Typical PK/PD Approaches in Preclinical and Clinical Development

A development project starts with someone identifying in a patient population an unmet medical need, which may offer a business opportunity. Even when successful treatments are available for the target disease, a medical need may exist when standard treatments fail or unacceptable adverse events (AE) occur after longtime treatment. Typical for such situation is that a sound knowledge of the disease progression is available. The mechanism of action of a new drug is crucial to develop the pharmacodynamic model of the target drug action. Combining the disease model with the pharmacodynamics and the pharmacokinetics of the new drug allowed us to optimize the pivotal clinical study, confirming the efficacy in the target patient population.