Evidence mapPaperPMID 39792215Full record

ReviewTherapeutic innovation & regulatory science2025

A Consistent Lack of Consistency: Definitions, Evidentiary Expectations and Potential Use of Meaningful Change Data in Clinical Outcome Assessments Across Stakeholders. Results from a DIA Working Group Literature Review and Survey.

M Reaney, V Shih, A Wilson, B Byrom, N Medic, D Oberdhan, C Mamolo, M Majumder

Abstract readReview
In one paragraph

Review in Therapeutic innovation & regulatory science, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Review
  2. Article
  3. 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

8 authors.

M ReaneyIQVIA Patient-Centered Solutions, 3 Forbury Place, 23 Forbury Road, Reading, RG1 3JH, London, UK. Matthew.reaney@iqvia.com.
V ShihAstraZeneca, Gaithersburg, USA.
A WilsonPAREXEL International, Waltham, NC, USA.
B ByromeCOA Science, Signant Health, Nottingham, UK.
N MedicAstraZeneca, Cambridge, UK.
D OberdhanOtsuka Pharmaceutical Development & Commercialization, Inc., Rockville, USA.
C MamoloPatient-Centered Outcomes Research, Genentech Inc., A Member of the Roche Group, South San Francisco, CA, USA.
M MajumderBayer US LLC, Whippany, NJ, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundClinical outcome assessments (COAs) measure how patients feel or function and can be used to understand which patients experience benefits of treatment and which do not. Interpretation of COA data is influenced by how meaningful change is defined. We aimed to compare how different stakeholders define, assess, and use meaningful change for decisions that impact patients.

methodsA targeted literature review was undertaken in July 2021 using Medline, Embase, online grey literature search engines, and stakeholder organization websites. Additionally, a stakeholder survey on meaningful change was fielded between March and June 2023. Both quantitative and qualitative methods were used to analyze responses and identify key themes.

resultsThe literature review resulted in 86 references. These revealed different approaches to define, measure and validate meaningful change. There were 248 survey responses. Many respondents felt the terminology and methods for defining meaningful change are confusing. Respondents also emphasized the importance of distinguishing within-patient and between-group change, and defining meaningfulness from the patient perspective (most patients and caregivers do not share a similar definition of meaningfulness as their healthcare professionals).

conclusionFour key recommendations for defining, establishing, and interpreting meaningful change estimates for COAs are: (1) Be clear on the type of "meaningful change" that is discussed or needed for a COA, (2) Ensure the "patient voice" is informing meaningful change estimates/definitions, (3) Acknowledge that a meaningful change estimate for a COA may differ between populations, diseases, and disease states, and (4) Disseminate data in a way that reduces ambiguity.

Indexed as

Outcome Assessment, Health CareHumansStakeholder ParticipationSurveys and QuestionnairesBetween-group differenceClinical outcome assessment (COA)DIA working groupLiterature reviewMeaningful changePatient engagementStakeholder surveyWithin-patient change

Identifiers

PMID39792215
PMCPMC11880173

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.