Abstract
BACKGROUND: Intravenous (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. METHODS: The 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. RESULTS: Artesunate 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. CONCLUSIONS: NPPs 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.