Evidence map›Paper›PMID 28830464›Full record

SynthesisBMC medical research methodology2017

Using Bayesian statistics to estimate the likelihood a new trial will demonstrate the efficacy of a new treatment.

David J Biau, Samuel Boulezaz, Laurent Casabianca, Moussa Hamadouche, Philippe Anract, Sylvie Chevret

Abstract readMeta-Analysis
In one paragraph

Synthesis in BMC medical research methodology, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed, 1 pooled it
–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

5 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
  3. Article
  4. Article
  5. Article
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

6 authors.

David J BiauINSERM U1153, Paris, France. david.biau@aphp.fr.
Samuel BoulezazService de chirurgie orthopédique,Hôpital Cochin, 27 rue du faubourg Saint-Jacques, 75014, Paris, France.
Laurent CasabiancaService de chirurgie orthopédique,Hôpital Cochin, 27 rue du faubourg Saint-Jacques, 75014, Paris, France.
Moussa HamadoucheService de chirurgie orthopédique,Hôpital Cochin, 27 rue du faubourg Saint-Jacques, 75014, Paris, France.
Philippe AnractINSERM U1153, Paris, France.
Sylvie ChevretINSERM U1153, Paris, France.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundThe common frequentist approach is limited in providing investigators with appropriate measures for conducting a new trial. To answer such important questions and one has to look at Bayesian statistics.

methodsAs a worked example, we conducted a Bayesian cumulative meta-analysis to summarize the benefit of patient-specific instrumentation on the alignment of total knee replacement from previously published evidence. Data were sourced from Medline, Embase, and Cochrane databases. All randomised controlled comparisons of the effect of patient-specific instrumentation on the coronal alignment of total knee replacement were included. The main outcome was the risk difference measured by the proportion of failures in the control group minus the proportion of failures in the experimental group. Through Bayesian statistics, we estimated cumulatively over publication time of the trial results: the posterior probabilities that the risk difference was more than 5 and 10%; the posterior probabilities that given the results of all previous published trials an additional fictive trial would achieve a risk difference of at least 5%; and the predictive probabilities that observed failure rate differ from 5% across arms.

resultsThirteen trials were identified including 1092 patients, 554 in the experimental group and 538 in the control group. The cumulative mean risk difference was 0.5% (95% CrI: -5.7%; +4.5%). The posterior probabilities that the risk difference be superior to 5 and 10% was less than 5% after trial #4 and trial #2 respectively. The predictive probability that the difference in failure rates was at least 5% dropped from 45% after the first trial down to 11% after the 13th. Last, only unrealistic trial design parameters could change the overall evidence accumulated to date.

conclusionsBayesian probabilities are readily understandable when discussing the relevance of performing a new trial. It provides investigators the current probability that an experimental treatment be superior to a reference treatment. In case a trial is designed, it also provides the predictive probability that this new trial will reach the targeted risk difference in failure rates.

trial registrationCRD42015024176 .

Indexed as

Clinical Trials as TopicBayes TheoremBiasHumansLikelihood FunctionsTreatment OutcomeBayesian statisticsCumulativeDirect probabilityMeta-analysisPosterior probabilityPredictive probabilitySuperiority

Identifiers

PMID28830464
PMCPMC5568256

What OpenQuestion holds

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LicenceCC BY
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Registered trials

None linked

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.