Relevance of PK and PKPD modelling in rare diseases

Article type: Research Article

Authors: Della Pasqua, Oscar^; | Felisi, Mariagrazia

Affiliations: Division of Pharmacology, Leiden/Amsterdam Center for Drug Research, The Netherlands | Clinical Pharmacology and Discovery Medicine, GlaxoSmithKline, UK | Consorzio per Valutazioni Biologiche e Farmacologiche, Pavia, Italy

Note: [] Corresponding author. E-mail: [email protected]

Abstract: Evidence from a limited population may be inappropriate to characterise exposure-response relationships and subsequently define dosing recommendations for a wider target group. This limitation is particularly important if standard trial designs and data analysis methods are used to assess pharmacokinetics and pharmacodynamics. Models are a theoretical representation of a phenomenon. According to this definition, PKPD models can be applied to the development of new compounds, enabling inferences about efficacy and safety, which cannot be obtained otherwise.

Keywords: Pharmacokinetics, pharmacodynamics, modelling, Bayesian

DOI: 10.3233/PPL-2010-0271

Journal: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 73-74, 2010