Galenic formulations in children for rare diseases or to call for a different pharmaceutical form from those on the market: Data collection and statistical study

Abstract

The dosage and route of administration are the two key points in the planning of therapy designed for a child who needs medication appropriate to his/her physiological characteristics, that are different compared to the adults. The "pediatric clinic Galen" is a valid tool for ensuring a highly personalized treatment in and out of hospital. A formulation must, where possible, be adapted to the patient, while guaranteeing the quality, safety and efficacy of the preparation, a good palatability and a "reasonable" period of validity, but it also must be designed in such a way as to facilitate the adult administering the child without forgetting the young patient's compliance, and enable them to give the right dosage, without making easy unintentional mistakes. From 2009 to 2014, at the galenic laboratory of Versilia Hospital pharmacy, galenic formulations were prepared for about 225 children, to treat acute diseases such as gastroesophageal reflux, or follow a chronic therapy, for example in case of metabolic deficiencies, or rare syndromes. What has been performed is a retrospective statistical study of requirements and activity of the service and a collection of users and operators' feedback to determine the social impact