ReviewPharmacoepidemiology and drug safety2020
Trial designs using real-world data: The changing landscape of the regulatory approval process.
Review in Pharmacoepidemiology and drug safety, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 121 papers, 6 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
121 citing papers in PubMed, 6 syntheses or guidelines pooled it.
- Effect of Multimodal App-Based Interventions on Glycemic Control in Patients With Type 2 Diabetes: Systematic Review and Meta-Analysis.Journal of medical Internet research · 2025Pooled it
- Advancing the role of real-world evidence in comparative effectiveness research.Journal of comparative effectiveness research · 2024Pooled it
- The Role of Real-World Evidence in FDA-Approved New Drug and Biologics License Applications.Clinical pharmacology and therapeutics · 2022Pooled it
- Systematic review of combinations of targeted or immunotherapy in advanced solid tumors.Journal for immunotherapy of cancer · 2021Pooled it
- How well can we assess the validity of non-randomised studies of medications? A systematic review of assessment tools.BMJ open · 2021Pooled it
- Assessing the uncertainty of treatment outcomes in a previous systematic review of venous leg ulcer randomized controlled trials: Additional secondary analysis.Wound repair and regeneration : official publication of the Wound Healing Society [and] the European Tissue Repair Society · 2021Pooled it
- Trial
- Trial
- 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
- Methodological innovations to advance substance use disorder research: Proceedings of a NIDA workshop on target trial emulation and translational testing of digital health tools.Journal of substance use and addiction treatment · 2026Article
- Cost, context, and confounding: rethinking real-world data generation and interpretation in advanced NSCLC.The oncologist · 2026Article
- Premarket and postmarket real-world evidence studies supporting U.S. Food and Drug Administration regulatory decision-making, 2016-2024.Clinical trials (London, England) · 2026Article
- Effectiveness and Tolerability of Vortioxetine in Major Depressive Disorder: A Real-World Study in Switzerland.Clinical drug investigation · 2026Observational
- Article
- A scoping review on using real-world data to evaluate the effectiveness of mHealth applications.NPJ digital medicine · 2026Article
- A Cross-Sectional Survey of Evidence Needs for Medicinal Products in Europe With a Focus on Real-World Evidence.Pharmacoepidemiology and drug safety · 2026Article
- Psilocybin in Neuropsychiatric Disorders: Seeking Valuable Evidence in History, Pure Science, Clinical Trials and Real-World Data (RWD).Brain sciences · 2026Review
- Adult SMA REACH: A UK clinical network and real-world data collection study for adults living with spinal muscular atrophy.Journal of neuromuscular diseases · 2026Article
- Evaluating the Feasibility of Using Historical Placebo Control in Osteoarthritis Trials.Epidemiologia (Basel, Switzerland) · 2026Article
- A review of use of external data and update on reporting standards in Sequential Multiple-Assignment Randomised Trials.Clinical trials (London, England) · 2026Review
61 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
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
purposeThere is a need to develop hybrid trial methodology combining the best parts of traditional randomized controlled trials (RCTs) and observational study designs to produce real-world evidence (RWE) that provides adequate scientific evidence for regulatory decision-making.
methodsThis review explores how hybrid study designs that include features of RCTs and studies with real-world data (RWD) can combine the advantages of both to generate RWE that is fit for regulatory purposes.
resultsSome hybrid designs include randomization and use pragmatic outcomes; other designs use single-arm trial data supplemented with external comparators derived from RWD or leverage novel data collection approaches to capture long-term outcomes in a real-world setting. Some of these approaches have already been successfully used in regulatory decisions, raising the possibility that studies using RWD could increasingly be used to augment or replace traditional RCTs for the demonstration of drug effectiveness in certain contexts. These changes come against a background of long reliance on RCTs for regulatory decision-making, which are labor-intensive, costly, and produce data that can have limited applicability in real-world clinical practice.
conclusionsWhile RWE from observational studies is well accepted for satisfying postapproval safety monitoring requirements, it has not commonly been used to demonstrate drug effectiveness for regulatory purposes. However, this position is changing as regulatory opinions, guidance frameworks, and RWD methodologies are evolving, with growing recognition of the value of using RWE that is acceptable for regulatory decision-making.
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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.