ReviewClinical pharmacology and therapeutics2025
PMDA Perspective on Use of Real-World Data and Real-World Evidence as an External Control: Recent Examples and Considerations.
Review in Clinical pharmacology and therapeutics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 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
8 citing papers in PubMed.
- External Control Arms in Japan: A Systematic Case Analysis of Documented Regulatory Implementation and Uncertainty Management.Clinical pharmacology and therapeutics · 2026Article
- Feasibility of Reconstructing Survival Outcomes from Unstructured Electronic Medical Records under Japan's Next-Generation Medical Infrastructure Act: A Historical Cohort Study of Atezolizumab in Non-small Cell Lung Cancer.Drugs - real world outcomes · 2026Article
- A scoping review of real-world data sources for retrospective oncology analysis in Japan.ESMO real world data and digital oncology · 2026Review
- Characteristics and Future Perspectives of Available Electronic Health Record Databases in Japan.Drugs - real world outcomes · 2026Review
- Challenges in Implementing Revocation of Oncology Drugs Under Conditional Early Approval in Japan: Regulatory Insights From Comparisons With FDA/EMA.Clinical and translational science · 2026Article
- The impact of China's drug regulatory reform on access to orphan drugs: a cross-sectional study of diseases listed in the rare disease catalog.Frontiers in pharmacology · 2026Article
- Editorial: Advancement of RWD/RWE utilization for enhancing drug development and benefit/risk assessment.Frontiers in pharmacology · 2026Article
- 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
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
Recent discussions about the utilization of real-world data (RWD) and real-world evidence (RWE) have been more focused on drug development for regulatory approval rather than during the post-marketing stage. In Japan, RWD/RWE have been practically utilized as an external control for drug approval. Most cases were related to orphan diseases where the feasibility of conducting randomized controlled clinical trials was generally low. The utilization of RWD/RWE as an external control provides additional information that can support regulatory review for drug approval. However, many points should be taken into consideration through all stages of a study that is based on RWD/RWE, including planning, analysis, and interpretation. In this article, we present our recent review experience focusing on efficacy evaluations with an external control based on RWD/RWE that were submitted as a part of new drug applications in Japan, and we describe our regulatory consideration of the utilization of RWD/RWE for drug evaluation and approval. Points described in this article promote appropriate drug development based on RWD/RWE and facilitate a proper discussion about RWD/RWE utilization with PMDA. Further accumulation of regulatory experience in PMDA with RWD/RWE utilization will enhance our knowledge and contribute to better regulatory decision making for drug approvals based on RWD/RWE.
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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.