Therapeutic profile of orphan medicines

Pharmacoepidemiology and Drug Safety
Carla Maria Teixeira de Barros, Ana Luísa Papoila


To characterise the therapeutic profile of orphan medicines. A cross-sectional study was performed during 2 months in a convenience sample of seven hospital pharmacy services, in the region of Lisbon. Data were collected, from pharmaceutical service's records. A total of 18 orphan medicines, were dispensed to 355 patients with rare diseases. Most patients were adults (76.4%). Premature and neonates accounted with 50.0% of the paediatric patients. Differences were not found between the proportion of male and female patients across age groups (p = 0.762). Only 18.3% were inpatients. A high proportion of paediatric inpatients (58.3%) were seen in relation to adult inpatients (5.9%) (p = 0.001). In general, anti-neoplastic and immunomodulating agents for rare cancers were the most frequent dispensed medicines (51.3%). In relation to paediatrics, Caffeine Citrate for primary apnoea of premature newborns had the higher frequency distribution (57.1%). Five (71.4%) medicines dispensed for paediatrics, do not have market authorisation and the remaining (28.6%) were used off-label. For pulmonary arterial hypertension 19 of 27 patients (70.3%) were treated with Bosentan. According to evidence-based clinical practice guidelines, Bosentan h...Continue Reading


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Oct 15, 2008·Current Opinion in Psychiatry·Michael KoelchJoerg M Fegert
Jan 20, 2010·Drug Safety : an International Journal of Medical Toxicology and Drug Experience·Harald E HeemstraHubert G M Leufkens
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