Background

Appropriately designed clinical trials can provide the evidence needed to broadly implement therapeutic drug monitoring (TDM). In the past 30 years, some stunning successes but also some fascinating failures in demonstrating the benefits of TDM have been observed. Future TDM studies can be designed based on this experience.

Methods

The manuscript is based on a combination of personal experience and published articles and discusses several aspects of the design and conduct of TDM studies.

Results

Recommendations are provided to reduce the risk of protocol violations and to maximize the potential impact of a TDM study on clinical practice.

Conclusions

There are lessons that can be learned from previous experience, and this article gives an overview of potential TDM study designs, endpoints, pitfalls, and perspectives.