ReviewClinical epidemiology2020
Synthetic and External Controls in Clinical Trials - A Primer for Researchers.
Review in Clinical epidemiology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 125 papers, 4 of them syntheses that pooled 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.
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
125 citing papers in PubMed, 4 syntheses or guidelines pooled it.
- Model-Based Meta-Analysis of Objective Response Rate and Survival Endpoints to Compare PD-1 and PD-L1 Treatment Outcomes in Non-Small Cell Lung Cancer.CPT: pharmacometrics & systems pharmacology · 2026Pooled it
- Pooled it
- Use of external control arms in immune-mediated inflammatory diseases: a systematic review.BMJ open · 2023Pooled it
- Model-based meta-analysis of non-small cell lung cancer with standard of care PD-1 inhibitors and chemotherapy for early development decision making.CPT: pharmacometrics & systems pharmacology · 2023Pooled it
- Comparability of external and internal control patients for the prospective randomized HOVON-103 trial in older AML patients.British journal of haematology · 2026Trial
- Oral lamivudine in diabetic macular edema: A randomized, double-blind, placebo-controlled clinical trial.Med (New York, N.Y.) · 2025Trial
- Synthetic control methods for n-of-1 and parallel-group trials in Alzheimer's disease: A proof-of-concept study using the I-CONECT.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2025Trial
- Trial
- Embracing Digital as a Paradigm Shift in Medical Affairs.Pharmaceutical medicine · 2026Review
- A Frequentist Approach Using Weighted Estimating Equations for Survival Analysis of Randomized Controlled Trial With External Control Arm.Biometrical journal. Biometrische Zeitschrift · 2026Article
- Constructing an External Control Arm Using the French REALYSA Cohort to Replicate Outcomes of a Randomized Trial Arm in Advanced Hodgkin Lymphoma.Clinical pharmacology and therapeutics · 2026Article
- Leveraging Synthetic Clinical Data for Validation and Operational Readiness in Clinical Trials.Healthcare (Basel, Switzerland) · 2026Article
- Use of a Molecular Signature Response Classifier to Inform Treatment Selection Improves Clinical Disease Activity Among Patients with Rheumatoid Arthritis Initiating a Biologic or Targeted Synthetic Disease-Modifying Antirheumatic Drug.Arthritis care & research · 2026Article
- Mechanistic Artificial Intelligence for Personalized Drug Therapy: Integrating Pharmacokinetics, Pharmacodynamics, Therapeutic Drug Monitoring, and Multiomic Systems Biology.Pharmaceutics · 2026Review
- Combining external placebo-arm data in a prevention trial: effects of cocoa extract supplementation on cardiovascular disease.European journal of epidemiology · 2026Article
- External controls for rare disease drug development: Lessons for emerging and advanced therapeutic modalities.Molecular therapy. Advances · 2026Article
- The arrival of digital twins and in silico trials in drug development.Nature medicine · 2026Article
- Survival and Surrogate Biomarkers in Interventional Oncology Trials: Pitfalls, Challenges, and Future Directions.Cardiovascular and interventional radiology · 2026Review
- Clearing the bar: adding anti-VEGF therapy to chemoimmunotherapy for biliary tract cancer.Journal of gastrointestinal oncology · 2026Article
- A digital twin methodology using retrospective patient data for sample size reduction in Alzheimer's disease clinical trials.Alzheimer's research & therapy · 2026Article
65 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
There has been a rapid expansion in the use of non-randomized evidence in the regulatory approval of treatments globally. An emerging set of methodologies have been utilized to provide greater insight into external control data used for these purposes, collectively known as synthetic control methods. Through this paper, we provide the reader with a set of key questions to help assess the quality of literature publications utilizing synthetic control methodologies. Common challenges and real-life examples of synthetic controls are provided throughout, alongside a critical appraisal framework with which to assess future publications.
Indexed as
Identifiers
What OpenQuestion holds
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.