Inclusion of patient-reported outcome measures in registered clinical trials: Evidence from ClinicalTrials.gov (2007–2013)

Vodicka, Elisabeth; Kim, K.; Devine, Emily Beth; Gnanasakthy, Ari; Scoggins, John F.; Patrick, Donald L.

doi:10.1016/j.cct.2015.04.004

Cited by 178 publications

(117 citation statements)

References 25 publications

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“…Only 26 RCTs appropriately cited CONSORT-PRO during the study period, which represents a minute proportion of RCTs reporting PRO results overall in that period, given 26,337 RCTs with PRO endpoints were registered between 2007-2013 [18], and that we identified 397 RCTs including PROs published 2014-2015 in the 100 top-ranked PRO RCT journals alone.…”

Section: Discussionmentioning

confidence: 99%

Preliminary evidence on the uptake, use and benefits of the CONSORT-PRO extension

et al. 2017

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Section: Discussionmentioning

confidence: 99%

Preliminary evidence on the uptake, use and benefits of the CONSORT-PRO extension

et al. 2017

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“…11 An esophagectomy is a major operation with substantial morbidity and mortality and may have a profound effect on patients' QOL. [12][13][14] However, in the field of esophageal cancer, only limited high-quality data on HRQOL are available.…”

Section: Clinical Trial Information: Ntr487 (Netherlands Trial Registmentioning

confidence: 99%

Effect of Neoadjuvant Chemoradiotherapy on Health-Related Quality of Life in Esophageal or Junctional Cancer: Results From the Randomized CROSS Trial

Noordman

Verdam

Lagarde

et al. 2018

JCO

102

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To compare pre-agreed health-related quality of life (HRQOL) domains in patients with esophageal or junctional cancer who received neoadjuvant chemoradiotherapy (nCRT) followed by surgery or surgery alone. Secondary aims were to examine the effect of nCRT on HRQOL before surgery and the effect of surgery on HRQOL. Patients and MethodsPatients were randomly assigned to nCRT (carboplatin plus paclitaxel with concurrent 41.4-Gy radiotherapy) followed by surgery or surgery alone. HRQOL was measured using the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (QLQ-C30) and -Oesophageal Cancer Module (QLQ-OES24) questionnaires pretreatment and at 3, 6, 9, and 12 months postoperatively. The nCRT group also received preoperative questionnaires. Physical functioning (PF; QLQ-C30) and eating problems (EA; QLQ-OES24) were chosen as predefined primary end points. Predefined secondary end points were global QOL (GQOL; QLQ-C30), fatigue (FA; QLQ-C30), and emotional problems (EM; QLQ-OES24). ResultsA total of 363 patients were analyzed. No statistically significant differences in postoperative HRQOL were found between treatment groups. In the nCRT group, PF, EA, GQOL, FA, and EM scores deteriorated 1 week after nCRT (Cohen's d: 20.93, P , .001; 0.47, P , .001; 20.84, P , .001; 1.45, P , .001; and 0.32, P = .001, respectively). In both treatment groups, all end points declined 3 months postoperatively compared with baseline (Cohen's d: 21.00, 0.33, 20.47, 20.34, and 0.33, respectively; all P , .001), followed by a continuous gradual improvement. EA, GQOL, and EM were restored to baseline levels during follow-up, whereas PF and FA remained impaired 1 year postoperatively (Cohen's d: 0.52 and 20.53, respectively; both P , .001). ConclusionAlthough HRQOL declined during nCRT, no effect of nCRT was apparent on postoperative HRQOL compared with surgery alone. In addition to the improvement in survival, these findings support the view that nCRT according to the Chemoradiotherapy for Esophageal Cancer Followed by Surgery Study-regimen can be regarded as a standard of care.

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“…In lieu of self-report, observer-reported outcomes measures (ObsRO) provide invaluable insight into the patient experience of a disease or therapy, that may be otherwise unobtainable, and complement existing clinical indices. COA data are typically collected via standardized questionnaires designed to measure an explicit concept or construct such as symptoms, activity limitations, and health status/health-related quality of life, and are commonly utilized in randomized controlled trials [3]. Regulatory guidance recommends rigorous development and validation for any COAs used in support of medical labeling.…”

Section: Introductionmentioning

confidence: 99%

Development of a novel observer-reported outcome measure for the assessment of Respiratory Syncytial Virus (RSV) infection symptoms in pediatric clinical trials

Lewis

DeMuro

Block

et al. 2018

J Patient Rep Outcomes

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BackgroundRespiratory syncytial virus (RSV) is a seasonal infection affecting most children by 2 years of age and the leading cause of lower respiratory tract infection requiring hospitalization in infants. Novel antiviral medications are in development to improve the clinical outcomes of RSV; however, no clinical outcome assessments (COAs) for RSV have been developed in alignment with the United States Food and Drug Administration patient-reported outcome guidance to assist in the evaluation of new therapies. To address this need, an observer-reported outcome (ObsRO) measure designed to assess observable RSV symptoms was created.MethodsThe literature was reviewed to evaluate existing COAs and identify constructs of interest. Individual caregiver interviews elicited concepts that informed item development, and candidate items were subsequently evaluated in two rounds of cognitive testing. Separate cohorts of caregivers of RSV-infected nonhospitalized and hospitalized infants participated. Therapeutic-area experts provided input throughout the instrument development process.ResultsCaregivers of 39 children < 24 months old with RSV (31 nonhospitalized, 8 hospitalized) participated in in-depth, individual interviews during concept elicitation and cognitive debriefing, resulting in 21 concepts identified as potentially observable and relevant to young children with RSV. The item pool was reduced to 12 cardinal symptoms and behavior impacts reported to be directly observable by caregivers, with 10 daytime and 9 nighttime symptoms to capture diurnal variation in severity.ConclusionsThe RSV Caregiver Diary assesses RSV symptom severity and change from the parent or caregiver perspective in a standardized manner to measure treatment benefit. Following psychometric evaluation and refinement, this tool is expected to be suitable for assisting in the clinical development of RSV therapeutics.Electronic supplementary materialThe online version of this article (10.1186/s41687-018-0034-9) contains supplementary material, which is available to authorized users.

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Inclusion of patient-reported outcome measures in registered clinical trials: Evidence from ClinicalTrials.gov (2007–2013)

Cited by 178 publications

References 25 publications

Preliminary evidence on the uptake, use and benefits of the CONSORT-PRO extension

Preliminary evidence on the uptake, use and benefits of the CONSORT-PRO extension

Effect of Neoadjuvant Chemoradiotherapy on Health-Related Quality of Life in Esophageal or Junctional Cancer: Results From the Randomized CROSS Trial

Development of a novel observer-reported outcome measure for the assessment of Respiratory Syncytial Virus (RSV) infection symptoms in pediatric clinical trials

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