ArticleEuropean journal of cancer (Oxford, England : 1990)2021
Real-world outcomes associated with new cancer medicines approved by the Food and Drug Administration and European Medicines Agency: A retrospective cohort study.
Article in European journal of cancer (Oxford, England : 1990), 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 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
22 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Pooled it
- Augmenting randomized clinical trial data with historical control data: Precision medicine applications.Journal of the National Cancer Institute · 2023Trial
- A Comprehensive Mapping of Real-World Studies and Data Sources for Oncology in China: Insights into Landscape, Challenges, and Future Direction.Oncology and therapy · 2026Review
- Improving survival outcomes of people with ABC: a global expert review and call-to-action for 2025-2035 (Goal 1).Breast (Edinburgh, Scotland) · 2025Review
- Real-world management and outcomes of immune-related adverse events in German cancer care: A multicenter analysis using the SERIO registry.The oncologist · 2025Article
- The WHO global landscape of cancer clinical trials.Nature medicine · 2025Review
- Strategies to reduce the cancer burden and improve access to effective and affordable cancer interventions in Europe.Molecular oncology · 2025Article
- The role and value of real-world evidence in health technology decision-making in France, Germany, Italy, Spain, and the UK: insights on external control arms.International journal of technology assessment in health care · 2025Review
- Characteristics and impact of real-world evidence studies in oncology: comprehensive mapping review of publications evaluating targeted therapies in solid tumours.ESMO real world data and digital oncology · 2024Review
- Academia and society should join forces to make anti-cancer treatments more affordable.Molecular oncology · 2024Review
- Real-world evidence reported for clinical efficacy evaluation in European Public Assessment Reports of authorised targeted therapies for solid malignancies: a comprehensive review (2018-2022).ESMO real world data and digital oncology · 2024Article
- Real-world evidence in the reassessment of oncology therapies: payer perceptions from five countries.Future oncology (London, England) · 2024Article
- Cancer medicines: a private vice for public benefit?Ecancermedicalscience · 2024Article
- ESMO Guidance for Reporting Oncology real-World evidence (GROW).ESMO real world data and digital oncology · 2023Article
- The Innovative Medicines Fund: a universal model for faster and fairer access to new promising medicines or a Trojan horse for low-value creep?Journal of the Royal Society of Medicine · 2023Article
- Analysis of the characteristics and the degree of pragmatism exhibited by pragmatic-labelled trials of antineoplastic treatments.BMC medical research methodology · 2023Review
- Changes in survival in de novo metastatic cancer in an era of new medicines.Journal of the National Cancer Institute · 2023Article
- Association Between Age and Survival Trends in Advanced Non-Small Cell Lung Cancer After Adoption of Immunotherapy.JAMA oncology · 2023Article
- Outcomes of Chemoimmunotherapy Among Patients With Extensive-Stage Small Cell Lung Cancer According to Potential Clinical Trial Eligibility.JAMA network open · 2023Article
- Implications of Oncology Trial Design and Uncertainties in Efficacy-Safety Data on Health Technology Assessments.Current oncology (Toronto, Ont.) · 2022Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors.
Funding
Abstract
purposeReal-World Data (RWD) studies are increasingly used to support regulatory approvals, reimbursement decisions, and changes in clinical practice for novel cancer drugs. However, few studies have systematically appraised their quality or compared outcomes to pivotal trials.
methodsAll RWD studies (2010-2019) for drugs approved by the Food and Drug Administration (FDA) and European Medicines Agency (EMA) from 2010 to 2015 for solid organ tumours in the non-curative setting were identified. Quality assessment was undertaken using the Newcastle Ottawa Scale. Survival differences between each RWD study and the pivotal trial were determined using a related sample Wilcoxon signed-rank test.
results293 RWD studies for 45 of the 57 drug indications approved by the FDA/EMA were identified. The most common tumour types were prostate cancer (29%, n = 86) and melanoma (15%, n = 43). A quarter of the studies had industry funding. No high-quality studies were identified, and 78% were low quality. Comparative survival analysis between RWD and pivotal trials was possible for 224 studies (37 drug indications). Differences in median survival between the RWD studies and their corresponding trial ranged from -32 months to 21 months (IQR -4·2 months to 1·6 months). Low-quality studies were more likely to report superior survival outcomes (23%) compared to higher quality studies (8%) (p = 0.02).
conclusionRWD study quality for novel cancer drugs is low and of insufficient rigour to inform reimbursement decisions and clinical practice. RWD studies seeking publication should provide a completed quality assessment tool on submission. Greater investment in properly designed RWD studies is required.
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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.