Evidence map›Paper›PMID 37355603›Full record

ReviewBMC medical research methodology2023

Analysis of the characteristics and the degree of pragmatism exhibited by pragmatic-labelled trials of antineoplastic treatments.

Robbe Saesen, Kevin Depreytere, Karyna Krupianskaya, Joël Langeweg, Julie Verheecke, Denis Lacombe, Isabelle Huys

Abstract readReview
In one paragraph

Review in BMC medical research methodology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing 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

4 citing papers in PubMed.

  1. A methodological review of pragmatic designs in acute stroke trials.International journal of stroke : official journal of the International Stroke Society · 2026
    Review
  2. Review
  3. Article
  4. Article
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

7 authors.

Robbe SaesenClinical Pharmacology and Pharmacotherapy Research Unit, Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium. robbe.saesen@eortc.org.
Kevin DepreytereClinical Pharmacology and Pharmacotherapy Research Unit, Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium.
Karyna KrupianskayaClinical Pharmacology and Pharmacotherapy Research Unit, Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium.
Joël LangewegClinical Pharmacology and Pharmacotherapy Research Unit, Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium.
Julie VerheeckeClinical Pharmacology and Pharmacotherapy Research Unit, Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium.
Denis Lacombe *European Organisation for Research and Treatment of Cancer (EORTC), Avenue E. Mounier 83, 1200, Brussels, Belgium.
Isabelle Huys *Clinical Pharmacology and Pharmacotherapy Research Unit, Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundPragmatic clinical trials (PCTs) are designed to reflect how an investigational treatment would be applied in clinical practice. As such, unlike their explanatory counterparts, they measure therapeutic effectiveness and are capable of generating high-quality real-world evidence. However, the conduct of PCTs remains extremely rare. The scarcity of such studies has contributed to the emergence of the efficacy-effectiveness gap and has led to calls for launching more of them, including in the field of oncology. This analysis aimed to identify self-labelled pragmatic trials of antineoplastic interventions and to evaluate whether their use of this label was justified.

methodsWe searched PubMed® and Embase® for publications corresponding with studies that investigated antitumor therapies and that were tagged as pragmatic in their titles, abstracts and/or index terms. Subsequently, we consulted all available source documents for the included trials and extracted relevant information from them. The data collected were then used to appraise the degree of pragmatism displayed by the PCTs with the help of the validated PRECIS-2 tool.

resultsThe literature search returned 803 unique records, of which 46 were retained upon conclusion of the screening process. This ultimately resulted in the identification of 42 distinct trials that carried the 'pragmatic' label. These studies examined eight different categories of neoplasms and were mostly randomized, open-label, multicentric, single-country trials sponsored by non-commercial parties. On a scale of one (very explanatory) to five (very pragmatic), the median PCT had a PRECIS-2 score per domain of 3.13 (interquartile range: 2.57-3.53). The most and least pragmatic studies in the sample had a score of 4.44 and 1.57, respectively. Only a minority of trials were described in sufficient detail to allow them to be graded across all domains of the PRECIS-2 instrument. Many of the studies examined also had features that arguably precluded them from being pragmatic altogether, such as being monocentric or placebo-controlled in nature.

conclusionPCTs of antineoplastic treatments are generally no more pragmatic than they are explanatory.

Indexed as

Antineoplastic AgentsResearch DesignHumansMedical OncologyPragmatic Clinical Trials as TopicAntineoplastic AgentsCancerEfficacy-effectiveness gapExplanatory trialsNeoplasmsOncologyPragmatic trialsPRECIS-2Randomized controlled trialsReal-world evidenceTreatments

Identifiers

PMID37355603
PMCPMC10290324

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Registered trials

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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.