Evidence map›Paper›PMID 40480201›Full record

ArticleAmerican journal of human genetics2025

Consultation informs strategies for improving the use of functional evidence in variant classification.

Rehan M Villani, Bronwyn Terrill, Emma Tudini, Maddison E McKenzie, Corrina C Cliffe, Christopher N Hahn, Ben Lundie, Tessa Mattiske, Ebony Matotek, Abbye E McEwen and 7 more

Abstract read
In one paragraph

Article in American journal of human genetics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing 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

9 citing papers in PubMed.

  1. Article
  2. Review
  3. A scalable approach to resolving variants of uncertain significance.bioRxiv : the preprint server for biology · 2026
    Article
  4. Review
  5. Article
  6. Article
  7. MaveMD: A functional data resource for genomic medicine.medRxiv : the preprint server for health sciences · 2025
    Article
  8. Article
  9. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

17 authors.

Rehan M VillaniQIMR Berghofer, Brisbane, QLD, Australia.
Bronwyn TerrillAustralian Genomics, Murdoch Children's Research Institute, Melbourne, VIC, Australia; Clinical Translation and Engagement Platform, Garvan Institute of Medical Research, Sydney, NSW, Australia; Faculty of Medicine and Health, UNSW Sydney, Sydney, NSW, Australia.
Emma TudiniQIMR Berghofer, Brisbane, QLD, Australia; Australian Genomics, Murdoch Children's Research Institute, Melbourne, VIC, Australia.
Maddison E McKenzieQIMR Berghofer, Brisbane, QLD, Australia.
Corrina C CliffeDouglass Hanly Moir Pathology, Macquarie Park, NSW, Australia.
Christopher N HahnDepartment of Genetics and Molecular Pathology, SA Pathology, Adelaide, SA, Australia.
Ben LundiePathology Queensland, Queensland Health, Brisbane, QLD, Australia; The University of Queensland, Brisbane, QLD, Australia.
Tessa MattiskeAustralian Genomics, Murdoch Children's Research Institute, Melbourne, VIC, Australia; Australian Functional Genomics Network, Murdoch Children's Research Institute, Parkville, VIC, Australia.
Ebony MatotekAustralian Functional Genomics Network, Murdoch Children's Research Institute, Parkville, VIC, Australia.
Abbye E McEwenDepartment of Laboratory Medicine and Pathology, University of Washington, Seattle, WA, USA; Department of Genome Sciences, University of Washington, Seattle, WA, USA; Brotman Baty Institute, Seattle, WA, USA.
Sarah L NickersonDepartment of Diagnostic Genomics, PathWest, QEII Medical Centre, Perth, WA, Australia; The University of Western Australia, Perth, WA, Australia.
James BreenBlack Ochre Data Labs (Indigenous Genomics), The Kids Research Institute Australia & Australian National University, Adelaide, SA, Australia; National Centre for Indigenous Genomics, Australian National University, Canberra, ACT, Australia.
Douglas M FowlerDepartment of Genome Sciences, University of Washington, Seattle, WA, USA; Brotman Baty Institute, Seattle, WA, USA; Department of Bioengineering, University of Washington, Seattle, WA, USA.
John ChristodoulouGenomic Medicine Theme, Murdoch Children's Research Institute, Melbourne, VIC, Australia; Department of Paediatrics, University of Melbourne, Melbourne, VIC, Australia.
Lea StaritaDepartment of Genome Sciences, University of Washington, Seattle, WA, USA; Brotman Baty Institute, Seattle, WA, USA.
Alan F RubinThe Walter and Eliza Hall Institute of Medical Research, University of Melbourne, Melbourne, VIC, Australia; Department of Medical Biology, University of Melbourne, Melbourne, VIC, Australia. Electronic address: alan.rubin@wehi.edu.au.
Amanda B SpurdleQIMR Berghofer, Brisbane, QLD, Australia; The University of Queensland, Brisbane, QLD, Australia. Electronic address: amanda.spurdle@qimrb.edu.au.

Funding

Advancing the implementation of variant-level functional data into clinical databases and clinical practiceR01HG013025 · NHGRI · UNIVERSITY OF WASHINGTON · PI Lea Starita, Andrew Ben Stergachis · 2023 to 2026
$3.1M
NHGRI NIH HHS R01 HG013025
6 · The paper itself

Abstract

When investigating whether a variant identified by diagnostic genetic testing is causal for disease, applied genetics professionals evaluate all available evidence to assign a clinical classification. Functional assays of higher and higher throughput are increasingly being generated and, when appropriate, can provide strong functional evidence for or against pathogenicity in variant classification. Despite functional assay data representing unprecedented value for genomic diagnostics, challenges remain around the application of functional evidence in variant curation. To investigate a growing gap articulated in recent international studies, we surveyed genetic diagnostic professionals in Australasia to assess their application of functional evidence in clinical practice. The survey results echo the universal difficulty in evaluating functional evidence but expand on this by indicating that even self-proclaimed expert respondents are not confident to apply functional evidence, mainly due to uncertainty around practice recommendations. Respondents also identified the need for support resources and educational opportunities, and in particular requested expert recommendations and updated practice guidelines to improve translation of experimental data to curation evidence. We then collated a list of 226 functional assays and the evidence strength recommended by 19 ClinGen Variant Curation Expert Panels. Specific assays for more than 45,000 variants were evaluated, but evidence recommendations were generally limited to lower throughput and strength. As an initial step, we provide our collated list of assay evidence as a source of international expert opinion on the evaluation of functional- evidence and conclude that these results highlight an opportunity to develop additional support resources to fully utilize functional evidence in clinical practice.

Indexed as

Genetic TestingGenetic VariationReferral and ConsultationHumansSurveys and QuestionnairesACMG/AMP guidelinesassayclinical genomicsdiagnostic geneticseducationfunctional evidencepathogenicity assessmentvariant classification

Identifiers

PMID40480201
PMCPMC12256917

What OpenQuestion holds

Textmetadata
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.