Abstract
For a paediatric development to be rationally informed by all available knowledge, it is necessary to systematically collect and learn from available data, expert knowledge and prior developments. Aspects such as drug formulation, bioanalytical methodology, pharmacokinetics/pharmacodynamics (PK/PD), study design and statistics should be considered when defining a development plan. In this context, model informed drug discovery and development (MID3) methodology [1] is likely to prove useful.
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References
Marshall S, Burghaus R, Cosson V, Cheung A, Chenel M, Della Pasqua O, Frey N, Hamren B, Harnisch L, Ivanow F, Kerbusch T, Lippert J, Milligan P, Rohou S, Staab A, Steimer J-L, Tornoe C, Visser S (2016) Good practices in model-informed drug discovery and development (MID3): practice, application and documentation. CPT Pharmacometrics Syst Pharmacol 5(3):93–122
Guidance for industry: exposure-response relationships — study design, data analysis, and regulatory applications. [cited 2015 18 September]. Available from: http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm072109.pdf
Dunne J, Rodriguez W, Murphy MD, Beasley BN, Burckart GJ, Filie JD, Lewis LL, Sachs HC, Sheridan PH, Starke P, Yao LP (2011) Extrapolation of adult data and other data in pediatric drug-development programs. Pediatrics 128(5):e1242–e1249
ICH topic E 11 clinical investigation of medicinal products in the paediatric population. [cited 2015 18 September]. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500002926.pdf
Guideline on the role of pharmacokinetics in the development of medicinal products in the paediatric population (2006) [cited 2015 18 September]. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003066.pdf
Manolis E, Osman T, Herold R, Koenig F, Tomasi P, Vamvakas S, Saint Raymond A (2011) Role of modeling and simulation in pediatric investigation plans. Pediatr Anesth 21(3):214–221
EMA Concept paper on extrapolation of efficacy and safety in medicine development (2013) [cited 2015 18 September]. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2013/04/WC500142358.pdf
MSWG activity reports 2013. [cited 2015 18 September]. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Report/2014/06/WC500167815.pdf
MSWG activity reports 2014. [cited 2015 18 September]. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Report/2015/04/WC500185895.pdf
Sorger PK, AllerheiligenS, Abernethy DR, Altman RB, Brouwer KL, Califano A et al (2011) Quantitative and systems pharmacology in the post-genomic era: new approaches to discovering drugs and understanding therapeutic mechanisms. In: An NIH white paper by the QSP workshop group. NIH, Bethesda
EMA points to consider on applications with 1. meta-analyses; 2.one pivotal study (2001) [cited 2015 18 September]. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003657.pdf
Council NR (2012) Assessing the reliability of complex models: mathematical and statistical foundations of verification, validation, and uncertainty quantification. The National Academies Press, Washington, DC, p 131
Manolis E, Rohou S, Hemmings R, Salmonson T, Karlsson M, Milligan P (2013) The role of modeling and simulation in development and registration of medicinal products: output from the EFPIA/EMA modeling and simulation workshop. CPT Pharmacometrics Syst Pharmacol 2:1–4
ICH topic E 9 statistical principles for clinical trials. [cited 2015 18 September]. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500002928.pdf
Wang Y, Jadhav P, Lala M, Gobburu JV (2012) Clarification on precision criteria to derive sample size when designing pediatric pharmacokinetic studies. J Clin Pharmacol 52:1601–1606
Ruiz-Garcia A et al (2008) Pharmacokinetics in drug discovery. J Pharm Sci 97(2):654–690
Batchelor HK, Marriott JF (2015) Paediatric pharmacokinetics: key considerations. Br J Clin Pharmacol 79(3):395–404
Mooij M, Koninig BA, Huijsman ML, de Wildt SN (2012) Ontogeny of oral drug absorption processes in children. Expert Opin Drug Metab Toxicol 8(10):1293–1303
Edlund H, Melin J, Parra-Guillen ZP, Kloft C (2015) Pharmacokinetics and pharmacokinetic–pharmacodynamic relationships of monoclonal antibodies in children. Clin Pharmacokinet 54(1):35–80
Momper JD, Mulugeta Y, Green DJ, Karesh A, Krudys KM, Sachs HC, Yao LP, Burckart GJ (2013) Adolescent dosing and labeling since the food and drug administration amendments act of 2007. JAMA Pediatr 167(10):926–932
Guideline on the investigation of medicinal products in the term and preterm neonate (2009) [cited 2015 18 September]. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003750.pdf
Shepard T et al (2015) Physiologically based models in regulatory submissions: output from the ABPI/MHRA forum on physiologically based modeling and simulation. CPT Pharmacometrics Syst Pharmacol 4(4):221–225
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Rusten, I.S. et al. (2016). Scaling Dose-Exposure-Response from Adults to Children. In: Mahmood, I., Burckart, G. (eds) Fundamentals of Pediatric Drug Dosing. Adis, Cham. https://doi.org/10.1007/978-3-319-43754-5_7
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