Evidence map›Paper›PMID 39386161›Full record

ReviewEClinicalMedicine2024

Advancing patient-centric care: integrating patient reported outcomes for tolerability assessment in early phase clinical trials - insights from an expert virtual roundtable.

Christina Yap, Olalekan Lee Aiyegbusi, Emily Alger, Ethan Basch, Jill Bell, Vishal Bhatnagar, David Cella, Philip Collis, Amylou C Dueck, Alexandra Gilbert and 22 more

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in EClinicalMedicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
17citing 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

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

  1. Pooled it
  2. Article
  3. Review
  4. Review
  5. Mapping the EORTC item library to the World Health Organization international classification of functioning, disability and health: a content analysis.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2026
    Article
  6. Article
  7. Article
  8. Review
  9. The association between GLP-1 receptor agonists and fractures: a nationwide Danish nested case-control study.Osteoporosis international : a journal established as result of cooperation between the European Foundation for Osteoporosis and the National Osteoporosis Foundation of the USA · 2026
    Article
  10. Article
  11. Article
  12. Article
  13. Article
  14. Review
  15. Article
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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

32 authors.

Christina YapClinical Trials and Statistics Unit, The Institute of Cancer Research, London, UK.
Olalekan Lee AiyegbusiCentre for Patient-Reported Outcomes Research (CPROR), Institute of Applied Health Research, University of Birmingham, Birmingham, UK.
Emily AlgerClinical Trials and Statistics Unit, The Institute of Cancer Research, London, UK.
Ethan BaschLineberger Comprehensive Cancer Center, Chapel Hill, NC, 27514, USA.
Jill BellAstraZeneca, Oncology Research and Development, Gaithersburg, MD, USA.
Vishal BhatnagarOncology Center of Excellence, US Food and Drug Administration, Silver Spring, MD, USA.
David CellaDepartment of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.
Philip CollisCentre for Patient-Reported Outcomes Research (CPROR), Institute of Applied Health Research, University of Birmingham, Birmingham, UK.
Amylou C DueckDepartment of Quantitative Health Sciences, Mayo Clinic, Scottsdale, AZ, USA.
Alexandra GilbertLeeds Institute for Medical Research, University of Leeds, St James's University Hospital, Leeds, UK.
Ari GnanasakthyRTI Health Solutions, Patient Centered Outcomes Research, Research Triangle Park, NC, USA.
Alastair GreystokeNU Cancer, Newcastle University, Newcastle upon Tyne, UK.
Aaron R HansenPrincess Alexandra Hospital, Cancer Services, Brisbane, Queensland, Australia.
Paul KamudoniMerck KGaA, Darmstadt, Germany.
Olga KholmanskikhFederal Agency for Medicines and Health Products (FAMHP), Brussels, Belgium.
Bellinda L King-KallimanisLUNGevity Foundation, Bethesda, MD, USA.
Harlan KrumholzCenter for Outcomes Research and Evaluation, Yale New Haven Hospital, New Haven, CT, USA.
Anna MinchomDrug Development Unit, Royal Marsden/Institute of Cancer Research, Sutton, UK.
Daniel O'ConnorThe Association of the British Pharmaceutical Industry, UK.
Joan PetrieCanadian Cancer Trials Group (CCTG), Kingston, Ontario, Canada.
Claire PiccininQuality of Life Department, European Organisation for Research and Treatment of Cancer, Brussels, Belgium.
Khadija Rerhou RantellMedicines and Healthcare Products Regulatory Agency (MHRA), UK.
Saaeha RauzAcademic Unit of Ophthalmology, Institute of Inflammation and Ageing, University of Birmingham, UK.
Ameeta RetzerCentre for Patient Reported Outcomes Research (CPROR), Institute of Applied Health Research, University of Birmingham, Birmingham, UK.
Steven RizkVeloxis Pharmaceuticals, Cary, NC, USA.
Lynne WagnerUniversity of North Carolina Gillings School of Global Public Health, Chapel Hill, NC, USA.
Maxime SassevilleHealth Canada, Ottawa, Ontario, Canada.
Lesley K SeymourCanadian Cancer Trials Group (CCTG), Kingston, Ontario, Canada.
Harald A WeberPfizer Oncology, Global Medical Affairs/Early-Stage Development, Zug, Switzerland.
Roger WilsonCancer Research Advocates Forum UK, Sarcoma Patient Advocacy Global Network, UK.
Melanie CalvertCentre for Patient-Reported Outcomes Research (CPROR), Institute of Applied Health Research, University of Birmingham, Birmingham, UK.
John Devin PeipertDepartment of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.

Funding

Women's Cancer ProgramP30CA015083 · NCI · MAYO CLINIC ROCHESTER · PI Lila J. Rutten · 1985 to 2026
$151.3M
NCI NIH HHS P30 CA015083
6 · The paper itself

Abstract

Early phase clinical trials provide an initial evaluation of therapies' risks and benefits to patients, including safety and tolerability, which typically relies on reporting outcomes by investigator and laboratory assessments. Use of patient-reported outcomes (PROs) to inform risks (tolerability) and benefits (improvement in disease symptoms) is more common in later than early phase trials. We convened a two-day expert roundtable covering: (1) the necessity and feasibility of a universal PRO core conceptual model for early phase trials; (2) the practical integration of PROs in early phase trials to inform tolerability assessment, guide dose decisions, or as real-time safety alerts to enhance investigator-reported adverse events. Participants (n = 22) included: patient advocates, regulators, clinicians, statisticians, pharmaceutical representatives, and PRO methodologists working across diverse clinical areas. In this manuscript, we report major recommendations resulting from the roundtable discussions corresponding to each theme. Additionally, we highlight priority areas necessitating further investigation.

Indexed as

Dose-findingEarly phase trialsPatient-centred clinical developmentPatient-reported outcomesPhase IPhase IIQuality of lifeTolerabilityTrial designs

Identifiers

PMID39386161
PMCPMC11462221

What OpenQuestion 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 OpenQuestion graph.