ArticleBritish journal of clinical pharmacology2020
Expanded Access as a source of real-world data: An overview of FDA and EMA approvals.
Article in British journal of clinical pharmacology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 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
19 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Pooled it
- An Industry Perspective on Compassionate Use in Europe: A Call for Change.Therapeutic innovation & regulatory science · 2026Review
- Access to anticancer and orphan medicines through compassionate use programs and named patient basis in seven European countries.ESMO open · 2025Article
- Navigating challenges and opportunities in orphan medicines: A spotlight commentary on rare diseases.British journal of clinical pharmacology · 2025Article
- Clinical Evidence Supporting FDA Approval of Gene and RNA Therapies for Rare Inherited Conditions.Paediatric drugs · 2024Article
- Investigating the influence of long-axial versus short-axial field of view PET/CT on stage migration in lymphoma and non-small cell lung cancer.Nuclear medicine communications · 2023Article
- Measuring Safety and Outcomes for the Use of Compassionate and Off-Label Therapies for Children, Adolescents, and Young Adults With Cancer in the SACHA-France Study.JAMA network open · 2023Article
- Selection Bias in Real-World Data Studies Used to Support Health Technology Assessments: A Case Study in Metastatic Cancer.Current oncology (Toronto, Ont.) · 2023Article
- Access to medicines for rare diseases: A European regulatory roadmap for academia.Frontiers in pharmacology · 2023Article
- Applying real-world data from expanded-access ("compassionate use") patients to drug development.Journal of clinical and translational science · 2023Article
- International Country-Level Trends, Factors, and Disparities in Compassionate Use Access to Unlicensed Products for Patients With Serious Medical Conditions.JAMA health forum · 2022Article
- Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals.BMJ open · 2022Review
- Status of Medications Prescribed for Psychiatric Disorders in Korean Pediatric and Adolescent Patients.Children (Basel, Switzerland) · 2022Article
- Generating Evidence from Expanded Access Use of Rare Disease Medicines: Challenges and Recommendations.Frontiers in pharmacology · 2022Article
- A Systematic Review of Cost-Effectiveness Analyses of Novel Agents in the Treatment of Multiple Myeloma.Cancers · 2021Review
- Financial considerations in expanded access policy for gene therapies: A tough nut to crack?Molecular therapy : the journal of the American Society of Gene Therapy · 2021Article
- Gene therapy companies have an ethical obligation to develop expanded access policies.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Article
- Expanded Access as a source of real-world data: An overview of FDA and EMA approvals.British journal of clinical pharmacology · 2020Article
- Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
Abstract
aimsTo identify, characterize and compare all Food and Drug Administration (FDA) and European Medicines Agency (EMA) approvals that included real-world data on efficacy from expanded access (EA) programmes.
methodsCross-sectional study of FDA (1955-2018) and EMA (1995-2018) regulatory approval documentation. We automated searching for terms related to EA in 22,506 documents using machine learning techniques. We included all approvals where EA terms appeared in the regulatory documentation. Our main outcome was the inclusion of EA data as evidence of clinical efficacy. Characterization was based on approval date, disease area, orphan designation and whether the evidence was supportive or pivotal.
resultsEA terms appeared in 693 out of 22,506 (3.1%) documents, which referenced 187 approvals. For 39 approvals, data from EA programmes were used to inform on clinical efficacy. The yearly number of approvals with EA data increased from 1.25 for 1993-2013 to 4.6 from 2014-2018. In 13 cases, these programmes formed the main evidence for approval. Of these, patients in EA programmes formed over half (median 71%, interquartile range: 34-100) of the total patient population available for efficacy evaluation. Almost all (12/13) approvals were granted orphan designation. In 8/13, there were differences between regulators in approval status and valuation of evidence. Strikingly, 4 treatments were granted approval based solely on efficacy from EA.
conclusionSponsors and regulators increasingly include real-world data from EA programmes in the efficacy profile of a treatment. The indications of the approved treatments are characterized by orphan designation and high unmet medical need.
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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.