Definitions, acceptability, limitations, and guidance in the use and reporting of surrogate end points in trials : a scoping review
Copyright © 2023 The Author(s). Published by Elsevier Inc. All rights reserved..
OBJECTIVE: To synthesize the current literature on the use of surrogate end points, including definitions, acceptability, and limitations of surrogate end points and guidance for their design/reporting, into trial reporting items.
STUDY DESIGN AND SETTING: Literature was identified through searching bibliographic databases (until March 1, 2022) and gray literature sources (until May 27, 2022). Data were thematically analyzed into four categories: (1) definitions, (2) acceptability, (3) limitations and challenges, and (4) guidance, and synthesized into reporting guidance items.
RESULTS: After screening, 90 documents were included: 79% (n = 71) had data on definitions, 77% (n = 69) on acceptability, 72% (n = 65) on limitations and challenges, and 61% (n = 55) on guidance. Data were synthesized into 17 potential trial reporting items: explicit statements on the use of surrogate end point(s) and justification for their use (items 1-6); methodological considerations, including whether sample size calculations were informed by surrogate validity (items 7-9); reporting of results for composite outcomes containing a surrogate end point (item 10); discussion and interpretation of findings (items 11-14); plans for confirmatory studies, collecting data on the surrogate end point and target outcome, and data sharing (items 15-16); and informing trial participants about using surrogate end points (item 17).
CONCLUSION: The review identified and synthesized items on the use of surrogate end points in trials; these will inform the development of the Standard Protocol Items: Recommendations for Interventional Trials-SURROGATE and Consolidated Standards of Reporting Trials-SURROGATE extensions.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2023 |
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| Erschienen: |
2023 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:160 |
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| Enthalten in: |
Journal of clinical epidemiology - 160(2023) vom: 15. Aug., Seite 83-99 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Manyara, Anthony Muchai [VerfasserIn] |
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| Links: |
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| Anmerkungen: |
Date Completed 01.09.2023 Date Revised 13.03.2024 published: Print-Electronic Citation Status MEDLINE |
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| doi: |
10.1016/j.jclinepi.2023.06.013 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM358814103 |
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| 100 | 1 | |a Manyara, Anthony Muchai |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Definitions, acceptability, limitations, and guidance in the use and reporting of surrogate end points in trials |b a scoping review |
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| 500 | |a Date Completed 01.09.2023 | ||
| 500 | |a Date Revised 13.03.2024 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a Copyright © 2023 The Author(s). Published by Elsevier Inc. All rights reserved. | ||
| 520 | |a OBJECTIVE: To synthesize the current literature on the use of surrogate end points, including definitions, acceptability, and limitations of surrogate end points and guidance for their design/reporting, into trial reporting items | ||
| 520 | |a STUDY DESIGN AND SETTING: Literature was identified through searching bibliographic databases (until March 1, 2022) and gray literature sources (until May 27, 2022). Data were thematically analyzed into four categories: (1) definitions, (2) acceptability, (3) limitations and challenges, and (4) guidance, and synthesized into reporting guidance items | ||
| 520 | |a RESULTS: After screening, 90 documents were included: 79% (n = 71) had data on definitions, 77% (n = 69) on acceptability, 72% (n = 65) on limitations and challenges, and 61% (n = 55) on guidance. Data were synthesized into 17 potential trial reporting items: explicit statements on the use of surrogate end point(s) and justification for their use (items 1-6); methodological considerations, including whether sample size calculations were informed by surrogate validity (items 7-9); reporting of results for composite outcomes containing a surrogate end point (item 10); discussion and interpretation of findings (items 11-14); plans for confirmatory studies, collecting data on the surrogate end point and target outcome, and data sharing (items 15-16); and informing trial participants about using surrogate end points (item 17) | ||
| 520 | |a CONCLUSION: The review identified and synthesized items on the use of surrogate end points in trials; these will inform the development of the Standard Protocol Items: Recommendations for Interventional Trials-SURROGATE and Consolidated Standards of Reporting Trials-SURROGATE extensions | ||
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| 700 | 1 | |a Stewart, Derek |e verfasserin |4 aut | |
| 700 | 1 | |a Weir, Christopher J |e verfasserin |4 aut | |
| 700 | 1 | |a Young, Amber E |e verfasserin |4 aut | |
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| 700 | 1 | |a Chan, An-Wen |e verfasserin |4 aut | |
| 700 | 1 | |a Collins, Gary S |e verfasserin |4 aut | |
| 700 | 1 | |a Dawoud, Dalia |e verfasserin |4 aut | |
| 700 | 1 | |a Offringa, Martin |e verfasserin |4 aut | |
| 700 | 1 | |a Ouwens, Mario |e verfasserin |4 aut | |
| 700 | 1 | |a Ross, Joseph S |e verfasserin |4 aut | |
| 700 | 1 | |a Taylor, Rod S |e verfasserin |4 aut | |
| 700 | 1 | |a Ciani, Oriana |e verfasserin |4 aut | |
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