ArticleOrphanet journal of rare diseases2013
Roll out of intraveneous artesunate under named patient programmes in the Netherlands, Belgium and France.
Article in Orphanet journal of rare diseases, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.
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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.
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Who cites it
6 citing papers in PubMed, 5 citations in OpenAlex.
- Pediatric malaria in a non-endemic European setting: clinical patterns, laboratory markers, and the ambiguous role of immunity.Infection · 2026Article
- Access to anticancer and orphan medicines through compassionate use programs and named patient basis in seven European countries.ESMO open · 2025Article
- Trends in orphan medicinal products approvals in the European Union between 2010-2022.Orphanet journal of rare diseases · 2024Article
- Access to medicines for rare diseases: A European regulatory roadmap for academia.Frontiers in pharmacology · 2023Article
- Severe malaria in Europe: an 8-year multi-centre observational study.Malaria journal · 2017Observational
- The influence of the European paediatric regulation on marketing authorisation of orphan drugs for children.Orphanet journal of rare diseases · 2014Article
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Authors and funding
3 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundIntravenous (IV) artesunate is the treatment of choice for severe malaria. In Europe, this treatment is only available in a few countries via named patient programmes (NPPs). As a case study, the legal and organisational aspects and pharmacovigilance of these NPPs and possibilities for harmonisation within the EU were studied over time and space using IV artesunate (Malacef) in the Netherlands, Belgium and France.
methodsThe legal base and organisation of NPPs in the Netherlands, Belgium and France were studied. The diffusion and cumulative availability of IV artesunate and the pharmacovigilance components were compared among the three countries using distribution data from the period 2007 through 2012.
resultsArtesunate has quickly gained acceptance for treating severe malaria in the Netherlands, whereas both Belgium and France have introduced this treatment more hesitantly. This difference in acceptance is due to differences in the implementation of NPP legislation among the countries. France currently has a proactive system in which treatment requires the permission for each patient and an intensive follow-up protocol. On the other hand, Belgium and Dutch NPPs are more dependent on the investigators' initiative and are therefore potentially faster and more flexible, facilitating the discovery of adverse effects that have not been reported by more formal comparative clinical trials.
conclusionsNPPs provide a unique opportunity to study both the benefits and risks of unregistered products for treating rare diseases, provided that the patients are actively vigilated. Thus, we recommend that NPPs should be harmonised throughout Europe in order to ensure equal availability of treatment and therapeutic benefit to all Europeans without compromising patient safety.
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