Relevance of PK and PKPD modelling in rare diseases

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.