P77 Pediatric drug data in Canadian drug monographs

BackgroundOptimal drug therapy in children relies on availability of pediatric-specific information. European and American legislative initiatives have resulted in advancement of pediatric pharmacotherapy data. We aim to describe the quality and quantity of pediatric information in drug monographs of New Active Substances (NASs) approved by Health Canada.Design/MethodsCanadian drug monographs of NASs approved by Health Canada, from January 2007 until December 2016, were systematically reviewed for pediatric-specific information. Pediatric-specific information defined as: pediatric indication, dosing, pediatric-friendly dosage forms, and pediatric safety data.ResultsOver the period of the study, Health Canada approved 281 NASs. Of all the non-biologic NASs (205, 74%), 39(19%) were approved for use in pediatric patients. The number of drugs with pediatric approval was lowest in 2008 (1, 8%) and highest in 2016 (8, 32%), following no specific pattern. Neonates had the lowest rate of drug approvals through all pediatric age groups (4, 2%). All drugs with pediatric approval had pediatric-specific dosing information with the majority of them presenting pediatric safety data (79%). Pediatric friendly formulation was only available in 20%(8) of drugs with pediatric approval. Studies in pediatric populations were the source of pediatric information in 59%(23) of drugs with pediatric approval.Conclusion(s)Less than 20% of the NASs approved by Health Canada for use in adults contain pediatric approval. Neonatal populations remain a therapeutic orphan, with severe lack of dosing and safety information. Safe and effective pediatric pharmacotherapy requires well-conducted pediatric research to enhance pediatric drug data. Canadian children are in need for legislative initiatives to promote pediatric drug development.Disclosure(s)Nothing to disclose

Biliary Atresia: 50 Years after the First Kasai

Biliary atresia is a rare neonatal disease of unknown etiology, where obstruction of the biliary tree causes severe cholestasis, leading to biliary cirrhosis and death in the first years of life, if the condition is left untreated. Biliary atresia is the most frequent surgical cause of cholestatic jaundice in neonates and should be evoked whenever this clinical sign is associated with pale stools and hepatomegaly. The treatment of biliary atresia is surgical and currently recommended as a sequence of, eventually, two interventions. During the first months of life a hepatoportoenterostomy (a “Kasai,” modifications of which are discussed in this paper) should be performed, in order to restore the biliary flow to the intestine and lessen further damage to the liver. If this fails and/or the disease progresses towards biliary cirrhosis and life-threatening complications, then liver transplantation is indicated, for which biliary atresia represents the most frequent pediatric indication. Of importance, the earlier the Kasai is performed, the later a liver transplantation is usually needed. This warrants a great degree of awareness of biliary atresia, and the implementation of systematic screening for this life-threatening pathology.