ReviewDrug safety2020
A Framework for Methodological Choice and Evidence Assessment for Studies Using External Comparators from Real-World Data.
Review in Drug safety, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 38 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
38 citing papers in PubMed.
- Comparing g-computation, propensity score-based weighting, and targeted maximum likelihood estimation for analyzing externally controlled trials with both measured and unmeasured confounders: a simulation study.BMC medical research methodology · 2023Trial
- Augmenting randomized clinical trial data with historical control data: Precision medicine applications.Journal of the National Cancer Institute · 2023Trial
- Considerations for the Integration of Clinical Trials and Real-World Data.Clinical pharmacology and therapeutics · 2026Review
- Enabling evidence generation for extremely preterm neonates: the use of neonatal real-world data.Pediatric research · 2026Review
- Model-Informed Decision Making from Drug Discovery to Reimbursement: A Roadmap for Clinical Pharmacology in Value-Based Healthcare.Journal of clinical pharmacology · 2026Review
- Review
- Association between exon-skipping therapy with eteplirsen and cardiac outcomes in Duchenne muscular dystrophy.Journal of neuromuscular diseases · 2026Article
- Article
- Dental Research in the Digital Age: The Registry-Based Clinical Trial.Clinical oral implants research · 2026Article
- Review
- Acceptability of external control-arm use in nononcology health technology assessment submissions.Journal of comparative effectiveness research · 2026Article
- Augmented two-stage estimation for treatment switching in oncology trials: Leveraging external data for improved precision.Statistical methods in medical research · 2025Article
- Article
- Challenges and Criteria for Single-Arm Trials Leading to an Added Benefit in German Health Technology Assessments.PharmacoEconomics · 2025Article
- Design, Conduct, and Analysis of Externally Controlled Trials.JAMA network open · 2025Article
- An External Control Arm for the Oral Factor XIa Inhibitor Asundexian Phase 2 Trial in Atrial Fibrillation (PACIFIC-AF) Using Electronic Health Records.Cardiology and therapy · 2025Article
- Adopting life-cycle HTA: a tumor-agnostic precision oncology index economic evaluation from publicly available reimbursement reviews.International journal of technology assessment in health care · 2025Article
- Keep asking: What do I want? What do I have? What do I do? : An approach for combining information to learn about a target population.European journal of epidemiology · 2025Article
- AUGMENTed Real-World Data Enhances Comparative Efficacy Between Once-Weekly Insulin Icodec with Dosing Guide App Versus Once-Daily Insulin Glargine U300 in Insulin-Naive Type 2 Diabetes.Diabetes therapy : research, treatment and education of diabetes and related disorders · 2025Article
- Adaptive Universal Principles for Real-world Observational Studies (AUPROS): an approach to designing real-world observational studies for clinical, epidemiologic, and precision oncology research.British journal of cancer · 2025Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Several approaches have been proposed recently to accelerate the pathway from drug discovery to patient access. These include novel designs such as using controls external to the clinical trial where standard randomised controls are not feasible. In parallel, there has been rapid growth in the application of routinely collected healthcare 'real-world' data for post-market safety and effectiveness studies. Thus, using real-world data to establish an external comparator arm in clinical trials is a natural next step. Regulatory authorities have begun to endorse the use of external comparators in certain circumstances, with some positive outcomes for new drug approvals. Given the potential to introduce bias associated with observational studies, there is a need for recommendations on how external comparators should be best used. In this article, we propose an evaluation framework for real-world data external comparator studies that enables full assessment of available evidence and related bias. We define the principle of exchangeability and discuss the applicability of criteria described by Pocock for consideration of the exchangeability of the external and trial populations. We explore how trial designs using real-world data external comparators fit within the evidence hierarchy and propose a four-step process for good conduct of external comparator studies. This process is intended to maximise the quality of evidence based on careful study design and the combination of covariate balancing, bias analysis and combining outcomes.
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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.