ArticleClinical pharmacology and therapeutics2023
Contribution of Real-World Evidence in European Medicines Agency's Regulatory Decision Making.
Article in Clinical pharmacology and therapeutics, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 40 papers.
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
40 citing papers in PubMed.
- Comparability of external and internal control patients for the prospective randomized HOVON-103 trial in older AML patients.British journal of haematology · 2026Trial
- Target trial emulation to assess real-world efficacy in the Epidemiological Strategy and Medical Economics metastatic breast cancer cohort.Journal of the National Cancer Institute · 2023Trial
- Repurposing Registries: Completeness of Real-World Data for Regulatory and HTA Purposes in Three Cancer-Focused Registries.Clinical pharmacology and therapeutics · 2026Article
- Contribution of Real-World Evidence for the Orphan Medicines Approvals in the European Union Between 2000 and 2025.Therapeutic innovation & regulatory science · 2026Article
- Toward Fit-for-Purpose Data for Drug Assessment in Non-small Cell Lung Cancer: A Core Dataset.Clinical pharmacology and therapeutics · 2026Article
- Epidemiology, temporal trends, and fibrosis risk stratification in metabolic dysfunction-associated steatotic liver disease in UK primary care: a population-based cohort and nested case-control study.The Lancet regional health. Europe · 2026Article
- Risk of Bias Associated with Varying Sampling Strategies for External Comparator Cohorts for Single-Arm Trials: Insights from Aggressive Lymphoma.Clinical pharmacology and therapeutics · 2026Article
- Methods Applied to Assess Real-World Effectiveness of Drugs: A Scoping Review.Pharmacology research & perspectives · 2026Article
- Assessing Overall Survival Benefits in Advanced Cancers: The Role of External Comparator Cohort Studies with Real-World Data.Clinical pharmacology and therapeutics · 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
- Article
- Emulating the LEADER trial in China: a regulatory science case study on non-interventional research.Frontiers in endocrinology · 2026Article
- Factors influencing pharmaceutical companies' decisions to pursue compassionate use programs in the EU: a qualitative study in The Netherlands.Journal of pharmaceutical policy and practice · 2026Article
- Navigating the Real World: A Scoping Review of Structured Frameworks to Effectively Identify, Evaluate, and Select Real-World Data Sources for Fit-for-Purpose Studies.Clinical pharmacology and therapeutics · 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
- Advancing Real-World Evidence Through a Federated Health Data Network (EHDEN): Descriptive Study.Journal of medical Internet research · 2025Article
- Stakeholders' Perspectives Toward the Use of Patient Registry Data for Decision-Making on Medicines: A Cross-Sectional Survey.Drug safety · 2025Article
- Incorporating genetic data improves target trial emulations and informs the use of polygenic scores in randomized controlled trial design.Nature genetics · 2025Article
- Untangling the Biosimilars Interchangeability Puzzle: A Provocative Dive into Concepts and Terminology, and Developing a Strategy to Minimize Uncertainties from Interchangeability.Therapeutic innovation & regulatory science · 2025Review
- Canakinumab treatment patterns in sJIA, FMF, TRAPS, and MKD/HIDS: real-world insights from a Belgian non-interventional study.BMC rheumatology · 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
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Real-world data/evidence (RWD/RWE) may provide insightful information on medicines' clinical effects to guide regulatory decisions. While its contribution has been recognized for safety monitoring and disease epidemiology across medicines' life cycles, using RWD/RWE to demonstrate efficacy requires further evaluation. This study aimed to (i) characterize RWD/RWE presented by applicants to support claims on medicines' efficacy within initial marketing authorization applications (MAAs) and extension of indication applications (EoIs), and (ii) analyze the contribution of RWD/RWE to regulatory decisions on medicines' benefit-risk profile. RWD/RWE was included to support efficacy in 32 MAAs and 14 EoIs submitted 2018-2019. Of these, RWD/RWE was part of the preauthorization package of 16 MAAs and 10 EoIs, and was (i) considered supporting the regulatory decision in 10 applications (five MAAs, five EoIs), (ii) considered not supporting the regulatory decision in 11 (seven MAAs, four EoIs), and (iii) not addressed at all in the evaluation of 5 applications (four MAAs, one EoI). Common limitations of submitted RWD/RWE included missing data, lack of representativeness of populations, small sample size, absence of an adequate or prespecified analysis plan, and risk of several types of bias. The suitability of RWD/RWE in a given application still requires a case-by-case analysis considering its purpose of use, implying reflection on the data source, together with its assets and limitations, study objectives and designs, and the overall data package issued. Early interactions and continuous dialogues with regulators and relevant stakeholders is key to optimize fit-for-purpose RWE generation, enabling its broader use in medicines development.
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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.