A Novel Modelling and Simulation Approach to Estimate the Efficacy of a Drug
Implications for drug development and registration
To register a new drug to manage renal or cardiovascular complications of diabetes, drugs are developed and targeted to normalize a single risk factor such as blood pressure. By targeting this single risk factor it is the intention to reduce long term renal or cardiovascular risk. However, in recent years, a number of large clinical trials in diabetes reported no such long term benefit despite the drugs tested in these trials improved the single target risk factor.
An explanation for these unexpected findings may be that drugs do not only change the on-target risk marker but also affect so called off-target risk markers. The effect on these off-target risk markers can have either beneficial or adverse effects on long-term renal or cardiovascular outcomes. It would therefore seem appropriate to incorporate the effect of a drug on multiple risk factors into a risk algorithm to obtain a more accurate estimate of the drug effect on ultimate renal or cardiovascular outcomes rather than using changes in single risk markers to establish drug efficacy.
In the presentation a novel simulation and modelling approach how to estimate a drug’s efficacy will be illustrated taking into account the multiple effects of a single drug and the implications of this new approach for drug development and registration will be discussed.
Presented by: HJ Lambers Heerspink