Impact of Blinding on Patient-Reported Outcome Differences Between Treatment Arms in Cancer Randomized Controlled Trials

2021 ◽  
Author(s):  
Fabio Efficace ◽  
David Cella ◽  
Neil K Aaronson ◽  
Melanie Calvert ◽  
Francesco Cottone ◽  
...  
Keyword(s):  
Randomized Controlled Trials ◽  
Experimental Treatment ◽  
Patient Reported Outcome ◽  
Controlled Trials ◽  
Open Label ◽  
Multivariable Logistic Regression Model ◽  
Randomized Controlled ◽  
Patient Reported ◽  
Independent Variable ◽  
Outcome Differences

Abstract Some concerns have been raised about potential bias in patient-reported outcome (PRO) results from open-label cancer randomized controlled trials (RCTs). We investigated if open-label trials favor the experimental treatment over the standard treatment more frequently than blinded trials. We also examined if the effect of blinding differs for distal vs more proximal PROs. We assessed 538 RCTs with a PRO endpoint conducted in the most prevalent cancers, of which 366 (68.0%) were open-label, 148 (27.5%) were blinded, and 24 (4.5%) were categorized as unclear. In our multivariable logistic regression model, we did not observe a statistically significant association of the independent variable treatment concealment (blinded vs open-label) on the dependent variable measuring the proportion of trials favoring the experimental treatment (adjusted odds ratio = 1.19, 95% confidence interval = 0.79 to 1.79; 2-sided P = .40). This was also the case when comparing distal and proximal PROs. Our findings provide novel evidence-based data that support the validity of PRO results from open-label cancer RCTs.

scholarly journals Investigating the impact of open label design on patient‐reported outcome results in prostate cancer randomized controlled trials

2020 ◽  
Vol 9 (20) ◽  
pp. 7363-7374
Author(s):  
Guillaume Mouillet ◽  
Fabio Efficace ◽  
Antoine Thiery‐Vuillemin ◽  
Emilie Charton ◽  
Mieke Van Hemelrijck ◽  
...  
Keyword(s):  
Prostate Cancer ◽  
Randomized Controlled Trials ◽  
Patient Reported Outcome ◽  
Controlled Trials ◽  
Open Label ◽  
Randomized Controlled ◽  
Patient Reported ◽  
The Impact

scholarly journals Factors in Randomized Controlled Trials Reported to Impact the Implementation of Patient-Reported Outcome Measures Into Routine Care: Protocol for a Systematic Review (Preprint)

2019 ◽  
Author(s):  
Natasha Anne Roberts ◽  
Kimberly Alexander ◽  
David Wyld ◽  
Monika Janda
Keyword(s):  
Systematic Review ◽  
Clinical Practice ◽  
Randomized Controlled Trials ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Controlled Trials ◽  
Evidence Based ◽  
Randomized Controlled ◽  
Patient Reported ◽  
Implementation Factors

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.

scholarly journals Factors in Randomized Controlled Trials Reported to Impact the Implementation of Patient-Reported Outcome Measures Into Routine Care: Protocol for a Systematic Review

10.2196/14579 ◽  
2019 ◽  
Vol 8 (11) ◽  
pp. e14579
Author(s):  
Natasha Anne Roberts ◽  
Kimberly Alexander ◽  
David Wyld ◽  
Monika Janda
Keyword(s):  
Systematic Review ◽  
Clinical Practice ◽  
Randomized Controlled Trials ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Controlled Trials ◽  
Evidence Based ◽  
Randomized Controlled ◽  
Patient Reported ◽  
Implementation Factors

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

The Duty to Rescue and Randomized Controlled Trials Involving Serious Diseases

2018 ◽  
Vol 15 (3) ◽  
pp. 298-323
Author(s):  
Joseph Millum ◽  
David Wendler
Keyword(s):  
Medical Research ◽  
Randomized Controlled Trials ◽  
Experimental Treatment ◽  
Controlled Trials ◽  
Potential Treatment ◽  
Randomized Controlled ◽  
Experimental Treatments ◽  
Effective Treatments

During the recent Ebola epidemic, some commentators and stakeholders argued that it would be unethical to carry out a study that withheld a potential treatment from affected individuals with such a serious, untreatable disease. As a result, the initial trials of experimental treatments did not have control arms, despite important scientific reasons for their inclusion. In this paper, we consider whether the duty to rescue entails that it would be unethical to withhold an experimental treatment from patient-participants with serious diseases for which there are no effective treatments, even when doing so is scientifically necessary to test the effectiveness of the treatment. We argue that the duty to rescue will rarely apply. The context of medical research also throws new light on the content of the duty to rescue, since the interests of future patients—who stand to benefit from the fruits of medical research—are relevant to whether the duty applies.

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