ArticleNature communications2022
Addressing challenges with real-world synthetic control arms to demonstrate the comparative effectiveness of Pralsetinib in non-small cell lung cancer.
Article in Nature communications, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03037385 (A Phase 1/2 Study of the Highly-selective RET Inhibitor, BLU-667, in Patients With Thyroid Cancer, Non-Small Cell Lung Cancer), which is not on this map. Cited by 41 papers, 1 of them a synthesis 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.
A Phase 1/2 Study of the Highly-selective RET Inhibitor, BLU-667, in Patients With Thyroid Cancer, Non-Small Cell Lung Cancer (NSCLC) and Other Advanced Solid Tumors
Who cites it
41 citing papers in PubMed, 1 synthesis or guideline pooled it, 73 citations in OpenAlex.
- Avelumab real-world use in advanced Merkel cell carcinoma: a systematic review and non-comparative meta-analysis.Future oncology (London, England) · 2026Pooled it
- 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
- R WE ready for reimbursement? A round-up of developments in real-world evidence relating to health technology assessment: part 26.Journal of comparative effectiveness research · 2026Review
- Causal inference and digital twins: a roadmap for the future of clinical trials.NPJ digital medicine · 2026Review
- Real-World Evidence: Better than Nothing or the Future? A Perspective on Clinical Evidence Generation in Interventional Oncology.Cardiovascular and interventional radiology · 2026Review
- AI-powered in silico twins: redefining precision medicine through simulation, personalization, and predictive healthcare.Saudi pharmaceutical journal : SPJ : the official publication of the Saudi Pharmaceutical Society · 2025Review
- Enhancing confidence in complex health technology assessments by using real-world evidence: highlighting existing strategies for effective drug evaluation.BMC medical research methodology · 2025Review
- Artificial Intelligence for Cardiovascular Care in Action: From Learning to Implementation in Health Systems.JACC. Advances · 2025Review
- Cancer treatment paradigms in the precision medicine era.Nature medicine · 2025Article
- Exploring synthetic controls in rare diseases with a proof of concept in spinal cord injury.BMC medicine · 2025Article
- Privacy-by-Design Approach to Generate Two Virtual Clinical Trials for Multiple Sclerosis and Release Them as Open Datasets: Evaluation Study.Journal of medical Internet research · 2025Article
- Precision cancer medicine 2025: some concerns.Acta oncologica (Stockholm, Sweden) · 2025Article
- Accounting for Misclassification of Binary Outcomes in External Control Arm Studies for Unanchored Indirect Comparisons: Simulations and Applied Example.Statistics in medicine · 2025Article
- Design, structure, and application of conductive polymer hybrid materials: a comprehensive review of classification, fabrication, and multifunctionality.RSC advances · 2025Review
- The complexity of finding fit-for-purpose real-world data for oncology patients with rareESMO real world data and digital oncology · 2025Article
- Synthetic Data in Healthcare and Drug Development: Definitions, Regulatory Frameworks, Issues.CPT: pharmacometrics & systems pharmacology · 2025Review
- Predicting tumor dynamics in treated patients from patient-derived-xenograft mouse models: a translational model-based approach.Journal of pharmacokinetics and pharmacodynamics · 2025Article
- Challenges and applications in generative AI for clinical tabular data in physiology.Pflugers Archiv : European journal of physiology · 2025Review
- Quantitative Bias Analysis for Single-Arm Trials With External Control Arms.JAMA network open · 2025Article
- Addressing challenges with Matching-Adjusted Indirect Comparisons to demonstrate the comparative effectiveness of entrectinib in metastatic ROS-1 positive Non-Small Cell Lung Cancer.BMC medical research methodology · 2025Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors at 7 institutions in 4 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
As advanced non-small cell lung cancer (aNSCLC) is being increasingly divided into rare oncogene-driven subsets, conducting randomised trials becomes challenging. Using real-world data (RWD) to construct control arms for single-arm trials provides an option for comparative data. However, non-randomised treatment comparisons have the potential to be biased and cause concern for decision-makers. Using the example of pralsetinib from a RET fusion-positive aNSCLC single-arm trial (NCT03037385), we demonstrate a relative survival benefit when compared to pembrolizumab monotherapy and pembrolizumab with chemotherapy RWD cohorts. Quantitative bias analyses show that results for the RWD-trial comparisons are robust to data missingness, potential poorer outcomes in RWD and residual confounding. Overall, the study provides evidence in favour of pralsetinib as a first-line treatment for RET fusion-positive aNSCLC. The quantification of potential bias performed in this study can be used as a template for future studies of this nature.
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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 Socratic graph.