ReviewFrontiers in oncology2021
Application of Real-World Data to External Control Groups in Oncology Clinical Trial Drug Development.
Review in Frontiers in oncology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
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.
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
22 citing papers in PubMed, 40 citations in OpenAlex.
- Relapse Rates With Paliperidone Palmitate in Adult Patients With Schizophrenia: Results for the 6-Month Formulation From an Open-label Extension Study Compared to Real-World Data for the 1-Month and 3-Month Formulations.The international journal of neuropsychopharmacology · 2024Trial
- Augmenting randomized clinical trial data with historical control data: Precision medicine applications.Journal of the National Cancer Institute · 2023Trial
- RWD-derived response in multiple myeloma.PloS one · 2023Trial
- Patient-centred and fit-for-purpose clinical trials in rare cancers: lessons from sarcomas for operationalising 2024 WHO Guidance and ICH E6(R3).EClinicalMedicine · 2026Review
- Advancing clinical trials for rare renal cell carcinoma subtypes: Consensus statements from the International Kidney Cancer Symposium North America 2025 think tank.Urologic oncology · 2026Article
- Trajectory timelines and treatment efficacy ofCanadian Urological Association journal = Journal de l'Association des urologues du Canada · 2026Article
- 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
- Keeping the End in Mind: Reviewing U.S. FDA Inspections of Submissions including Real-World Data.Therapeutic innovation & regulatory science · 2025Review
- Real-time data in cancer registries: Validation of an automated data extraction system.iScience · 2025Article
- Ag(e)nostic precision oncology therapy approvals across the years.Trends in cancer · 2025Review
- Comparative effectiveness among BRAF plus MEK inhibitors for patients with BRAF V600-mutant melanoma.ESMO real world data and digital oncology · 2024Article
- Examining external control arms in oncology: A scoping review of applications to date.Cancer medicine · 2024Article
- Comparison of Synthetic Data Generation Techniques for Control Group Survival Data in Oncology Clinical Trials: Simulation Study.JMIR medical informatics · 2024Article
- Real-world effectiveness of post-trastuzumab emtansine treatment for human epidermal growth factor receptor 2-positive metastatic breast cancer: a multicenter, matched cohort analysis from the Epidemiology Strategy and Medical Economics database (2008-2018).ESMO real world data and digital oncology · 2024Article
- The Next Horizon of Drug Development: External Control Arms and Innovative Tools to Enrich Clinical Trial Data.Therapeutic innovation & regulatory science · 2024Review
- TheEClinicalMedicine · 2024Article
- Utilization of anonymization techniques to create an external control arm for clinical trial data.BMC medical research methodology · 2023Observational
- ESMO Guidance for Reporting Oncology real-World evidence (GROW).ESMO real world data and digital oncology · 2023Article
- Overview of approaches to estimate real-world disease progression in lung cancer.JNCI cancer spectrum · 2023Review
- Enriching single-arm clinical trials with external controls: possibilities and pitfalls.Blood advances · 2023Article
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 at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Randomized controlled trials (RCTs) that assess overall survival are considered the "gold standard" when evaluating the efficacy and safety of a new oncology intervention. However, single-arm trials that use surrogate endpoints (e.g., objective response rate or duration of response) to evaluate clinical benefit have become the basis for accelerated or breakthrough regulatory approval of precision oncology drugs for cases where the target and research populations are relatively small. Interpretation of efficacy in single-arm trials can be challenging because such studies lack a standard-of-care comparator arm. Although an external control group can be based on data from other clinical trials, using an external control group based on data collected outside of a trial may not only offer an alternative to both RCTs and uncontrolled single-arm trials, but it may also help improve decision-making by study sponsors or regulatory authorities. Hence, leveraging real-world data (RWD) to construct external control arms in clinical trials that investigate the efficacy and safety of drug interventions in oncology has become a topic of interest. Herein, we review the benefits and challenges associated with the use of RWD to construct external control groups, and the relevance of RWD to early oncology drug development.
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Registered trials
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.