Systematic Evaluation of Patient-Reported Outcome Protocol Content and Reporting in Cancer Trials.
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ABSTRACT: Background
Patient-reported outcomes (PROs) are captured within cancer trials to help future patients and their clinicians make more informed treatment decisions. However, variability in standards of PRO trial design and reporting threaten the validity of these endpoints for application in clinical practice.Methods
We systematically investigated a cohort of randomized controlled cancer trials that included a primary or secondary PRO. For each trial, an evaluation of protocol and reporting quality was undertaken using standard checklists. General patterns of reporting where also explored.Results
Protocols (101 sourced, 44.3%) included a mean (SD) of 10 (4) of 33 (range = 2-19) PRO protocol checklist items. Recommended items frequently omitted included the rationale and objectives underpinning PRO collection and approaches to minimize/address missing PRO data. Of 160 trials with published results, 61 (38.1%, 95% confidence interval = 30.6% to 45.7%) failed to include their PRO findings in any publication (mean 6.43-year follow-up); these trials included 49 568 participants. Although two-thirds of included trials published PRO findings, reporting standards were often inadequate according to international guidelines (mean [SD] inclusion of 3 [3] of 14 [range = 0-11]) CONSORT PRO Extension checklist items). More than one-half of trials publishing PRO results in a secondary publication (12 of 22, 54.5%) took 4 or more years to do so following trial closure, with eight (36.4%) taking 5-8 years and one trial publishing after 14 years.Conclusions
PRO protocol content is frequently inadequate, and nonreporting of PRO findings is widespread, meaning patient-important information may not be available to benefit patients, clinicians, and regulators. Even where PRO data are published, there is often considerable delay and reporting quality is suboptimal. This study presents key recommendations to enhance the likelihood of successful delivery of PROs in the future.
SUBMITTER: Kyte D
PROVIDER: S-EPMC6855977 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:IntroductionPatient-reported outcomes (PROs) are increasingly included within cancer clinical trials. If appropriately collected, analysed and transparently reported, these data might provide invaluable evidence to inform patient care. However, there is mounting indication that the design and reporting of PRO data in cancer trials may be suboptimal. This programme of research will establish via three interlinked studies whether these findings are applicable to UK cancer trials, and if so, how to best enhance the way PROs are assessed, managed and reported in clinical trials. This study will explore with key stakeholders factors that influence optimal PRO protocol content, implementation and reporting and make recommendations for training and guidance.Methods and analysisSemistructured interviews will be conducted with members of key stakeholder groups. The purposive sample of up to 48 participants will include: (1) trial chief investigators, trial management group members, statisticians and research nurses of cancer trials including primary or secondary PRO recruited via the National Cancer Research Institute (NCRI) Clinical Studies Group and Consumer Liaison Group and the UK Clinical Research Collaboration Registered UK Clinical Trial Unit Network; (2) NCRI Consumer Liaison Group members; (3) international experts in PRO oncology trial design; and (4) journal editors and funding bodies. Data will be analysed using directed thematic analysis employing a coding frame and modified as analysis progresses. Formal triangulation of coding and member checking will be employed to enhance credibility.Ethics and disseminationThis study was approved by the University of Birmingham Ethics Committee (Ref: ERN_17-0085). Findings will be disseminated via conference presentations, peer-reviewed journals, patient groups and social media (@CPROR_UoB; http://www.birmingham.ac.uk/cpror).Prospero registration numberCRD42016036533.
| S-EPMC5829743 | biostudies-literature
Project description:BackgroundThe main objectives of this study were to identify the number of randomized controlled trials (RCTs) including a patient-reported outcome (PRO) endpoint across a wide range of cancer specialties and to evaluate the completeness of PRO reporting according to the Consolidated Standards of Reporting Trials (CONSORT) PRO extension.MethodsRCTs with a PRO endpoint that had been performed across several cancer specialties and published between 2004 and 2013 were considered. Studies were evaluated on the basis of previously defined criteria, including the CONSORT PRO extension and the Cochrane Collaboration's tool for assessing the risk of bias of RCTs. Analyses were also conducted by the type of PRO endpoint (primary vs secondary) and by the cancer disease site.ResultsA total of 56,696 potentially eligible records were scrutinized, and 557 RCTs with a PRO evaluation, enrolling 254,677 patients overall, were identified. PROs were most frequently used in RCTs of breast (n = 123), lung (n = 85), and colorectal cancer (n = 66). Overall, PROs were secondary endpoints in 421 RCTs (76%). Four of 6 evaluated CONSORT PRO items were documented in less than 50% of the RCTs. The level of reporting was higher in RCTs with a PRO as a primary endpoint. The presence of a supplementary report was the only statistically significant factor associated with greater completeness of reporting for both RCTs with PROs as primary endpoints (β = .19, P = .001) and RCTs with PROs as secondary endpoints (β = .30, P < .001).ConclusionsImplementation of the CONSORT PRO extension is equally important across all cancer specialties. Its use can also contribute to revealing the robust PRO design of some studies, which might be obscured by poor outcome reporting.
| S-EPMC4755292 | biostudies-literature
Project description:Qualitative evidence suggests patient-reported outcome (PRO) information is frequently absent from clinical trial protocols, potentially leading to inconsistent PRO data collection and risking bias. Direct evidence regarding PRO trial protocol content is lacking. The aim of this study was to systematically evaluate the PRO-specific content of UK National Institute for Health Research (NIHR) Health Technology Assessment (HTA) programme trial protocols.We conducted an electronic search of the NIHR HTA programme database (inception to August 2013) for protocols describing a randomised controlled trial including a primary/secondary PRO. Two investigators independently reviewed the content of each protocol, using a specially constructed PRO-specific protocol checklist, alongside the 'Standard Protocol Items: Recommendations for Interventional Trials' (SPIRIT) checklist. Disagreements were resolved through discussion with a third investigator. 75 trial protocols were included in the analysis. Protocols included a mean of 32/51 (63%) SPIRIT recommendations (range 16-41, SD 5.62) and 11/33 (33%) PRO-specific items (range 4-18, SD 3.56). Over half (61%) of the PRO items were incomplete. Protocols containing a primary PRO included slightly more PRO checklist items (mean 14/33 (43%)). PRO protocol content was not associated with general protocol completeness; thus, protocols judged as relatively 'complete' using SPIRIT were still likely to have omitted a large proportion of PRO checklist items.The PRO components of HTA clinical trial protocols require improvement. Information on the PRO rationale/hypothesis, data collection methods, training and management was often absent. This low compliance is unsurprising; evidence shows existing PRO guidance for protocol developers remains difficult to access and lacks consistency. Study findings suggest there are a number of PRO protocol checklist items that are not fully addressed by the current SPIRIT statement. We therefore advocate the development of consensus-based supplementary guidelines, aimed at improving the completeness and quality of PRO content in clinical trial protocols.
| S-EPMC4198237 | biostudies-literature
Project description:BackgroundThis study aimed to evaluate the patient-reported outcome (PRO) content of ovarian cancer randomised-controlled trial (RCT) publications, describe PRO compliance, and explore potential relationships among these and completeness of PRO protocol content.MethodsPublications of Phase III ovarian cancer RCTs with PRO endpoints were identified by Medline and Cochrane systematic search: January 2000 to February 2016. Two reviewers determined the number of Consolidated Standards of Reporting Trials (CONSORT)-PRO Extension items addressed in publications. Compliance rates (defined as the proportion of participants included in the principal PRO analysis, of those from whom PRO assessments were expected) were extracted. The relationship between CONSORT-PRO score and compliance rates was explored using scatter plots. Additionally CONSORT-PRO score and PRO compliance rates respectively were compared with corresponding PRO protocol scores obtained from a previous study.ResultsThirty-six eligible RCTs (n = 33 with secondary PRO endpoint) were identified and analysed. The average number of CONSORT-PRO items addressed in publications was 6.7 (48%; Range 0-13.5/14). Three RCTs did not report PRO results; in 1 case due to poor compliance. Some compliance information was reported in 26 RCTs, but was considered complete for only 10 (28%) RCTs. Compliance rates were poor overall, ranging from 59 to 83%; therefore missing PRO data from 17 to 41% of participants in these trials could have been avoided.Of the 26 (73%) RCTs for which PRO protocol completeness scores were available, 6 RCTs reported complete compliance information and the 3 of these RCTs with highest PRO compliance had highest protocol checklist scores.ConclusionsFew RCTs reported PRO compliance information in a manner enabling assessment of the generalisability of PRO results. This information is particularly important in RCTs of advanced ovarian cancer because it is important to be able to determine if missing data was due to worsening illness compared to methodological issues. Poor compliance appeared related to poor PRO protocol content, and in one case prevented PRO results from being reported, highlighting the need to address compliance strategies in the protocol. Adhering to protocol and CONSORT-PRO reporting guidance should improve PRO implementation and reporting respectively in ovarian cancer RCTs and allow results to meaningfully inform clinical practice.
| S-EPMC5934909 | biostudies-literature
Project description:BACKGROUND:Mind-body exercise interventions are typically multimodal, complex, and pluralistic, and few have been developed with the goal of therapeutically targeting a specific medical population. It is thus important that clinical trials evaluating mind-body interventions provide some justification for the use of the specific protocol being evaluated. OBJECTIVES:This article reports the results of a systematic review of the quality of reporting of protocol rationale and content validity for using a specific t'ai chi protocol in a randomized controlled trial (RCT). METHODS:Electronic literature searches were conducted using PubMed/MEDLINE, EBSCOhost, and the Cochrane Library from inception through June 2015. Search terms were Tai Chi, Taiji, Tai Chi Chuan; searches were limited to English-language RCTs. Inclusion and exclusion of trials were reported in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. The authors assessed the level of reporting with respect to t'ai chi protocol validation using a 5-point grading system based on whether (1) a specific protocol was mentioned, (2) rationale for the specific protocol was stated and supported, and (3) formal evaluation for content validity was conducted. RESULTS:There was significant heterogeneity in the quality of reporting related to protocol rationale and content validity. A total of 171 publications were identified. Studies met between 0 and 4 validity criteria (of possible 5, more indicating better quality), with a mean of 2.52 (±SD 1.2) and median of 3. Twenty (12%) trials did not mention a specific t'ai chi protocol, 10 (6%) trials met 0 of 5 criteria, and 47 (31%) studies met 4 of 5 criteria. Formal validity assessments were employed in only one trial. CONCLUSIONS:The poor quality of protocol rationale and content validity reporting limits our ability to accurately evaluate the evidence of t'ai chi as a therapeutic intervention. The development of formal guidelines for developing and reporting intervention validity for multimodal mind-body exercises like t'ai chi may improve the quality and interpretability of research.
| S-EPMC6909733 | biostudies-literature
Project description:PurposeEvidence suggests that the patient-reported outcome (PRO) content of cancer trial protocols is frequently inadequate and non-reporting of PRO findings is widespread. This qualitative study examined the factors influencing suboptimal PRO protocol content, implementation, and reporting, and use of PRO data during clinical interactions.MethodsSemi-structured interviews were conducted with four stakeholder groups: (1) trialists and chief investigators; (2) people with lived experience of cancer; (3) international experts in PRO cancer trial design; (4) journal editors, funding panelists, and regulatory agencies. Data were analyzed using directed thematic analysis with an iterative coding frame.ResultsForty-four interviews were undertaken. Several factors were identified that could influenced effective integration of PROs into trials and subsequent findings. Participants described (1) late inclusion of PROs in trial design; (2) PROs being considered a lower priority outcome compared to survival; (3) trialists' reluctance to collect or report PROs due to participant burden, missing data, and perceived reticence of journals to publish; (4) lack of staff training. Strategies to address these included training research personnel and improved communication with site staff and patients regarding the value of PROs. Examples of good practice were identified.ConclusionMisconceptions relating to PRO methodology and its use may undermine their planning, collection, and reporting. There is a role for funding, regulatory, methodological, and journalistic institutions to address perceptions around the value of PROs, their position within the trial outcomes hierarchy, that PRO training and guidance is available, signposted, and readily accessible, with accompanying measures to ensure compliance with international best practice guidelines.
| S-EPMC8366078 | biostudies-literature
Project description: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.
| S-EPMC4198295 | biostudies-literature
Project description:BACKGROUND:Patient-reported outcome measures (PROMs) are tools that enable patients to directly report their own assessments of well-being, or symptoms, in a structured and consistent way. Despite the usefulness of PROMs in optimizing health outcomes, their use in clinical practice is not routine. PROMs are complex to integrate into the clinical setting, with many elements potentially impacting on the success of implementation. For this reason, a protocol has been developed to guide a systematic review to collate information on implementation as presented in the randomized controlled trials (RCTs) to date. OBJECTIVE:The primary objective of this systematic review is to identify and synthesize factors available from RCT data about the fidelity of PROM interventions in clinical practice. The secondary objective will be an assessment of how implementation factors impact fidelity outcomes. METHODS:Preferred Reporting Items for Systematic Reviews and Meta-Analyses reporting standards will be followed. MEDLINE, EMBASE, and the Cumulative Index to Nursing and Allied Health Literature via OvidSP will be accessed using a defined search strategy. Grey literature and ClinicalTrials.gov will be reviewed for unpublished studies. Data extraction will be done to identify fidelity and factors impacting implementation, summarized using a narrative synthesis. An evidence-based implementation science framework will assist in identifying potential elements of importance and their effect on the process and outcomes of implementation. A meta-analysis to assess the impact of implementation factors will be attempted. A Cochrane risk of bias tool will be used. RESULTS:This protocol has received funding, and searches of databases will commence at the end of May 2019. It is planned that this systematic review will be finalized for publication in (December) 2019. CONCLUSIONS:Applying an implementation science evidence-based framework to the published literature may identify factors present in the data that impact on the implementation of PROMs into routine clinical care. This systematic review aims to improve understanding of how these factors impact the fidelity of this intervention, so that PROMs can be more effectively used in the care of patients. This systematic review can also offer more detailed information about the process and outcomes of successful implementation of PROMs. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID):PRR1-10.2196/14579.
| S-EPMC6904897 | biostudies-literature
Project description:BackgroundGout is a common, monosodium urate crystal-driven inflammatory arthritis. Besides its clinical manifestations, patients often also suffer from pain, physical impairment, emotional distress and work productivity loss, as a result of the disease. Patient-reported outcome measures (PROMs) are commonly used to assess these consequences of the disease. However, current instrument endorsements for measuring such outcomes in acute and chronic gout clinical settings are based on limited psychometric evidence. The objective of this systematic literature review was to identify currently available PROMs for gout, and to critically evaluate their content and psychometric properties, in order to evaluate the current status regarding PROMs for use in gout patients.MethodsSystematic literature searches were performed in the PubMed and EMBASE databases. The methodological quality of included papers was appraised using the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) checklist, and evaluation of measurement properties (reliability, responsiveness, construct validity, floor and ceiling effects) was done in accordance with published quality criteria. Item content was appraised by linking health concepts to the International Classification of Functioning Disability and Health (ICF) framework.ResultsIn total, 13 PROMs were identified, of which three were targeted specifically at gout patients. The majority of the PROMs were rated positively for content validity. For most instruments, limited evidence was available for construct validity and reliability. Instruments to assess pain scored well on responsiveness and floor and ceiling effects, but not much is known about their reliability in gout.ConclusionsThe physical functioning subscale of the SF-36v2 (Short Form-36 item version 2) is the only PROM that had sufficient supporting evidence for all its psychometric properties. Many of the commonly used PROMs in gout are currently not yet well supported and more studies on their measurement properties are needed among both acute and chronic gout populations.
| S-EPMC6460780 | biostudies-literature
Project description:BACKGROUND:Patients with cystic fibrosis (CF) can struggle with burdensome symptoms and treatment regimens that negatively affect every aspect of their life. As physiological parameters can fail to capture these complications, the assessment of health-related quality of life (HRQOL) has gained prominence. HRQOL can be measured using standardized patient questionnaires called patient-reported outcome measures (PROMs). The Australian Cystic Fibrosis Data Registry (ACFDR) collects clinical data on adult and pediatric patients with CF. The incorporation of PROMs into the ACFDR would enable monitoring of HRQOL trends, benchmarking of HRQOL outcomes, and support of HRQOL research in CF. OBJECTIVE:Prior to incorporation of a PROM in the ACFDR, this systematic review was planned to evaluate whether any suitable PROMs are currently being used for CF. METHODS:This systematic review will be conducted in compliance with the PRISMA-P (Preferred Reporting Items for Systematic Reviews and Meta-Analyses Protocols) guidelines. MEDLINE, EMBASE, Scopus, CINAHL (Cumulative Index of Nursing and Allied Health Literature), PsycINFO, and Cochrane Library databases were searched for articles published between January 2009 and February 2019 on the use of PROMs to measure HRQOL in adult and pediatric patients with CF. Study designs such as observational studies, reviews and validation studies were included. Studies describing randomized controlled trials, dissertations, books, guideline statements, and abstracts were excluded. The COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) risk of bias checklist was used to assess the methodological quality of included studies. A descriptive synthesis of the results will be undertaken in line with the outcomes of this study. RESULTS:As of July 2019, the search has been conducted and 4530 records were screened. After two phases of screening, 97 studies were included in the final review and subjected to data extraction. Reviewers are currently in the process of critical appraisal. CONCLUSIONS:This review will identify any PROM(s) that may be used to measure HRQOL in the ACFDR. TRIAL REGISTRATION:PROSPERO International Prospective Register of Systematic Reviews CRD42019126931; https://www.crd.york.ac.uk/prospero/display_record.php?RecordID=126931.
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