with emotional and/or ethical burden.ConclusionsOur findings suggest there are inconsistencies in the standards of HRQL data collection in some trials resulting from a general lack of HRQL-specific protocol content, training and education. These inconsistencies could lead to biased HRQL trial results. Future research should aim to develop HRQL guidelines and training programmes aimed at supporting researchers to carry out high quality data collection.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Inconsistencies in Quality of Life Data Collection in Clinical Trials: A Potential Source of Bias? Interviews with Research Nurses and Trialists

et al. 2013

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“…This approach is welcome: both experience and empirical research suggest that a failure to incorporate these design features during trial planning may result in PRO data that are uninformative or inappropriate for evaluating the harms and benefits of the intervention under study [4,5]. The EMA recommendations also align with those presented in other contemporary PRO guidance documents, including those produced by the Center for Medical Technology Policy [6] and the US Food and Drug Administration (FDA) [7]. The apparent harmonization of EMA and FDA guidance is encouraging, and it is hoped that further alignment in the coming years may allow sponsors to adopt a unified PRO claim strategy across the two agencies.…”

Section: Signs Of Encouragementmentioning

confidence: 67%

International Society for Quality of Life Research commentary on the draft European Medicines Agency reflection paper on the use of patient-reported outcome (PRO) measures in oncology studies

et al. 2015

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“…It is uncertain, however, whether they also reflect a deficiency in the published literature in this area. There are recent publications concerning the design of trials with a PRO outcome[7], [10] and, with the development of the CONSORT PRO extension[11], there is now guidance to improve PRO reporting: it remains unclear if the literature provides adequate coverage of in-trial issues.…”

Section: Introductionmentioning

confidence: 99%

Patient Reported Outcomes (PROs) in Clinical Trials: Is ‘In-Trial’ Guidance Lacking? A Systematic Review

et al. 2013

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BackgroundPatient reported outcomes (PROs) are increasingly assessed in clinical trials, and guidelines are available to inform the design and reporting of such trials. However, researchers involved in PRO data collection report that specific guidance on ‘in-trial’ activity (recruitment, data collection and data inputting) and the management of ‘concerning’ PRO data (i.e., data which raises concern for the well-being of the trial participant) appears to be lacking. The purpose of this review was to determine the extent and nature of published guidelines addressing these areas.Methods and FindingsSystematic review of 1,362 articles identified 18 eligible papers containing ‘in-trial’ guidelines. Two independent authors undertook a qualitative content analysis of the selected papers. Guidelines presented in each of the articles were coded according to an a priori defined coding frame, which demonstrated reliability (pooled Kappa 0.86–0.97), and validity (<2% residual category coding). The majority of guidelines present were concerned with ‘pre-trial’ activities (72%), for example, outcome measure selection and study design issues, or ‘post-trial’ activities (16%) such as data analysis, reporting and interpretation. ‘In-trial’ guidelines represented 9.2% of all guidance across the papers reviewed, with content primarily focused on compliance, quality control, proxy assessment and reporting of data collection. There were no guidelines surrounding the management of concerning PRO data.ConclusionsThe findings highlight there are minimal in-trial guidelines in publication regarding PRO data collection and management in clinical trials. No guidance appears to exist for researchers involved with the handling of concerning PRO data. Guidelines are needed, which support researchers to manage all PRO data appropriately and which facilitate unbiased data collection.

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Development of a guidance for including patient-reported outcomes (PROs) in post-approval clinical trials of oncology drugs for comparative effectiveness research (CER).

Cited by 6 publications

References 0 publications

Inconsistencies in Quality of Life Data Collection in Clinical Trials: A Potential Source of Bias? Interviews with Research Nurses and Trialists

Inconsistencies in Quality of Life Data Collection in Clinical Trials: A Potential Source of Bias? Interviews with Research Nurses and Trialists

International Society for Quality of Life Research commentary on the draft European Medicines Agency reflection paper on the use of patient-reported outcome (PRO) measures in oncology studies

Patient Reported Outcomes (PROs) in Clinical Trials: Is ‘In-Trial’ Guidance Lacking? A Systematic Review

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