Evidence map›Paper›PMID 39825875›Full record

ReviewClinical pharmacology and therapeutics2025

Methodological Insights on Biomarker-Based Patient Selection: A Review of Scientific Advice Procedures at the European Medicines Agency.

Cynthia Huber, Joerg Zinserling, Norbert Benda, Thorsten Vetter, Marcia Rueckbeil

Abstract readReview
In one paragraph

Review in Clinical pharmacology and therapeutics, 2025. 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. Review
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

5 authors.

Cynthia HuberDepartment of Medical Statistics, University Medical Center, Göttingen, Germany.ORCID 0000-0003-2035-3682
Joerg ZinserlingFederal Institute for Drugs and Medical Devices (BfArM), Bonn, Germany.
Norbert BendaDepartment of Medical Statistics, University Medical Center, Göttingen, Germany.
Thorsten VetterScientific Advice Office, European Medicines Agency, Amsterdam, The Netherlands.
Marcia RueckbeilData Analytics and Methods Task Force, European Medicines Agency, Amsterdam, The Netherlands.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Biomarkers play a pivotal role in the selection and enrollment of trial participants. Particularly, predictive biomarkers help tailor medical care to individual patients; however, also prognostic biomarkers require consideration at the design stage. At the time of initiating a clinical trial, there may be uncertainty about whether a biomarker is predictive or prognostic, and the trial design may need to account for this. Relevant discussions between drug developers and regulators on the role of a biomarker in a specific drug development program are expected to take place during Scientific Advice (SA) procedures. SA procedures at the European Medicines Agency from January 1, 2018, to December 31, 2020, were systematically searched for methodological discussions around the use of predictive or prognostic biomarkers. The final analysis included 45 SA procedures for which key characteristics were summarized quantitatively. Selected methodological issues such as the cutoff selection of continuous biomarkers or study design considerations were elaborated in a qualitative summary. Our results identify commonly encountered points for discussion between drug developers and the European Medicines Agency for biomarker-informed patient selection and enrollment. Identified topics addressed during SA procedures include cutoff selection, study design, multiplicity control, and data-driven subgroup selection. The majority of the identified 45 SA procedures concern development programs in oncology. Addressing these issues upfront will allow for an improved dialogue between drug developers and regulators and support the drug development program and ultimately patient-centered access to medicines.

Indexed as

BiomarkersClinical Trials as TopicDrug DevelopmentPatient SelectionEuropeHumansResearch DesignBiomarkers

Identifiers

PMID39825875
PMCPMC11993284

What Socratic holds

Textmetadata
LicenceCC BY
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 Socratic graph.