Therapeutic profile of orphan medicines

Purpose To characterise the therapeutic profile of orphan medicines. Materials and Methods 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. Resu...

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Bibliographic Details
Published in:	Pharmacoepidemiology and drug safety Vol. 16; no. 4; pp. 435 - 440
Main Authors:	de Barros, Carla Maria Teixeira, Papoila, Ana Luísa
Format:	Journal Article
Language:	English
Published:	Chichester, UK John Wiley & Sons, Ltd 01-04-2007
Subjects:	Adolescent Adult Aged Antihypertensive Agents - therapeutic use Antineoplastic Agents - therapeutic use Caffeine - therapeutic use Central Nervous System Stimulants - therapeutic use Child Child, Preschool Citrates - therapeutic use Cross-Sectional Studies Drug Labeling Drug Prescriptions - statistics & numerical data drug utilisation studies Drug Utilization - statistics & numerical data Female Humans Immunologic Factors - therapeutic use Infant Infant, Newborn Male Middle Aged Orphan Drug Production - statistics & numerical data orphan medicines Pharmacy Service, Hospital - statistics & numerical data Portugal rare diseases Rare Diseases - drug therapy Sulfonamides - therapeutic use therapeutic profile
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Summary:	Purpose To characterise the therapeutic profile of orphan medicines. Materials and Methods 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. Results 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 has a good level of evidence and substantial benefit: grade of recommendation A. Conclusions Most orphan medicines dispensed to paediatrics and adults were not licensed. A special pharmacovigilance program and a risk management plan through the entire life cycle should be implemented, towards effectiveness and safety of orphan medicines. Copyright © 2006 John Wiley & Sons, Ltd.
Bibliography:	ark:/67375/WNG-N378BDM8-0 No conflict of interest was declared. istex:32BAA27C17DA5A048CAC82DA49198E963AD36707 ArticleID:PDS1315 ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 23
ISSN:	1053-8569 1099-1557
DOI:	10.1002/pds.1315