Metaresearch on patient-reported outcomes in trial protocols and results publications suggested large outcome reporting bias

Ala Taji Heravi; Dmitry Gryaznov; Jason W. Busse; Christof Manuel Schönenberger; Belinda von Niederhäusern; Lena Hausheer; Manuela Covino; Johannes M. Schwenke; Selina Epp; Alexandra Griessbach; Malena Chiaborelli; Arnav Agarwal; Szimonetta Lohner; Julian Hirt; Stefan Schandelmaier; Simon B. Egli; Moshao Amos Makhele; Alain Amstutz; Dominik Mertz; Anette Blümle; Erik von Elm; Ramon Saccilotto; Ayodele Odutayo; Sally Hopewell; Benjamin Speich; Matthias Briel; Adherence to SPIRIT Recommendations (ASPIRE) Study Group

doi:10.1016/j.jclinepi.2025.111822

Metaresearch on patient-reported outcomes in trial protocols and results publications suggested large outcome reporting bias

Ala Taji Heravi^*, Dmitry Gryaznov, Jason W. Busse, Christof Manuel Schönenberger, Belinda von Niederhäusern, Lena Hausheer, Manuela Covino, Johannes M. Schwenke, Selina Epp, Alexandra Griessbach, Malena Chiaborelli, Arnav Agarwal, Szimonetta Lohner, Julian Hirt, Stefan Schandelmaier, Simon B. Egli, Moshao Amos Makhele, Alain Amstutz, Dominik Mertz, Anette BlümleErik von Elm, Ramon Saccilotto, Ayodele Odutayo, Sally Hopewell, Benjamin Speich, Matthias Briel, Adherence to SPIRIT Recommendations (ASPIRE) Study Group

^*Corresponding author for this work

Bristol Medical School (PHS)

Research output: Contribution to journal › Article (Academic Journal) › peer-review

4 Citations (Scopus)

Abstract

Objectives:

Patient-reported outcomes (PROs) provide crucial information for evaluating health-care interventions, but previous research in specific disease areas suggested infrequent use and incomplete reporting of PROs. We examined the prevalence and characteristics of PROs in randomized clinical trial (RCT) protocols across medical fields, their reporting quality, and the consistency between PROs specified in trial protocols and subsequent reporting in trial publications.

Study Design and Setting:

We included 237 RCT protocols approved in 2012 and 251 approved in 2016, by ethics committees in Switzerland, Germany, and Canada. We systematically searched for corresponding peer-reviewed results publications and results on trial registries. Pairs of reviewers independently extracted characteristics of RCT protocols, PROs specified in protocols and reported in corresponding results publications, and assessed the reporting quality of RCTs with a PRO as the primary outcome using the Consolidated Standards of Reporting Trials–patient-reported outcome (CONSORTs-PRO) extension.

Results:

Out of 488 included RCT protocols, 147 (30%) did not report use of a PRO; 97 (20%) specified a PRO as the primary outcome and an additional 244 (50%) as a secondary outcome. The prevalence of PROs varied substantially across medical fields, ranging from 100% in rheumatology and psychiatry to about one-third in cardiology and anesthesiology. At 8–10 years after RCT approval, results were available for 264 of the 341 (77%) trial protocols that prespecified PROs. Forty-four percent of the published trials (115/264) reported all PROs as defined in the protocol, 21% (55/264) did not report any prespecified PROs, and 36% (94/264) reported more, fewer, or different PROs than those prespecified. These findings were consistent between trial protocols approved in 2012 and 2016. Among 63 peer-reviewed RCT publications that reported a PRO as their primary outcome, reporting quality was often inadequate, with seven of 13 CONSORT-PRO items.

Conclusion:

Less than half of RCT protocols with planned PROs reported them as specified in corresponding published results, suggesting outcome reporting bias, and PRO reporting quality was often deficient. These limitations complicate informed decision-making between patients and health-care providers, as well as the development of evidence-based clinical practice guidelines.

Original language	English
Article number	111822
Number of pages	10
Journal	Journal of Clinical Epidemiology
Volume	185
Early online date	9 May 2025
DOIs	https://doi.org/10.1016/j.jclinepi.2025.111822
Publication status	Published - 1 Sept 2025

Bibliographical note

Publisher Copyright:
© 2025 The Author(s)

Keywords

Outcome reporting bias
Patient-reported outcomes
Randomized clinical trials
Reporting guidelines
Trial protocols
Trial publications

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Taji Heravi, A., Gryaznov, D., Busse, J. W., Schönenberger, C. M., von Niederhäusern, B., Hausheer, L., Covino, M., Schwenke, J. M., Epp, S., Griessbach, A., Chiaborelli, M., Agarwal, A., Lohner, S., Hirt, J., Schandelmaier, S., Egli, S. B., Makhele, M. A., Amstutz, A., Mertz, D., ... Adherence to SPIRIT Recommendations (ASPIRE) Study Group (2025). Metaresearch on patient-reported outcomes in trial protocols and results publications suggested large outcome reporting bias. Journal of Clinical Epidemiology, 185, Article 111822. https://doi.org/10.1016/j.jclinepi.2025.111822

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title = "Metaresearch on patient-reported outcomes in trial protocols and results publications suggested large outcome reporting bias",

abstract = "Objectives:Patient-reported outcomes (PROs) provide crucial information for evaluating health-care interventions, but previous research in specific disease areas suggested infrequent use and incomplete reporting of PROs. We examined the prevalence and characteristics of PROs in randomized clinical trial (RCT) protocols across medical fields, their reporting quality, and the consistency between PROs specified in trial protocols and subsequent reporting in trial publications. Study Design and Setting:We included 237 RCT protocols approved in 2012 and 251 approved in 2016, by ethics committees in Switzerland, Germany, and Canada. We systematically searched for corresponding peer-reviewed results publications and results on trial registries. Pairs of reviewers independently extracted characteristics of RCT protocols, PROs specified in protocols and reported in corresponding results publications, and assessed the reporting quality of RCTs with a PRO as the primary outcome using the Consolidated Standards of Reporting Trials–patient-reported outcome (CONSORTs-PRO) extension. Results:Out of 488 included RCT protocols, 147 (30\%) did not report use of a PRO; 97 (20\%) specified a PRO as the primary outcome and an additional 244 (50\%) as a secondary outcome. The prevalence of PROs varied substantially across medical fields, ranging from 100\% in rheumatology and psychiatry to about one-third in cardiology and anesthesiology. At 8–10 years after RCT approval, results were available for 264 of the 341 (77\%) trial protocols that prespecified PROs. Forty-four percent of the published trials (115/264) reported all PROs as defined in the protocol, 21\% (55/264) did not report any prespecified PROs, and 36\% (94/264) reported more, fewer, or different PROs than those prespecified. These findings were consistent between trial protocols approved in 2012 and 2016. Among 63 peer-reviewed RCT publications that reported a PRO as their primary outcome, reporting quality was often inadequate, with seven of 13 CONSORT-PRO items. Conclusion:Less than half of RCT protocols with planned PROs reported them as specified in corresponding published results, suggesting outcome reporting bias, and PRO reporting quality was often deficient. These limitations complicate informed decision-making between patients and health-care providers, as well as the development of evidence-based clinical practice guidelines.",

keywords = "Outcome reporting bias, Patient-reported outcomes, Randomized clinical trials, Reporting guidelines, Trial protocols, Trial publications",

author = "\{Taji Heravi\}, Ala and Dmitry Gryaznov and Busse, \{Jason W.\} and Sch{\"o}nenberger, \{Christof Manuel\} and \{von Niederh{\"a}usern\}, Belinda and Lena Hausheer and Manuela Covino and Schwenke, \{Johannes M.\} and Selina Epp and Alexandra Griessbach and Malena Chiaborelli and Arnav Agarwal and Szimonetta Lohner and Julian Hirt and Stefan Schandelmaier and Egli, \{Simon B.\} and Makhele, \{Moshao Amos\} and Alain Amstutz and Dominik Mertz and Anette Bl{\"u}mle and \{von Elm\}, Erik and Ramon Saccilotto and Ayodele Odutayo and Sally Hopewell and Benjamin Speich and Matthias Briel and \{Adherence to SPIRIT Recommendations (ASPIRE) Study Group\}",

note = "Publisher Copyright: {\textcopyright} 2025 The Author(s)",

year = "2025",

month = sep,

day = "1",

doi = "10.1016/j.jclinepi.2025.111822",

language = "English",

volume = "185",

journal = "Journal of Clinical Epidemiology",

issn = "0895-4356",

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Taji Heravi, A, Gryaznov, D, Busse, JW, Schönenberger, CM, von Niederhäusern, B, Hausheer, L, Covino, M, Schwenke, JM, Epp, S, Griessbach, A, Chiaborelli, M, Agarwal, A, Lohner, S, Hirt, J, Schandelmaier, S, Egli, SB, Makhele, MA, Amstutz, A, Mertz, D, Blümle, A, von Elm, E, Saccilotto, R, Odutayo, A, Hopewell, S, Speich, B, Briel, M & Adherence to SPIRIT Recommendations (ASPIRE) Study Group 2025, 'Metaresearch on patient-reported outcomes in trial protocols and results publications suggested large outcome reporting bias', Journal of Clinical Epidemiology, vol. 185, 111822. https://doi.org/10.1016/j.jclinepi.2025.111822

TY - JOUR

T1 - Metaresearch on patient-reported outcomes in trial protocols and results publications suggested large outcome reporting bias

AU - Taji Heravi, Ala

AU - Gryaznov, Dmitry

AU - Busse, Jason W.

AU - Schönenberger, Christof Manuel

AU - von Niederhäusern, Belinda

AU - Hausheer, Lena

AU - Covino, Manuela

AU - Schwenke, Johannes M.

AU - Epp, Selina

AU - Griessbach, Alexandra

AU - Chiaborelli, Malena

AU - Agarwal, Arnav

AU - Lohner, Szimonetta

AU - Hirt, Julian

AU - Schandelmaier, Stefan

AU - Egli, Simon B.

AU - Makhele, Moshao Amos

AU - Amstutz, Alain

AU - Mertz, Dominik

AU - Blümle, Anette

AU - von Elm, Erik

AU - Saccilotto, Ramon

AU - Odutayo, Ayodele

AU - Hopewell, Sally

AU - Speich, Benjamin

AU - Briel, Matthias

AU - Adherence to SPIRIT Recommendations (ASPIRE) Study Group

PY - 2025/9/1

Y1 - 2025/9/1

N2 - Objectives:Patient-reported outcomes (PROs) provide crucial information for evaluating health-care interventions, but previous research in specific disease areas suggested infrequent use and incomplete reporting of PROs. We examined the prevalence and characteristics of PROs in randomized clinical trial (RCT) protocols across medical fields, their reporting quality, and the consistency between PROs specified in trial protocols and subsequent reporting in trial publications. Study Design and Setting:We included 237 RCT protocols approved in 2012 and 251 approved in 2016, by ethics committees in Switzerland, Germany, and Canada. We systematically searched for corresponding peer-reviewed results publications and results on trial registries. Pairs of reviewers independently extracted characteristics of RCT protocols, PROs specified in protocols and reported in corresponding results publications, and assessed the reporting quality of RCTs with a PRO as the primary outcome using the Consolidated Standards of Reporting Trials–patient-reported outcome (CONSORTs-PRO) extension. Results:Out of 488 included RCT protocols, 147 (30%) did not report use of a PRO; 97 (20%) specified a PRO as the primary outcome and an additional 244 (50%) as a secondary outcome. The prevalence of PROs varied substantially across medical fields, ranging from 100% in rheumatology and psychiatry to about one-third in cardiology and anesthesiology. At 8–10 years after RCT approval, results were available for 264 of the 341 (77%) trial protocols that prespecified PROs. Forty-four percent of the published trials (115/264) reported all PROs as defined in the protocol, 21% (55/264) did not report any prespecified PROs, and 36% (94/264) reported more, fewer, or different PROs than those prespecified. These findings were consistent between trial protocols approved in 2012 and 2016. Among 63 peer-reviewed RCT publications that reported a PRO as their primary outcome, reporting quality was often inadequate, with seven of 13 CONSORT-PRO items. Conclusion:Less than half of RCT protocols with planned PROs reported them as specified in corresponding published results, suggesting outcome reporting bias, and PRO reporting quality was often deficient. These limitations complicate informed decision-making between patients and health-care providers, as well as the development of evidence-based clinical practice guidelines.

AB - Objectives:Patient-reported outcomes (PROs) provide crucial information for evaluating health-care interventions, but previous research in specific disease areas suggested infrequent use and incomplete reporting of PROs. We examined the prevalence and characteristics of PROs in randomized clinical trial (RCT) protocols across medical fields, their reporting quality, and the consistency between PROs specified in trial protocols and subsequent reporting in trial publications. Study Design and Setting:We included 237 RCT protocols approved in 2012 and 251 approved in 2016, by ethics committees in Switzerland, Germany, and Canada. We systematically searched for corresponding peer-reviewed results publications and results on trial registries. Pairs of reviewers independently extracted characteristics of RCT protocols, PROs specified in protocols and reported in corresponding results publications, and assessed the reporting quality of RCTs with a PRO as the primary outcome using the Consolidated Standards of Reporting Trials–patient-reported outcome (CONSORTs-PRO) extension. Results:Out of 488 included RCT protocols, 147 (30%) did not report use of a PRO; 97 (20%) specified a PRO as the primary outcome and an additional 244 (50%) as a secondary outcome. The prevalence of PROs varied substantially across medical fields, ranging from 100% in rheumatology and psychiatry to about one-third in cardiology and anesthesiology. At 8–10 years after RCT approval, results were available for 264 of the 341 (77%) trial protocols that prespecified PROs. Forty-four percent of the published trials (115/264) reported all PROs as defined in the protocol, 21% (55/264) did not report any prespecified PROs, and 36% (94/264) reported more, fewer, or different PROs than those prespecified. These findings were consistent between trial protocols approved in 2012 and 2016. Among 63 peer-reviewed RCT publications that reported a PRO as their primary outcome, reporting quality was often inadequate, with seven of 13 CONSORT-PRO items. Conclusion:Less than half of RCT protocols with planned PROs reported them as specified in corresponding published results, suggesting outcome reporting bias, and PRO reporting quality was often deficient. These limitations complicate informed decision-making between patients and health-care providers, as well as the development of evidence-based clinical practice guidelines.

KW - Outcome reporting bias

KW - Patient-reported outcomes

KW - Randomized clinical trials

KW - Reporting guidelines

KW - Trial protocols

KW - Trial publications

U2 - 10.1016/j.jclinepi.2025.111822

DO - 10.1016/j.jclinepi.2025.111822

M3 - Article (Academic Journal)

C2 - 40349736

AN - SCOPUS:105009776119

SN - 0895-4356

VL - 185

JO - Journal of Clinical Epidemiology

JF - Journal of Clinical Epidemiology

M1 - 111822

ER -

Metaresearch on patient-reported outcomes in trial protocols and results publications suggested large outcome reporting bias

Abstract

Bibliographical note

Keywords

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