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Record W4413626650 · doi:10.1111/jebm.70058

A Systematic Survey of the Optimal Strategy for Dealing With Missing Binary Outcomes in Simulation Studies of Randomized Controlled Trials

2025· article· en· W4413626650 on OpenAlex
Yanjiao Shen, Parpia Sameer, X. M. Xia, Yuqing Zhang, Jinhui Ma, Qingyang Shi, Qiukui Hao, Xiaohong Gu, Wenbo He, Yamin Chen, Na Zhang, Le Wang, Y. Zeng, Xiaoyi Su, Qiang Zong, Zhi Qiao, S W Liu, Xinyao Wang, Xinyu Zou, Ying He, Qiong Guo, Liang Du, Zhengchi Li, Jin Huang

Why this work is in the frame

A frame that forgets how it found something cannot be audited. These are the routes that admitted this work.

affAt least one author lists a Canadian institution in the pinned OpenAlex snapshot.

Bibliographic record

VenueJournal of Evidence-Based Medicine · 2025
Typearticle
Languageen
FieldMathematics
TopicStatistical Methods and Bayesian Inference
Canadian institutionsMcMaster UniversityImpact
FundersNational Natural Science Foundation of China
KeywordsMissing dataRandomized controlled trialStatisticsImputation (statistics)Meta-analysisStatistical powerComputer scienceMEDLINEDescriptive statisticsPsychologyEconometricsMedicineMathematics

Abstract

fetched live from OpenAlex

AIM: To summarize the optimal strategies for dealing with missing binary outcome data (MBOD) in randomized controlled trials (RCTs) as informed by simulation studies, and to summarize the quality of reporting in these studies. METHODS: To identify simulation studies comparing at least two strategies to deal with MBOD and evaluating their performance (bias, coverage and power), we searched MEDLINE, EMBASE, Cochrane Central Register of Controlled Trials via Ovid, Web of Science, and JSTOR from their inception up to December 20, 2023. We evaluated reporting quality using established criteria for simulation studies in medical statistics. We summarized data using descriptive statistics and a narrative synthesis. RESULTS: Our search identified 29,460 citations, of which five proved eligible. Multiple imputation (MI), investigated in five studies, showed consistently good performance in all domains tested for missing completely at random (MCAR) and missing at random (MAR) but with important limitations in missing not at random (MNAR). Complete case analysis (CCA), investigated in four studies of which three addressed model-based CCA, performed well in bias and coverage under MAR and MCAR, but less well for MNAR. One study reported that non-model-based CCA performed poorly with respect to bias under MAR. Non-model-based single imputation, investigated in two studies, showed consistently poor performance across all domains tested for MAR, MCAR and MNAR. One study reported that model-based single imputation performed well with respect to bias under MAR. Regarding reporting quality, all studies reported the aims, dependence of simulated data sets, scenarios and statistical methods evaluated, number of simulations performed, justification of data generation and criteria used to evaluate the simulation performance. None of the studies reported the starting seeds, random number generators and failures occurring during simulation. CONCLUSIONS: Simulation studies address methods to deal with MBOD in RCTs, provided evidence that the MI approach is superior with respect to bias and coverage compared with CCA. Non-model-based single imputation generally performed poorly.

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.

Direct model labels (unvalidated)

Per-model category and study-design labels from the labeling rounds. They are machine output, unvalidated, and the disagreement between models ships as data. No study design here is MEDLINE-validated yet.

Model armCategoriesStudy designConfidence
gemmaMetaresearch
Domain: Methods · Genre: Review
About the Canadian research system: no · About a Canadian topic: no
Systematic reviewhigh
gptMetaresearch
Domain: Methods · Genre: Review
About the Canadian research system: no · About a Canadian topic: no
Systematic reviewhigh
models agreeAgreement compares identical category sets and study designs across arms.

Full frame distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.058
metaresearch head score (Gemma)0.529
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesMetaresearch
Consensus categoriesMetaresearch
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Randomized trial · Consensus signal: none
GenreCandidate signal: Methods · Consensus signal: none
Teacher disagreement score0.838
Threshold uncertainty score0.970

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0580.529
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0070.000
Bibliometrics0.0000.000
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0000.000
Research integrity0.0000.000
Insufficient payload (model declined to judge)0.0000.000

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 head0.484
GPT teacher head0.543
Teacher spread0.058 · 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