ReviewClinical and translational science2024
Real-world evidence to support regulatory submissions: A landscape review and assessment of use cases.
Review in Clinical and translational science, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 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
27 citing papers in PubMed.
- Beyond Blind Trust in Hospital Data Warehouses: Evaluating Real-World Data Accountability.Learning health systems · 2026Article
- External Control Arms in Japan: A Systematic Case Analysis of Documented Regulatory Implementation and Uncertainty Management.Clinical pharmacology and therapeutics · 2026Article
- A Practical Guide to Implementing Real-World Evidence as Primary Basis for Approval and Label Expansion.Clinical pharmacology and therapeutics · 2026Article
- Real-world data sources for pediatric cancers: a targeted literature review.ESMO real world data and digital oncology · 2026Article
- AI-Enabled Real-World Evidence in Oncology: A Statistical Perspective for Regulatory Decisions.Therapeutic innovation & regulatory science · 2026Review
- Use of real-world evidence in the U.S. Food and Drug Administration and the European Medicines Agency approvals of oncology products supported by single-arm trials (2022-2025).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
- Real-world effectiveness of pembrolizumab for first-line treatment of metastatic non-small-cell lung cancer: a decade of evidence and experience.Future oncology (London, England) · 2026Review
- Conduction of Pragmatic Clinical Trials in Routine Clinical Settings: Guidelines for Development of Protocol.Current therapeutic research, clinical and experimental · 2026Review
- Introducing the J-CONNECT database: a real-world oncology resource for Japan.International journal of clinical oncology · 2026Review
- What the future holds: the growing role of real-world evidence in health technology assessment in Malaysia.International journal of technology assessment in health care · 2026Article
- Real-World Effectiveness Following Benralizumab Use in Patients with Severe Eosinophilic Asthma in Romania: A Retrospective Cohort Study (BREEZE).Journal of clinical medicine · 2026Article
- Harnessing patient health data to benefit individuals and advance research: a focus on rheumatoid arthritis - a narrative review.BMJ digital health & AI · 2026Review
- Emulating the LEADER trial in China: a regulatory science case study on non-interventional research.Frontiers in endocrinology · 2026Article
- Comparison of the conditional approvals for anticancer drugs supported by single-arm trials in China and the United States: clinical evidence, post-marketing requirements, and regulatory outcomes.Frontiers in pharmacology · 2026Article
- The future directions of CAR-T Cell therapy: unlocking the potential of immunotherapy in cancer treatment.Frontiers in molecular medicine · 2026Review
- Article
- 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
- Data-Driven Medicine and Artificial Intelligence in Healthcare: Real-World Evidence, Opportunities, and Safeguards.International urogynecology journal · 2025Article
- Surrogate Biomarkers in Gene Therapy for Orphan Diseases: Validation, Application, and Regulatory Aspects.International journal of molecular sciences · 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
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Real-world evidence (RWE) has an increasing role in preapproval settings to support the approval of new medicines and indications. The main objectives of this study were to identify and characterize regulatory use cases that utilized RWE and other related observational approaches through targeted review of publications and regulatory review documents. After screening and inclusion/exclusion, the review characterized 85 regulatory applications with RWE. A total of 31 were in oncology and 54 were in non-oncology therapeutic areas. Most were for indications in adults only (N = 42, 49.4%), while 13 were in pediatrics only (15.3%), and 30 were in both (35.3%). In terms of regulatory context, 59 cases (69.4%) were for an original marketing application, 24 (28.2%) were for label expansion, and 2 (2.4%) were for label modification. Most also received special regulatory designations (e.g., orphan indication, breakthrough therapy, fast track, conditional, and accelerated approvals). There were 42 cases that utilized RWE to support single-arm trials. External data to support single-arm trials were utilized in various ways across use cases, including direct matching, benchmarking, natural history studies as well as literature or previous trials. A variety of data sources were utilized, including electronic health records, claims, registries, site-based charts. Endpoints in oncology use cases commonly included overall survival, progression-free survival. In 13 use cases, RWE was not considered supportive/definitive in regulatory decision-making due to design issues (e.g., small sample size, selection bias, missing data). Overall, RWE is utilized in regulatory approval processes for new indications/label expansion across various therapeutic areas with wide range of approaches. Multifaceted cross-sector efforts are needed to further improve the quality and utility of RWE in 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.