ArticleBlood advances2023
Enriching single-arm clinical trials with external controls: possibilities and pitfalls.
Article in Blood advances, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers, 1 of them a synthesis that pooled it.
What it found
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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.
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.
Who cites it
19 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Acceptance of external control arms by HTA agencies: a review of oncology submissions in France, England, Germany and Norway from 2021 to 2023.British journal of cancer · 2025Pooled it
- Feasibility of a prospective real-time monitoring system capturing real-world data from elderly patients with acute myeloid leukemia.ESMO real world data and digital oncology · 2026Article
- A five-phase evaluation framework for diagnostic and predictive medical artificial intelligence.NPJ digital medicine · 2026Article
- Outcomes of Patients with RET Fusion-Positive Non-Small Cell Lung Cancer Treated with First-Line Non-Molecularly Targeted Therapies: A Real-World Benchmark in Europe from the RECALIB-RET Study.Advances in therapy · 2026Article
- Article
- Real-World Data as External Control in Assessing the Efficacy of Allogeneic Adipose-Derived Stem Cells Therapy for Advanced Chronic Kidney Disease.Clinical and translational science · 2025Article
- A comparative study of hematologic response to ropeginterferon alfa-2b in hydroxyurea-resistant or intolerant polycythemia vera: a real-world data-based analysis.Blood research · 2025Article
- Synthetic control arm from mixed clinical trials and real-world data from the LYSA group for untreated diffuse large B-cell lymphoma patients aged over 80 years: a bona fide strategy for innovative clinical trials.Blood cancer journal · 2025Article
- Real-World Data and Causal Machine Learning to Enhance Drug Development.Therapeutic innovation & regulatory science · 2025Review
- Estimating comparative effectiveness using Single-Arm trials: A challenge in the field of agnosticism.BMC medical research methodology · 2025Article
- FedECA: federated external control arms for causal inference with time-to-event data in distributed settings.Nature communications · 2025Article
- Re-evaluating a single-arm trial of favipiravir for severe fever with thrombocytopenia syndrome in Japan: A proposal to use real-world data as external controls.PLoS neglected tropical diseases · 2025Article
- A Knowledge-Enhanced Platform (MetaSepsisKnowHub) for Retrieval Augmented Generation-Based Sepsis Heterogeneity and Personalized Management: Development Study.Journal of medical Internet research · 2025Article
- Matching-Assisted Power Prior for Incorporating Real-World Data in Randomized Clinical Trial Analysis.Statistics in medicine · 2025Article
- Matching plus regression adjustment for the estimation of the average treatment effect on survival outcomes: a case study with mosunetuzumab in relapsed/refractory follicular lymphoma.BMC medical research methodology · 2025Article
- Informative censoring in externally controlled clinical trials: a potential source of bias.ESMO open · 2025Article
- Expanding the reach of a fall prevention intervention for older adult emergency department patients through telehealth: a trial protocol.Frontiers in public health · 2025Article
- The PERMIT guidelines for designing and implementing all stages of personalised medicine research.Scientific reports · 2024Article
- Important tool in our rare disease toolbox: hybrid retrospective-prospective natural history studies serve well as external comparators for rare disease studies.Frontiers in drug safety and regulation · 2024Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
For the past decade, it has become commonplace to provide rapid answers and early patient access to innovative treatments in the absence of randomized clinical trials (RCT), with benefits estimated from single-arm trials. This trend is important in oncology, notably when assessing new targeted therapies. Some of those uncontrolled trials further include an external/synthetic control group as an innovative way to provide an indirect comparison with a pertinent control group. We aimed to provide some guidelines as a comprehensive tool for (1) the critical appraisal of those comparisons or (2) for performing a single-arm trial. We used the example of ciltacabtagene autoleucel for the treatment of adult patients with relapsed or refractory multiple myeloma after 3 or more treatment lines as an illustrative example. We propose a 3-step guidance. The first step includes the definition of an estimand, which encompasses the treatment effect and the targeted population (whole population or restricted to single-arm trial or external controls), reflecting a clinical question. The second step relies on the adequate selection of external controls from previous RCTs or real-world data from patient cohorts, registries, or electronic patient files. The third step consists of choosing the statistical approach targeting the treatment effect defined above and depends on the available data (individual-level data or aggregated external data). The validity of the treatment effect derived from indirect comparisons heavily depends on careful methodological considerations included in the proposed 3-step procedure. Because the level of evidence of a well-conducted RCT cannot be guaranteed, the evaluation is more important than in standard settings.
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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.