Evidence map›Paper›PMID 41642106›Full record

ArticleEpilepsia2026

Continuous outcome estimation in N-of-1 trials for accelerated decision-making.

Victoria Defelippe, František Bartoš, Eric-Jan Wagenmakers, Kees P J Braun, Floor E Jansen, Willem M Otte

Abstract read
In one paragraph

Article in Epilepsia, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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0citing papers in PubMed
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1 · What the graph read from it

What it found

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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

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3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

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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.

Victoria DefelippeDepartment of Pediatric Neurology, UMC Utrecht Brain Center, Member of ERN EpiCare, University Medical Center Utrecht and Utrecht University, Utrecht, The Netherlands.ORCID https://orcid.org/0000-0002-3080-5008
František BartošDepartment of Psychology, University of Amsterdam, Amsterdam, The Netherlands.ORCID https://orcid.org/0000-0002-0018-5573
Eric-Jan WagenmakersDepartment of Psychology, University of Amsterdam, Amsterdam, The Netherlands.
Kees P J BraunDepartment of Pediatric Neurology, UMC Utrecht Brain Center, Member of ERN EpiCare, University Medical Center Utrecht and Utrecht University, Utrecht, The Netherlands.
Floor E JansenDepartment of Pediatric Neurology, UMC Utrecht Brain Center, Member of ERN EpiCare, University Medical Center Utrecht and Utrecht University, Utrecht, The Netherlands.
Willem M OtteDepartment of Pediatric Neurology, UMC Utrecht Brain Center, Member of ERN EpiCare, University Medical Center Utrecht and Utrecht University, Utrecht, The Netherlands.

Funding

Health-Holland LSHM21053-SGF
6 · The paper itself

Abstract

objectiveN-of-1 trials aim to determine the therapeutic effect for a single individual. This individualized approach necessitates collecting multiple data points over time through repeated alternating periods of active treatment and a comparator or control condition. The extended duration of the treatment periods may increase patient burden, prolong placebo exposure, and increase the likelihood of study discontinuation. In theory, treatment responders (or non-responders) can be identified early during the trial if the therapeutic effect is strong (or completely lacking). There are no theoretical constraints to evaluate treatment efficacy more regularly-instead of only after a predetermined number of treatment periods. Regularly updating estimates on treatment effects allows clinicians to accelerate clinical decision-making regarding N-of-1 study termination. This study examined the value of continuous treatment effect estimation using Bayesian hypothesis testing in N-of-1 trials to accelerate and nuance clinical decision-making.

methodsAn N-of-1 trial with severe epilepsy was simulated and three N-of-1 trials in neurological conditions were (re-)analyzed continuously with consecutive data points using Bayesian hypothesis testing and/or a minimally clinically important threshold (30% seizure frequency reduction). Trial duration based on Bayesian testing with strong evidence for treatment effects was compared to original trial duration.

resultsOriginal trial duration could be reduced between 9.5% and 35% of the trial length by using continuous outcome estimation in two of the analyzed trial examples. The moment that strong evidence supporting beneficial treatment effects using Bayesian hypothesis testing and a significant probability of minimally clinically important differences are achieved during the trial may differ. Obtaining additional data points and alternating interventions over time improve certainty of the estimates of treatment effects. SIGNIFICANCE: Treatment efficacy decisions can be expedited when outcome estimation is performed continuously rather than delayed until the end of the trial. Clinical significance of N-of-1 trial outcome can be improved combining both Bayesian hypothesis testing and a minimally clinically important threshold.

Indexed as

AnticonvulsantsClinical Decision-MakingEpilepsyBayes TheoremHumansTreatment OutcomeAnticonvulsantsadaptive clinical trialBayesianepilepsyN‐of‐1 trialseizuresstatistics

Identifiers

PMID41642106
PMCPMC13179663

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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.