ReviewClinical pharmacology and therapeutics2022
Marketing Authorization Applications Made to the European Medicines Agency in 2018-2019: What was the Contribution of Real-World Evidence?
Review 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 60 papers, 5 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
60 citing papers in PubMed, 5 syntheses or guidelines pooled it.
- Treatment Effects in Randomized and Nonrandomized Studies of Pharmacological Interventions: A Meta-Analysis.JAMA network open · 2024Pooled it
- Pooled it
- Methodological considerations on real-world evidence studies of monoclonal antibodies against the CGRP-pathway for migraine: a systematic review.The journal of headache and pain · 2023Pooled it
- Randomized Versus Real-World Evidence on the Efficacy and Toxicity of Checkpoint Inhibitors in Cancer in Patients with Advanced Non-small Cell Lung Cancer or Melanoma: A Meta-analysis.Targeted oncology · 2022Pooled 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
- Comparability of external and internal control patients for the prospective randomized HOVON-103 trial in older AML patients.British journal of haematology · 2026Trial
- Considerations for the Integration of Clinical Trials and Real-World Data.Clinical pharmacology and therapeutics · 2026Review
- External Control Arms in Japan: A Systematic Case Analysis of Documented Regulatory Implementation and Uncertainty Management.Clinical pharmacology and therapeutics · 2026Article
- Toward Fit-for-Purpose Data for Drug Assessment in Non-small Cell Lung Cancer: A Core Dataset.Clinical pharmacology and therapeutics · 2026Article
- A Comprehensive Mapping of Real-World Studies and Data Sources for Oncology in China: Insights into Landscape, Challenges, and Future Direction.Oncology and therapy · 2026Review
- Core Concepts in Pharmacoepidemiology-Round 3: Call for Writing Groups.Pharmacoepidemiology and drug safety · 2026Article
- Methods Applied to Assess Real-World Effectiveness of Drugs: A Scoping Review.Pharmacology research & perspectives · 2026Article
- Use of Real-World Evidence in Health Technology Assessments of Non-Oncology Rare Disease Therapies.Journal of market access & health policy · 2026Article
- Assessing Research Impact With Bibliometrics and Altmetrics: The Case of the Canadian Network for Observational Drug Effect Studies (CNODES).Pharmacoepidemiology and drug safety · 2026Article
- Cascade Valorisation of Lemon Processing Residues (Part II): Integrated Biorefinery Design, Circular Economy, and Techno-Economic Feasibility.Foods (Basel, Switzerland) · 2026Review
- Advancing real-world evidence in Brazil: regulatory gaps and global lessons.Lancet regional health. Americas · 2026Review
- Real-world data and evidence: pioneering frontiers in precision oncology.Journal of Zhejiang University. Science. B · 2025Review
- Key Stakeholders' Knowledge, Opinions, and Interests on Real-World Evidence in the Regulatory Process-Results of an EU-Wide Survey.Clinical and translational science · 2025Article
- Evolving Real-World Data and Evidence Use for New Drugs and Regenerative Medical Products Approvals in Japan-An Analysis of the 6-Year Trend.Clinical pharmacology and therapeutics · 2025Review
- Acceptance and Utilization of Real-World Evidence among Cancer Care Physicians in the USA: A National Survey.Pharmaceutical medicine · 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
9 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Information derived from routinely collected real-world data has for a long time been used to support regulatory decision making on the safety of drugs and has more recently been used to support marketing authorization submissions to regulators. There is a lack of detailed information on the use and types of this real-world evidence (RWE) as submitted to regulators. We used resources held by the European Medicines Agency (EMA) to describe the characteristics of RWE included in new marketing authorization applications (MAAs) and extensions of indication (EOIs) for already authorized products submitted to the EMA in 2018 and 2019. For MAAs, 63 of 158 products (39.9%) contained RWE with a total of 117 studies. For 31.7% of these products, the RWE submitted was derived from data collected before the planned authorization. The most common data sources were registries (60.3%) followed by hospital data (31.7%). RWE was mainly included to support safety (87.3%) and efficacy (49.2%) with cohort studies being the most frequently used study design (88.9%). For EOIs, 28 of 153 products (18.3%) contained RWE with a total of 36 studies. For 57.1% of these products, studies were conducted prior to the EOIs. RWE sources were mainly registries (35.6%) and hospital data (27.0%). RWE was typically used to support safety (82.1%) and efficacy (53.6%). Cohort studies were the most commonly used study design (87.6%). We conclude that there is widespread use of RWE to support evaluation of MAAs and EOIs submitted to the EMA and identify areas where further research is required.
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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.