TY - JOUR
T1 - Systematic Evaluation of Patient-Reported Outcome Protocol Content and Reporting in Cancer Trials
AU - Kyte, Derek
AU - Retzer, Ameeta
AU - Ahmed, Khaled
AU - Keeley, Thomas
AU - Armes, Jo
AU - Brown, Julia M.
AU - Calman, Lynn
AU - Gavin, Anna
AU - Glaser, Adam W.
AU - Greenfield, Diana M.
AU - Lanceley, Anne
AU - Taylor, Rachel M.
AU - Velikova, Galina
AU - Brundage, Michael
AU - Efficace, Fabio
AU - Mercieca-Bebber, Rebecca
AU - King, Madeleine T.
AU - Turner, Grace
AU - Calvert, Melanie
N1 - Publisher Copyright:
© 2019 Published by Oxford University Press.
PY - 2019/11/1
Y1 - 2019/11/1
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=85073601485&partnerID=8YFLogxK
U2 - 10.1093/jnci/djz038
DO - 10.1093/jnci/djz038
M3 - Article
C2 - 30959516
AN - SCOPUS:85073601485
SN - 0027-8874
VL - 111
SP - 1170
EP - 1178
JO - Journal of the National Cancer Institute
JF - Journal of the National Cancer Institute
IS - 11
ER -
