Evidence map›Paper›PMID 31651877›Full record

ArticleMedicine2019

A test for treatment effects in randomized controlled trials, harnessing the power of ultrahigh dimensional big data.

Wen-Chung Lee, Jui-Hsiang Lin

Abstract read
In one paragraph

Article in Medicine, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Observational
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

2 authors.

Wen-Chung LeeInstitute of Epidemiology and Preventive Medicine, College of Public Health, National Taiwan University, Taipei, Taiwan.
Jui-Hsiang Lin

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundThe randomized controlled trial (RCT) is the gold-standard research design in biomedicine. However, practical concerns often limit the sample size, n, the number of patients in a RCT. We aim to show that the power of a RCT can be increased by increasing p, the number of baseline covariates (sex, age, socio-demographic, genomic, and clinical profiles et al, of the patients) collected in the RCT (referred to as the 'dimension').

methodsThe conventional test for treatment effects is based on testing the 'crude null' that the outcomes of the subjects are of no difference between the two arms of a RCT. We propose a 'high-dimensional test' which is based on testing the 'sharp null' that the experimental intervention has no treatment effect whatsoever, for patients of any covariate profile.

resultsUsing computer simulations, we show that the high-dimensional test can become very powerful in detecting treatment effects for very large p, but not so for small or moderate p. Using a real dataset, we demonstrate that the P value of the high-dimensional test decreases as the number of baseline covariates increases, though it is still not significant.

conclusionIn this big-data era, pushing p of a RCT to the millions, billions, or even trillions may someday become feasible. And the high-dimensional test proposed in this study can become very powerful in detecting treatment effects.

Indexed as

Big DataRandomized Controlled Trials as TopicComputer SimulationData Interpretation, StatisticalHumansOutcome Assessment, Health CareSample Size

Identifiers

PMID31651877
PMCPMC6824789

What OpenQuestion holds

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LicenceCC BY-NC
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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.