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Record W4401797296 · doi:10.1093/jrsssb/qkae084

A nonparametric framework for treatment effect modifier discovery in high dimensions

2024· article· en· W4401797296 on OpenAlexfundno aff
Philippe Boileau, Ning Leng, Nima S. Hejazi, Mark van der Laan, Sandrine Dudoit

Bibliographic record

VenueJournal of the Royal Statistical Society Series B (Statistical Methodology) · 2024
Typearticle
Languageen
FieldMathematics
TopicStatistical Methods in Clinical Trials
Canadian institutionsnot available
FundersNatural Sciences and Engineering Research Council of Canada
KeywordsNonparametric statisticsDrug discoveryComputational biologyComputer sciencePsychologyEconometricsMathematicsBiologyBioinformatics

Abstract

fetched live from OpenAlex

Abstract Heterogeneous treatment effects are driven by treatment effect modifiers (TEMs), pretreatment covariates that modify the effect of a treatment on an outcome. Current approaches for uncovering these variables are limited to low-dimensional data, data with weakly correlated covariates, or data generated according to parametric processes. We resolve these issues by proposing a framework for defining model-agnostic TEM variable importance parameters (TEM-VIPs), deriving one-step, estimating equation, and targeted maximum likelihood estimators of these parameters, and establishing these estimators’ asymptotic properties. This framework is showcased by defining TEM-VIPs for data-generating processes with continuous, binary, and time-to-event outcomes with binary treatments, and deriving accompanying asymptotically linear estimators. Simulation experiments demonstrate that these estimators’ asymptotic guarantees are approximately achieved in realistic sample sizes in randomized and observational studies alike. This methodology is also applied to gene expression data collected in a clinical trial assessing the effect of a novel therapy on disease-free survival in breast cancer patients. Predicted TEMs have previously been linked to treatment resistance.

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 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)0.011
metaresearch head score (Gemma)0.328
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesMetaresearch, Meta-epidemiology (narrow)
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Theoretical or conceptual · Consensus signal: Theoretical or conceptual
GenreCandidate signal: Methods · Consensus signal: Methods
Teacher disagreement score0.364
Threshold uncertainty score1.000

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0110.328
Meta-epidemiology (narrow)0.0010.000
Meta-epidemiology (broad)0.0030.001
Bibliometrics0.0000.001
Science and technology studies0.0000.001
Scholarly communication0.0000.000
Open science0.0010.000
Research integrity0.0010.001
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 head0.362
GPT teacher head0.529
Teacher spread0.167 · 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; a candidate call from one teacher head, not a consensus.

Study designTheoretical or conceptual
Domainnot available
GenreMethods

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

Citations4
Published2024
Admission routes1
Has abstractyes

Explore more

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