Evidence map›Paper›PMID 41120875›Full record

ArticleBMC medical research methodology2025

Estimating comparative effectiveness using Single-Arm trials: A challenge in the field of agnosticism.

Shang Cao, Hongwei Kan, Mingliang Lu, Jianhong Pan

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in BMC medical research methodology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed, 2 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

2 citing papers in PubMed, 2 syntheses or guidelines pooled it.

  1. Pooled it
  2. Pooled it
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

4 authors.

Shang CaoDepartment of Medical Review, Yangtze River Delta Center for Drug Evaluation and Inspection of National Medical Products Administration, Shanghai, China.
Hongwei KanDepartment of Medical Review, Yangtze River Delta Center for Drug Evaluation and Inspection of National Medical Products Administration, Shanghai, China.
Mingliang LuOffice of Biostatistics and Clinical Pharmacology, Center for Drug Evaluation, National Medical Products Administration, Beijing, China.
Jianhong PanOffice of Biostatistics and Clinical Pharmacology, Center for Drug Evaluation, National Medical Products Administration, Beijing, China. panjh@cde.org.cn.

Funding

Regulatory Research on Innovation and High-quality Development of Pharmaceuticals in the Yangtze River Delta of China 24692122700
6 · The paper itself

Abstract

Although single-arm trials (SATs) can serve as a pivotal study design for evaluating drug efficacy in specific scenarios, they are accompanied by inherent limitations and challenges. This paper explores the challenges of validity, reliability, statistical power, and the use of external control in SATs from a statistical perspective. We reflect on the rationale for employing SATs to support efficacy assessments, and provide recommendations based on their limitations and potential biases. The objective is to offer guidance for the improved design and implementation of SATs in future research.

Indexed as

Clinical Trials as TopicComparative Effectiveness ResearchResearch DesignBiasData Interpretation, StatisticalHumansReproducibility of ResultsBiasEfficacy evaluationSingle-arm trialStatistical principle

Identifiers

PMID41120875
PMCPMC12538941

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.