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Inequality in treatment benefits: Can we determine if a new treatment benefits the many or the few?

2016· article· en· W939145883 on OpenAlex

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.

fundA Canadian funder is recorded on the work.
no affNo Canadian affiliation: this work is invisible to an affiliation-only frame.
No Canadian affiliation. An affiliation-only frame, the usual design, would never have seen this work. It is one of the works that make the case for inverting the frame.

Bibliographic record

VenueBiostatistics · 2016
Typearticle
Languageen
FieldMathematics
TopicAdvanced Causal Inference Techniques
Canadian institutionsnot available
FundersNational Institute of Neurological Disorders and StrokePatient-Centered Outcomes Research InstituteU.S. Food and Drug AdministrationNational Institute on AgingNational Institutes of HealthHamilton Health Sciences Foundation
KeywordsRandomized controlled trialEstimatorFraction (chemistry)Computer scienceStatisticsEconometricsRandomized responseOutcome (game theory)MathematicsMedicineMathematical optimizationMathematical economics

Abstract

fetched live from OpenAlex

In many randomized controlled trials, the primary analysis focuses on the average treatment effect and does not address whether treatment benefits are widespread or limited to a select few. This problem affects many disease areas, since it stems from how randomized trials, often the gold standard for evaluating treatments, are designed and analyzed. Our goal is to learn about the fraction who benefit from a new treatment using randomized trial data. We consider the case where the outcome is ordinal, with binary outcomes as a special case. In general, the fraction who benefit is non-identifiable, and the best that can be obtained are sharp lower and upper bounds. Our contributions include (i) proving the plug-in estimator of the bounds can be inconsistent if support restrictions are made on the joint distribution of the potential outcomes; (ii) developing the first consistent estimator for this case; and (iii) applying this estimator to a randomized trial of a medical treatment to determine whether the estimates can be informative. Our estimator is computed using linear programming, allowing fast implementation. R code is provided.

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.

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)0.000
metaresearch head score (Gemma)0.001
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Theoretical or conceptual · Consensus signal: none
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.776
Threshold uncertainty score0.690

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0000.001
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0000.000
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 head0.250
GPT teacher head0.405
Teacher spread0.155 · 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