SynthesisClinical pharmacology and therapeutics2022
The Role of Real-World Evidence in FDA-Approved New Drug and Biologics License Applications.
Synthesis 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 79 papers, 6 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
79 citing papers in PubMed, 6 syntheses or guidelines pooled it.
- Methodological challenges using routine clinical care data for real-world evidence: a rapid review utilizing a systematic literature search and focus group discussion.BMC medical research methodology · 2025Pooled it
- Treatment Effects in Randomized and Nonrandomized Studies of Pharmacological Interventions: A Meta-Analysis.JAMA network open · 2024Pooled it
- A systematic review of real-world evidence (RWE) supportive of new drug and biologic license application approvals in rare diseases.Orphanet journal of rare diseases · 2024Pooled 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
- Efficacy and Safety of Ivabradine in Combination with Beta-Blockers in Patients with Stable Angina Pectoris: A Systematic Review and Meta-analysis.Advances in therapy · 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
- Contribution of Real-World Evidence for the Orphan Medicines Approvals in the European Union Between 2000 and 2025.Therapeutic innovation & regulatory science · 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
- Leveraging generative artificial intelligence for the development of non-interventional research study protocols: a proof-of-concept feasibility study.BMC medical research methodology · 2026Article
- A preoperative Artificial Intelligence model to estimate cancer-specific mortality in nonmetastatic kidney cancer patients.Nature communications · 2026Article
- Methods Applied to Assess Real-World Effectiveness of Drugs: A Scoping Review.Pharmacology research & perspectives · 2026Article
- A Perspective for Integrating Real-World Evidence Into the Investigational Device Exemption Study Design for Lumbar Total Joint Replacement.International journal of spine surgery · 2026Article
- Clinically Meaningful, Patient-Centered Endpoints for Real-World Evidence Generation in Breast Cancer: Perspectives from the Multidisciplinary TRIUMPH Initiative.Advances in therapy · 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
- Article
- Advancing real-world evidence harmonization: lessons from the UK, EMA and global policy frameworks.Journal of comparative effectiveness research · 2026Review
- A governance framework for medical code standardization to enhance multi-institutional data quality.BMC medical informatics and decision making · 2026Article
- An Unusual Cluster of Urinary Tract Infections, Including Salmonella spp., in an Iranian Ulcerative Colitis Cohort Treated With Vedolizumab: A Case Series and Comparative Analysis.JGH open : an open access journal of gastroenterology and hepatology · 2026Article
- Sieving for gold: external comparator studies to prioritize drugs for late-stage clinical trial.Journal of comparative effectiveness research · 2026Review
- Powering responsible artificial intelligence with high-quality real-world data: the S-RACE platform for scalable, multi-specialty clinical research.NPJ digital medicine · 2026Article
19 more citing papers are in PubMed but not listed here.
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
Abstract
The US Food and Drug Administration (FDA) is open to accepting real-world evidence (RWE) to support its assessment of medical products. However, RWE stakeholders lack a shared understanding of FDA's evidentiary expectations for the use of RWE in applications for new drugs and biologics. We conducted a systematic review of publicly available FDA approval documents from January 2019 to June 2021. We sought to quantify, by year, how many approvals incorporated RWE in any form, and the intended use of RWE in those applications. Among approvals with RWE intended to support safety and/or effectiveness, we classified whether and how those studies impacted FDA's benefit-risk considerations, whether those studies were incorporated into the product label, and the therapeutic area of the medical product. Finally, we qualified FDA's documented feedback where available. We found that 116 approvals incorporated RWE in any form, with the proportion of approvals incorporating RWE increasing each year. Of these approvals, 88 included an RWE study intended to provide evidence of safety or effectiveness. Among these 88 approvals, 65 of the studies influenced FDA's final decision and 38 were included in product labels. The 88 approvals spanned 18 therapeutic areas. FDA's feedback on RWE study quality included methodological issues, sample size concerns, omission of patient level data, and other limitations. Based on these findings, we would anticipate that future guidance on FDA's evidentiary expectations of RWE use will incorporate fit-for-purpose real-world data selection and careful attention to study design and analysis.
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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.