De Bruyne, Pauline

Vande Walle, Johan

English

Ghent University Academic Bibliography

1.  BACKGROUND2. METHODS4. CONCLUSIONContact information: Pauline De Bruyne; pauline.debruyne@ugent.be 3.  RESULTSFigure 1:ACKNOWLEDGEMENTSThe SAFE-PEDRUG project (IWT-SBO 130033) is supported by the Agency for Innovation by Science and Technology in Flanders (IWT).The SAFE-PEDRUG project: an opportunity for academia to close the gapPauline De Bruyne1, Johan Vande Walle2, The SAFE-PEDRUG consortium31Ghent University, GHENT, Belgium2Ghent University Hospital, GHENT, Belgium3Ghent University, Ghent University Hospital, KULeuven and VUB, GHENT, LEUVEN AND BRUSSELS, BelgiumThe Paediatric Regulation (1) was launched ten years ago. As wasalso identified in the ten year report (2), this regulation has had apositive impact on paediatric research in Europe. However, somespecific patient populations (such as neonates, critically ill children,children with comorbidities) do not receive enough attention inpaediatric drug development. Furthermore, the top-downapproach (from adults to children) results in considerable delays inmaking medicines available to children. For most of the drugs longterm follow-up is missing.The SAFE-PEDRUG project was initiated in Belgium in 2014 and is acollaboration of experts in paediatrics, pharmaceutical sciences,veterinary medicine, and ethics of three Belgian universities: GhentUniversity, KULeuven, and Vrije Universiteit Brussel. An advisoryboard and stakeholder group consisting of national andinternational stakeholders support this consortium in thevalorisation of results.The SAFE-PEDRUG project explored the value of the porcinejuvenile animal model and modelling and simulation (populationpharmacokinetics and physiologically based pharmacokineticmodelling) in providing prior paediatric PK/PD knowledge, beforethe actual adult trials have been completed. For the evaluation ofthis approach, three case compounds were selected: desmopressin,lisinopril, and fluoroquinolones. The results of the models areplotted against human paediatric data, including data in neonatesand critically ill children.A close collaboration of experts and stakeholders can help to tailorpaediatric clinical trials to the needs of children. Pharmaceuticalindustry and regulatory authorities are key players in the paediatricdrug development process. However, academia can also play animportant role in rendering the paediatric drug developmentprocess more efficient by development and correct use ofinnovative tools (figure 1). Besides, academia should defend therights of the most important stakeholders: patients and theirparents. During the SAFE-PEDRUG project additional opportunitiesfor academia have been identified: initiation of networking;centralisation in registries and networks to improve transparencyand efficiency; and education of paediatric clinical pharmacologists.DRUG DEVELOPMENT: ‘NEW’ TIMELINEADULTPAEDIATRIC PreclinicalPhase 1Phase 2Phase 3 Submission and reviewMA Post approval (Phase 4)PIP amendmentsPIP process beginsPIP compliance or deferral or waiverPaediatric studies defined in deferralPaediatricInvestigationalPlan(PIP) Paediatric studiesPIP compliance check or waiver or deferralPreclinical techniques: • Modelling & simulation• Predictive animal models• Pharmacovigilance• Clinical trial transparency• Honest post-study marketingCritical selection of compoundsFigure 1: Timelines for paediatric drug research. Opportunities forpaediatric drug research (indicated in yellow).MA: marketing authorisationREFERENCES1. European Medicines Agency. Paediatric Regulation. 2006. Available from:http://www.ema.europa.eu/ema/index.jsp?url=pages/regulation/document_listing/document_listing_000068.jsp.2. European Commission. The 2017 Commission Report on the PaediatricRegulation. Available from: https://ec.europa.eu/health/human-use/paediatric-medicines/developments/2016_pc_report_2017_en.3. Gasthuys E, Vermeulen A, Croubels S, et al. Population PharmacokineticModeling of a Desmopressin Oral Lyophilisate in Growing Piglets as aModel for the Pediatric Population. Front Pharmacol. 2018; 9:41.4. Michelet R, Dossche L, De Bruyne P, et al. Effects of Food andPharmaceutical Formulation on Desmopressin Pharmacokinetics inChildren. Clin Pharmacokinet. 2016;55(9):1159-70.

The SAFE-PEDRUG project : an opportunity for academia to close the gap

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The SAFE-PEDRUG project : an opportunity for academia to close the gap

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Ghent University Academic Bibliography