Evidence map›Paper›PMID 33734308›Full record

ArticleBrain : a journal of neurology2021

Towards realizing the vision of precision medicine: AI based prediction of clinical drug response.

Johann de Jong, Ioana Cutcutache, Matthew Page, Sami Elmoufti, Cynthia Dilley, Holger Fröhlich, Martin Armstrong

Open access · greenAbstract read
In one paragraph

Article in Brain : a journal of neurology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 47 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
47citing papers in PubMed, 1 pooled it
9.8field-weighted citation impact, top 2% of its field
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

47 citing papers in PubMed, 1 synthesis or guideline pooled it, 104 citations in OpenAlex.

  1. Pooled it
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  15. Applications of artificial intelligence in drug discovery for neurological diseases.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2025
    Review
  16. Article
  17. Article
  18. AI and Neurology.Neurological research and practice · 2025
    Review
  19. Article
  20. New epilepsy therapies in development.Nature reviews. Drug discovery · 2024
    Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

7 authors at 4 institutions in 3 countries.

Johann de JongData and Translational Sciences, UCB Biosciences GmbH, 40789 Monheim am Rhein, Germany.ORCID 0000-0002-2097-0915
Ioana CutcutacheData and Translational Sciences, UCB Pharma, Slough SL1 3WE, UK.
Matthew PageData and Translational Sciences, UCB Pharma, Slough SL1 3WE, UK.
Sami ElmouftiLate Development Statistics, UCB Biosciences Inc., Raleigh, NC 27617, USA.
Cynthia DilleyHead of Asset Strategy, UCB Inc., Smyrna, GA 30080, USA.
Holger FröhlichData and Translational Sciences, UCB Biosciences GmbH, 40789 Monheim am Rhein, Germany.
Martin ArmstrongData and Translational Sciences, UCB Pharma, 1420 Braine l'Alleud, Belgium.
UCB Pharma (United Kingdom) · GBUCB Pharma (Belgium) · BEUCB Pharma (Germany) · DEUniversity of Bonn · DE

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Accurate and individualized prediction of response to therapies is central to precision medicine. However, because of the generally complex and multifaceted nature of clinical drug response, realizing this vision is highly challenging, requiring integrating different data types from the same individual into one prediction model. We used the anti-epileptic drug brivaracetam as a case study and combine a hybrid data/knowledge-driven feature extraction with machine learning to systematically integrate clinical and genetic data from a clinical discovery dataset (n = 235 patients). We constructed a model that successfully predicts clinical drug response [area under the curve (AUC) = 0.76] and show that even with limited sample size, integrating high-dimensional genetics data with clinical data can inform drug response prediction. After further validation on data collected from an independently conducted clinical study (AUC = 0.75), we extensively explore our model to gain insights into the determinants of drug response, and identify various clinical and genetic characteristics predisposing to poor response. Finally, we assess the potential impact of our model on clinical trial design and demonstrate that, by enriching for probable responders, significant reductions in clinical study sizes may be achieved. To our knowledge, our model represents the first retrospectively validated machine learning model linking drug mechanism of action and the genetic, clinical and demographic background in epilepsy patients to clinical drug response. Hence, it provides a blueprint for how machine learning-based multimodal data integration can act as a driver in achieving the goals of precision medicine in fields such as neurology.

Indexed as

Computer SimulationMachine LearningAdultAgedAnticonvulsantsEpilepsyFemaleGenome-Wide Association StudyHumansMaleMiddle AgedPrecision MedicinePyrrolidinonesTreatment OutcomeAnticonvulsantsbrivaracetamPyrrolidinonesartificial intelligenceclinical study designepilepsypharmacogeneticsprecision medicine

Identifiers

PMID33734308
PMCPMC8320273
OpenAlexW3136539405

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

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.