ArticleTrials2010
Compassionate use of interventions: results of a European Clinical Research Infrastructures Network (ECRIN) survey of ten European countries.
Article in Trials, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 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
24 citing papers in PubMed, 1 synthesis or guideline pooled it.
- A National Approach to Reimbursement Decision-Making on Drugs for Rare Diseases in Canada? Insights from Across the Ponds.Healthcare policy = Politiques de sante · 2015Pooled it
- Access to anticancer and orphan medicines through compassionate use programs and named patient basis in seven European countries.ESMO open · 2025Article
- Use of convalescent plasma in COVID-19 treatment: is clinical severity more important than the intervention?Einstein (Sao Paulo, Brazil) · 2024Article
- International Country-Level Trends, Factors, and Disparities in Compassionate Use Access to Unlicensed Products for Patients With Serious Medical Conditions.JAMA health forum · 2022Article
- Early Access to Medicines: Use of Multicriteria Decision Analysis (MCDA) as a Decision Tool in Catalonia (Spain).Journal of clinical medicine · 2022Article
- Long-term survival analysis of masitinib in amyotrophic lateral sclerosis.Therapeutic advances in neurological disorders · 2021Article
- Systematic review of extracellular vesicle-based treatments for lung injury: are EVs a potential therapy for COVID-19?Journal of extracellular vesicles · 2020Review
- Characterizing expanded access and compassionate use programs for experimental drugs.BMC research notes · 2017Article
- Ethics review in compassionate use.BMC medicine · 2017Article
- An overview of Compassionate Use Programs in the European Union member states.Intractable & rare diseases research · 2016Review
- An analysis of common ethical justifications for compassionate use programs for experimental drugs.BMC medical ethics · 2016Article
- Compassionate use of drugs and medical devices in the United States, the European Union and Japan.Regenerative therapy · 2016Article
- The potential exploitation of research participants in high income countries who lack access to health care.British journal of clinical pharmacology · 2016Review
- Article
- Ethics of trial drug use: to give or not to give?BEOnline : journal of the West African bioethics training program · 2016Article
- Adapting Drug Approval Pathways for Bacteriophage-Based Therapeutics.Frontiers in microbiology · 2016Review
- Phage Therapy: Combating Infections with Potential for Evolving from Merely a Treatment for Complications to Targeting Diseases.Frontiers in microbiology · 2016Review
- Compassionate use of experimental therapies: who should decide?EMBO molecular medicine · 2015Article
- Compassionate use of orphan drugs.Orphanet journal of rare diseases · 2015Article
- Expanded Access Programs: Ethical and Practical Considerations for Biopharmaceutical Sponsors.Therapeutic innovation & regulatory science · 2015Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
31 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
background'Compassionate use' programmes allow medicinal products that are not authorised, but are in the development process, to be made available to patients with a severe disease who have no other satisfactory treatment available to them. We sought to understand how such programmes are regulated in ten European Union countries.
methodsThe European Clinical Research Infrastructures Network (ECRIN) conducted a comprehensive survey on clinical research regulatory requirements, including questions on regulations of 'compassionate use' programmes. Ten European countries, covering approximately 70% of the EU population, were included in the survey (Austria, Denmark, France, Germany, Hungary, Ireland, Italy, Spain, Sweden, and the UK).
resultsEuropean Regulation 726/2004/EC is clear on the intentions of 'compassionate use' programmes and aimed to harmonise them in the European Union. The survey reveals that different countries have adopted different requirements and that 'compassionate use' is not interpreted in the same way across Europe. Four of the ten countries surveyed have no formal regulatory system for the programmes. We discuss the need for 'compassionate use' programmes and their regulation where protection of patients is paramount.
conclusions'Compassionate use' is a misleading term and should be replaced with 'expanded access'. There is a need for expanded access programmes in order to serve the interests of seriously ill patients who have no other treatment options. To protect these patients, European legislation needs to be more explicit and informative with regard to the regulatory requirements, restrictions, and responsibilities in expanded access programmes.
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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.