De Bruyne, Pauline

Vande Walle, Johan

De Bruyne, PaulineUZGent0019992801348020011590839748707791170000-0002-3090-97550032A4F2-F0EE-11E1-A9DE-61C894A0A6B4

Vande Walle, JohanGE358010005090060000-0001-9700-5358F4129F7E-F0ED-11E1-A9DE-61C894A0A6B4

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

Ghent University Academic Bibliography

focus on the use of Angiotensin Converting Enzyme Inhibitors (ACE-I).Methods A Europe-wide web-based survey and a sub-sequent DELPHI questionnaire was developed in the con-text of EU’s Seventh Framework Programme under grant agreement n°6 02 295 using standard recommendations for survey design. The questionnaire consisted of 23 ques-tions addressing differ-ent aspects of drug therapy for HF in children. Use patterns of ACE-I i.e. dosage by age group, effectiveness and toxicity assessment according to HF ae-tiology where investigated. Clinicians from 204 different hospitals of 39 European coun-tries were invited via e-mail to participate. The subsequent DELPHI process discussed controversial responses within a selected expert panel in two rounds.Results The response rate of the survey had been 50%. The survey delivered valuable information about thcurrent paedi-atric heart failure therapy, especially with regard to the pat-tern of ACE-I use. Enalapril seems to be already the ACE-inhibitor of choice for children and ad-olescents. A suitable formulation and knowledge about dosing as well as adverse events might offer Enalapril also for neonates and infants. Several controversial aspects which were identified within the survey and which are re-lated to paediatric heart failure ther-apy had been put up for discussion to the DELPHI expert panel. They showed a high degree of consensus in their pro-fessional criteria about most of the contents presented for dis-cussion. Pos-sible starting points in the way towards a standardisation of paediatric heart failure therapy were identi-fied. With re-gard to non-consensus statements, DELPHI experts pro-vided a better visibility to some aspects of clinical practice with greater disparity of opinio Diagnostic and thera-peu-tic approachesns among physicians.Conclusion This survey and the subsequent DELPHI question-naire provided an overview of the clinical treat-ment routine of paediatric HF across Europe. ACE-I seem to be a crucial part of the treatment strategies. Consensus but also still con-troversial aspects of clinical practice rou-tines for a safe and effective use of heart failure treatment for children in Europe were identified.The research leading to these results has received fundingfrom the EU’s Seventh Framework Programme (FP7/2007-2013) under grant agreement n°6 02 295 (LENA).PP-5 PRESCRIPTION OF BIOSIMILAR SOMATROPIN IN THEITALIAN PAEDIATRIC POPULATIONClavenna, Pepino, Cartabia, Bonati. IRCCS Istituto di Ricerche Farmacologiche, Mario Negri,MILAN, Italy10.1136/archdischild-2017-esdppp.72Background Omnitrope (somatropin) was the first bi-osimilarapproved by the European Medicine Agency in 2006. Sincesomatropin is one of the biological products most commonlyprescribed to children and adolescents, a study was performedwith the aim to evaluate the pre-scription of this drug in theItalian paediatric population. To the best of our knowledge,no drug utilisation studies evaluated the prescription profile ofbiosimilars in the pe-diatric population.Methods Data collected in healthcare administrative databasesof Lombardy region, Italy, in the 2004–2012 period wereanalysed. Children and adolescents who received prescriptionsof somatropin (H01AC01code of the Anatomical TherapeuticChemical classification sys-tem) for at least two consecutiveyears were identified as prevalent cases. Subjects were definedincident cases if they had no somatropin prescriptions in theprevious 2 years. Prevalence and incidence of somatropin pre-scrip-tion were estimated by gender, age group and observa-tion year. Moreover, each youth with the first prescrip-tion(index prescription, IP) in the 2006–2010 period was moni-tored for 24 months, and somatropin prescriptions were ana-lysed to evaluate if a switch between products occurred. Inswitchers, the occurrence of specialist vis-its and/or hospitaliza-tions in the 60 days preceding the change was checked.Results During 2012, the prevalence of somatropin pre-scrip-tion in Lombardy region was 12.0 per 10,000, with an inci-dence of 2.8 per 10 000. Both prevalence and inci-denceincreased across time (from 9.6 and 1.6 per 10 000 in 2004,respectively). The prevalence was greater in boys than in girls(14 versus 10 per 10,000), and increased with increasing age(from 2.7 in pre-schoolers to 21.1 per 10 000 in adolescents).A total of 1415 children had the somatro-pin index prescrip-tion in the 2006–2010 period. Only 98 of them (7%) startedwith the biosimilar Omnitrope. The percentage of childrenstarting with the biosimilar slightly increased with increasingage, from 4.9% in the 1–5 years old to 7.5% in the adoles-cents. In all, 17 out of the 98 sub-jects (17.3%) with biosimi-lar as IP switched to another so-matropin product during the24 months after the starting date. Of the 1317 children whostarted with a ‘branded’ somatropin, 47 (3.6%) switched toanother products (no one to Omnitrope). The rate of switchwas higher in pre-school aged children (3 out of 10) anddecreased with in-creasing age (5 out of 45 in adolescents).On the contrary, the frequency of switch in subjects withother somatropin products did not change among age groups.Only 4 out of 17 subjects had a specialist visit and/or anhospitalisation in the 60 days before the switch from bio-simi-lar to ‘branded’ products, while in the non-biosimilar group, aspecialist visit and/or hospital admission was re-corded for 26out of 47 children.Conclusion Only 7% of incident (naïve) cases started with thebiosimilar somatropin. Subjects who started with the biosimilarswitched more frequently to anoth-er product and the changewas less likely preceded by a specialist visit.PP-7 THE SAFE-PEDRUG INITIATIVE: AN OPPORTUNITY FORACADEMIA TO CLOSE THE GAP1De Bruyne, 2Vande Walle, 3The SAFE-PEDRUG consortium. 1Ghent University, GHENT,Belgium; 2Ghent University Hospital, GHENT, Belgium; 3Ghent University, Ghent UniversityHospital, KULeuven and VUB, GHENT, LEUVEN AND BRUSSELS, Belgium10.1136/archdischild-2017-esdppp.73Background The Paediatric Regulation1 was launched ten yearsago. As was also identified in the ten year report2, this regula-tion has had a positive impact on paediatric research inEurope. However, some specific patient pop-ulations (such asneonates, critically ill children, children with comorbidities) donot receive enough attention in paediatric drug development.Furthermore, the top-down approach (from adults to children)results in considerable delays in making medicines available toAbstractsArch Dis Child 2017;X(Suppl X):A1–A43 A33children. For most of the drugs long term follow-up ismissing.Methods The SAFE-PEDRUG project was initiated in Belgium in2014 and is a collaboration of experts in pae-diatrics, pharma-ceutical sciences, veterinary medicine, and ethics of three Belgianuniversities: Ghent Universi-ty, KULeuven, and Vrije UniversiteitBrussel. An advisory board and stakeholder group consisting ofnational and international stakeholders support this consortiumin the valorisation of results.Results The SAFE-PEDRUG project explored the value of theporcine juvenile animal model3 and PK modelling4 (populationpharmacokinetics and physiological-ly based pharmacokineticmodelling) in providing prior paediatric PK/PD knowledge,before the actual adult trials have been completed. For theevaluation of this approach, three case compounds wereselected: des-mopressin, lisinopril, and fluoroquinolones. Theresults of the models are plotted against human paediatricdata, including data in neonates and critically ill children.Discussion A close collaboration of experts and stake-holderscan help to tailor paediatric clinical trials to the needs of chil-dren. Pharmaceutical industry and regulato-ry authorities arekey players in the paediatric drug de-velopment process. How-ever, academia can also play an important role in renderingthe paediatric drug development process more efficient bydevelopment and correct use of innovative tools. Besides, aca-demia should defend the rights of the most important stake-holders: patients and their parents. During the SAFE-PEDRUGproject additional opportunities for academia have been identi-fied: initiation of networking; centralisation in registries andnetworks to improve transparency and efficiency; and educa-tion of paediatric clinical pharmacologists.Acknowledgements The SAFE-PEDRUG project re-ceived agrant for Strategic Basic Research of the Agen-cy for Innova-tion by Science and Technology in Flanders (IWT-SBO130033).REFERENCES1. European Medicines Agency. Paediatric Regulation. 2006. Available from: http://www.ema.euro- pa.eu/ema/index.jsp?url=pages/regulation/document_ listing/docu-ment_listing_000068.jsp.2. European Com-mission. The 2017 Commission Report on the Paediatric Regula-tion. Available from: https://ec.europa.eu/health/ human-use/paediatric-medicines/developments/2016_ pc_report_2017_en.3. Gasthuys E, Vandecasteele T, De Bruyne P, Vande Walle J, De Backer P, CornillieP, et al. The Potential Use of Piglets as Human Paediatric Surrogate for PreclinicalPharmacokinetic and Pharmacodynamic Drug Testing. Curr Pharm Des. 2016;22(26):4069-85.4. Michelet R, Dossche L, De Bruyne P, Colin P, Boussery K, Vande Walle J, et al.Effects of Food and Pharmaceutical Formulation on Desmopressin Pharmacoki-netics in Children. Clin Pharmacokinet. 2016;55(9):1159-70.PP-9 PARTICIPATING IN PAEDIATRIC DRUG RESEARCH:IDENTIFYING THE BURDENDewolf, Amza, Christiaens, De Bruyne, De Cock, Vande Walle. UZ GENT, GENT, Belgium10.1136/archdischild-2017-esdppp.74Background Nowadays, academic researchers, pharma-ceuticalcompanies and regulatory authorities are more aware of theneed for paediatric drug research. Conse-quently, more aca-demic and industry-driven paediatric trials are conducted toevaluate the efficacy and safety of new drugs and to a lesserextent of off-patent and off-la-bel drugs. However little infor-mation is available on the burden associated with participatingin clinical trials for the patients and their family/caregivers. Inattempt of be-coming a Centre of Excellence in paediatricdrug research it is important for us to fully understand thisburden.Methods This is a retrospective, single centre, observa-tionalstudy. A questionnaire will be designed focusing on the overallcosts and time investment for the participants and their care-givers. Topics of interest will be absenteeism at school, atwork or in leisure; number of specific study related visits (outof standard of care); financial reward by the sponsor; etc.Additional questions will gauge the per-ception and experienceof the patients and their parents. We will contact the parentsof patients who participated in either an academic or industrydriven trial between 2010 and 2017 at the departments ofpaediatric nephrol-ogy and gastroenterology of the ElisabethChildren’s Hospital (Ghent University Hospital). We will dis-play the results of this questionnaire by using descriptivestatistics.Discussion By evaluating the results, we will identify whatbrings most burden to patients and their family/caregivers inparticipating in clinical trials. This will enable us to betterunderstand this burden and eventually to anticipate by moreand better information and support during the participation.This may increase compliance, especially important in drug tri-als. The data can help us to include these aspects in discus-sions with both ethical committee and sponsors (industry)during the develop-ment of the study design and during nego-tiation of the clinical trial agreement (inclusive of some com-pensation) between research centres and sponsors.Acknowledgements The survey is an initiative of the SAFE-PEDRUG project (IWT-SBO 130033), supported by theAgency for Innovation by Science and Technology in Flanders(IWT).PP-11 COMPARING COMPLETION RATES OF PAEDIATRICVERSUS ADULT RANDOMISED CONTROLLED TRIALS: ACROSS-SECTIONAL STUDYKaguelidou, Dufetelle. Hôpital Robert Debré, PARIS, France.10.1136/archdischild-2017-esdppp.75Background Clinical trial discontinuation represents a waste inresearch resources and raises ethical concerns. Conduct ofclinical trials is perceived to be more challeng-ing in childrenthan in adults. The aim of this study was to evaluate theimpact of the age of participants on comple-tion rates ofrandomised controlled trials (RCTs).Methods This is a cross-sectional study on RCTs regis-tered inthe ClinicalTrials.gov database. All RCTs registered in the data-base from its inception date (February 29, 2000) to December31, 2016, were extracted. RCTs with unknown recruitmentstatus or registered more than 60 days after their start datewere excluded. Remaining RCTs were classified according totheir recruitment status: active, completed, and discontinuedtrials, and according to the age of participants: children (0–17years), adults (18 years), and mixed age populations. FurtherRCT charac-teristics were assessed using information registeredin the database: study location, funding source, year of reg-istration, study phase, study design, type of intervention eval-uated, blinding procedure, study duration, and enroll-mentachieved. A logistic regression model was applied to assess theimpact of participant’s age category on trial completion whilecontrolling for other potentially rele-vant trial characteristics.AbstractsA34 Arch Dis Child 2017;X(Suppl X):A1–A43

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

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