ArticleClinical pharmacology and therapeutics2022
Use of Real-World Data and Evidence in Drug Development of Medicinal Products Centrally Authorized in Europe in 2018-2019.
Article in Clinical pharmacology and therapeutics, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers, 2 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
26 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Treatment Effects in Randomized and Nonrandomized Studies of Pharmacological Interventions: A Meta-Analysis.JAMA network open · 2024Pooled it
- Licensing of Orphan Medicinal Products-Use of Real-World Data and Other External Data on Efficacy Aspects in Marketing Authorization Applications Concluded at the European Medicines Agency Between 2019 and 2021.Frontiers in pharmacology · 2022Pooled it
- Contribution of Real-World Evidence for the Orphan Medicines Approvals in the European Union Between 2000 and 2025.Therapeutic innovation & regulatory science · 2026Article
- Contribution of Real-World Evidence for the Orphan Medicines Approvals in the European Union Between 2000 and 2025.Therapeutic innovation & regulatory science · 2026Article
- Core Concepts in Pharmacoepidemiology-Round 3: Call for Writing Groups.Pharmacoepidemiology and drug safety · 2026Article
- Maximizing the impact of real-world evidence on cancer treatment: what do we need to achieve it?Journal of comparative effectiveness research · 2026Review
- Real-World Data Included in Post-authorisation Measures: A Case Study of Approved Advanced Therapy Medicinal Products in the European Union between 2013 and 2024.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2025Article
- FRAME: Framework for Real-World Evidence Assessment to Mitigate Evidence Uncertainties for Efficacy/Effectiveness - An Evaluation of Regulatory and Health Technology Assessment Decision Making.Clinical pharmacology and therapeutics · 2025Article
- Beyond the Pill: Mapping Process-Oriented Decision Support Models in Pharmaceutical Policy.Healthcare (Basel, Switzerland) · 2025Review
- Predicting resistance to chemotherapy using chromosomal instability signatures.Nature genetics · 2025Article
- The EU project Real4Reg: unlocking real-world data with AI.Health research policy and systems · 2025Article
- Characteristics and impact of real-world evidence studies in oncology: comprehensive mapping review of publications evaluating targeted therapies in solid tumours.ESMO real world data and digital oncology · 2024Review
- Framework for Multistakeholder Patient Registries in the Field of Rare Diseases: Focus on Neurogenetic Diseases.Neurology · 2024Article
- Paediatric Drug Development in China: Current Status and Future Prospects.Paediatric drugs · 2024Review
- Real-world evidence to support regulatory submissions: A landscape review and assessment of use cases.Clinical and translational science · 2024Review
- Real-world evidence reported for clinical efficacy evaluation in European Public Assessment Reports of authorised targeted therapies for solid malignancies: a comprehensive review (2018-2022).ESMO real world data and digital oncology · 2024Article
- [The application of real-world evidence in drug regulatory decision-making].Bundesgesundheitsblatt, Gesundheitsforschung, Gesundheitsschutz · 2024Review
- Article
- Regulatory Procedures and Integrated Requirements for Early Phase Development of a Pharmaceutical Drug Product.Reviews on recent clinical trials · 2024Article
- Design, implementation, and inferential issues associated with clinical trials that rely on data in electronic medical records: a narrative review.BMC medical research methodology · 2023Review
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
Real-world data/real-world evidence (RWD/RWE) are considered to have a great potential to complement, in some cases, replace the evidence generated through randomized controlled trials. By tradition, use of RWD/RWE in the postauthorization phase is well-known, whereas published evidence of use in the pre-authorization phase of medicines development is lacking. The primary aim of this study was to identify and quantify the role of potential use of RWD/RWE (RWE signatures) during the pre-authorization phase, as presented in the initial marketing authorization applications of new medicines centrally evaluated with a positive opinion in 2018-2019 (n = 111) by the European Medicines Agency (EMA). Data for the study was retrieved from the evaluation overviews of the European Public Assessment Reports (EPARs), which reflect the scientific conclusions of the assessment process and are accessible through the EMA website. RWE signatures were extracted into an RWE Data Matrix, including 11 categories divided over 5 stages of the drug development lifecycle. Nearly all EPARs included RWE signatures for the discovery (98.2%) and life-cycle management (100.0%). Half of them included RWE signatures for the full development phase (48.6%) and for supporting regulatory decisions at the registration (46.8%), whereas over a third (35.1%) included RWE signatures for the early development. RWE signatures were more often seen for orphan and conditionally approved medicines. Oncology, hematology, and anti-infectives stood out as therapeutic areas with most RWE signatures in their full development phase. The findings bring unprecedented insights about the vast use of RWD/RWE in drug development supporting the regulatory decision making.
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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.