ReviewClinical pharmacology and therapeutics2022
Use of Real-World Evidence to Drive Drug Development Strategy and Inform Clinical Trial Design.
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 78 papers, 1 of them a synthesis 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
78 citing papers in PubMed, 1 synthesis or guideline pooled 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
- Patient-Reported Outcomes Used for Regulatory Approval of Medicines Indicated for the Treatment of Dermatological Diseases Authorized in the European Union Between 1998 and 2024.Therapeutic innovation & regulatory science · 2026Article
- Addressing Unmet Needs in the Management of Small Cell Lung Cancer: Treatment of Brain Metastases, Quality of Life, and the Role of Real-World Evidence.Current treatment options in oncology · 2026Review
- Maximizing the impact of real-world evidence on cancer treatment: what do we need to achieve it?Journal of comparative effectiveness research · 2026Review
- Prescription of off-label medications in patients on dialysis: time to challenge contraindications?Clinical kidney journal · 2026Review
- AI-guided ethnopharmacology for cardiovascular drug discovery: from biomedical data to experimental validation.Frontiers in pharmacology · 2026Review
- Genetic pharmacoepidemiology of JAK inhibitors in chronic immune-mediated skin diseases: implications for precision therapy and medication safety.Frontiers in pharmacology · 2026Article
- The Evolution of Medical Affairs Central Teams: Shaping Strategic Action and Driving Healthcare Transformation Through Scientific Interaction.Pharmaceutical 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
- Review
- Article
- Crossing borders securely: synthetic data and federated networks for privacy-preserving access to real-world data and emerging use cases.NPJ digital medicine · 2025Review
- Can Real-World Data From Hospital Information Systems Be Directly Utilized for Efficacy Studies: An Analysis of Data Time Points.Journal of evidence-based medicine · 2025Article
- AMCP real-world evidence standards: Overcoming barriers to using real-world evidence in US payer decision-making.Journal of managed care & specialty pharmacy · 2025Article
- Hope is Not a Strategy: Using Robust Real-World Evidence to Make Better Clinical Development Decisions.Therapeutic innovation & regulatory science · 2025Article
- Real-World Data and Causal Machine Learning to Enhance Drug Development.Therapeutic innovation & regulatory science · 2025Review
- Real-world data landscape for glaucoma in Europe: a questionnaire-based analysis of resources among European Glaucoma Society members.BMJ open ophthalmology · 2025Article
- Large Language Models for Automating Clinical Trial Criteria Conversion to Observational Medical Outcomes Partnership Common Data Model Queries: Validation and Evaluation Study.JMIR medical informatics · 2025Article
- Combining Real-World and Clinical Trial Data Through Privacy-Preserving Record Linkage: Opportunities and Challenges-A Narrative Review.Health science reports · 2025Article
- CLEAR: A vision to support clinical evidence lifecycle with continuous learning.Journal of biomedical informatics · 2025Article
18 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
No grant is acknowledged in the PubMed record.
Abstract
Interest in real-world data (RWD) and real-world evidence (RWE) to expedite and enrich the development of new biopharmaceutical products has proliferated in recent years, spurred by the 21st Century Cures Act in the United States and similar policy efforts in other countries, willingness by regulators to consider RWE in their decisions, demands from third-party payers, and growing concerns about the limitations of traditional clinical trials. Although much of the recent literature on RWE has focused on potential regulatory uses (e.g., product approvals in oncology or rare diseases based on single-arm trials with external control arms), this article reviews how biopharmaceutical companies can leverage RWE to inform internal decisions made throughout the product development process. Specifically, this article will review use of RWD to guide pipeline and portfolio strategy; use of novel sources of RWD to inform product development, use of RWD to inform clinical development, use of advanced analytics to harness "big" RWD, and considerations when using RWD to inform internal decisions. Topics discussed will include the use of molecular, clinicogenomic, medical imaging, radiomic, and patient-derived xenograft data to augment traditional sources of RWE, the use of RWD to inform clinical trial eligibility criteria, enrich trial population based on predicted response, select endpoints, estimate sample size, understand disease progression, and enhance diversity of participants, the growing use of data tokenization and advanced analytical techniques based on artificial intelligence in RWE, as well as the importance of data quality and methodological transparency in RWE.
Indexed as
Identifiers
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.