On June 8, Viviana Giannuzzi, head of the research department and coordinator of the scientific committee of Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus, had a speech in the framework of AFI Symposium in Rimini.
The AFI Symposium is one of the italian biggest events focused on the novelties and changes of the drug industry due to the widespread application of digital technologies. Over 1400 participants, 140 speakers and 100 companies in the exhibition area participated in this edition.
Viviana Giannuzzi has been one of the influential voices of the 58th edition of the Symposium, with her speech on “Clinical and regulatory aspects of the development of paediatric medicines”.
Children are commonly considered as “therapeutic orphfans” because most of the marketed medicines are not approved for paediatric use or they have not been studied in paediatric population. Thus, when there are no valid alternatives, off-label medicines and drugs formulated for adults are used, with the possible consequences related to the ineffectiveness and increase of adverse reactions.
In order to avoid this situation, the EU Paediatric Regulation EC 1901-1902/2006 obliges pharmaceutical companies to provide for a plan that includes the design of all the studies necessary for the approval of a paediatric drug considering its specificity in different ages, the Paediatric Investigasttion Plan (PIP), or a waiver. They should be approved by the EMA for every AIC marketing authorisation (MA) procedure or variation.
In case of medicines no more covered by patent, there is a specific volunteer MAAIC procedure, the so- called Paediatric Use Marketing Authorization (PUMA). This is often followed to ask for marketing new formulations appropriate for children containing old drugs.
For the EU Paediatric Regulation, therapeutic need and gravity severity of the clinical condition are really relevant. On the basis of these criteria, EMA releases and periodically reviews the therapeutic lists, which includes medicines of a particular therapeutic necessity, divided by age, area and indication.
The most salient element of this important regulatory innovation is represented by the PIP, which includes the design of all the studies necessary for the approval of a paediatric drug considering its specificity in different ages.
In addition to these regulatory requirements and a number of methodological issuesaspetcs, there is then, from a regulatory point of view, the need to ensure adapt to the paediatric age the complex authorization procedures and ethical protection for childrens required. Consent and agreementpproval of from the child, use and re-use of data and samples and privacy protection, re-use of data and samples represent an important regulatory challenge for performing high quality research activities even in children.