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Definitions, acceptability, limitations, and guidance in the use and reporting of surrogate end points in trials: a scoping review

2023· review· en· W4382045038 on OpenAlexaff
Anthony Muchai Manyara, Philippa Davies, Derek Stewart, Christopher J. Weir, Amber Young, Valerie Wells, Jane Blazeby, Nancy J. Butcher, Sylwia Bujkiewicz, An‐Wen Chan, Gary S. Collins, Dalia Dawoud, Martin Offringa, Mario Ouwens, Joseph S. Ross, Rod S Taylor, Oriana Ciani

Bibliographic record

VenueJournal of Clinical Epidemiology · 2023
Typereview
Languageen
FieldEconomics, Econometrics and Finance
TopicHealth Systems, Economic Evaluations, Quality of Life
Canadian institutionsInstitute for Clinical Evaluative SciencesSickKids FoundationHospital for Sick ChildrenWomen's College HospitalUniversity of Toronto
FundersNational Heart, Lung, and Blood InstituteMedical Research CouncilAgency for Healthcare Research and QualityCancer Research UKUniversity of BristolNational Institute for Health and Care ResearchNIHR Bristol Biomedical Research CentreManchester Biomedical Research CentreUK Research and Innovation
KeywordsMedicineMedical physicsMEDLINEPolitical science

Abstract

fetched live from OpenAlex

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.

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

How this classification was reachedexpand

Full frame machine prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.

metaresearch head score (Codex)0.687
metaresearch head score (Gemma)0.848
Version: metacan-v3-hybrid-931329e0061cValidation status: machine_predicted_unvalidated
Candidate categoriesMetaresearch
Consensus categoriesMetaresearch
DomainCandidate signal: Reporting · Consensus signal: none
Study designCandidate signal: Systematic review · Consensus signal: Systematic review
GenreCandidate signal: Review · Consensus signal: Review
Teacher disagreement score0.313
Threshold uncertainty score0.386

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.6870.848
Meta-epidemiology (narrow)0.0030.005
Meta-epidemiology (broad)0.0110.012
Bibliometrics0.0370.034
Science and technology studies0.0060.015
Scholarly communication0.0270.031
Open science0.0100.014
Research integrity0.0160.014
Insufficient payload (model declined to judge)0.0040.002

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.984
GPT teacher head0.717
Teacher spread0.267 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; the direct Gemma label and the distilled Codex classifier agree on what is shown here.

Study designSystematic review
DomainReporting
GenreReview

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations29
Published2023
Admission routes1
Has abstractyes

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