At the time of marketing authorization, many questions regarding the optimal use of new cancer drugs remain unanswered. This concerns, for example, the appropriate dosage for different patient groups and treatment duration, but also the comparison of the available treatment options with each other, or the optimal sequence. Because of these gaps in knowledge, the best possible results for patients are often not achieved. This problem has become more acute in recent years because efforts have focused more on accelerating the marketing authorization of new drugs than on optimizing their use in everyday clinical practice.
The publication summarizes the results of a workshop held by the Cancer Medicines Forum at the European regulatory authority European Medicines Agency (EMA), in which IQWiG also participated. The article highlights the potential of studies in the optimization of cancer treatment and describes how these studies can generate the evidence needed for cancer treatment decisions, both during the development of new drugs and after marketing authorization has been granted. Last but not least, the publication contains a roadmap for the systematic implementation of treatment optimization studies in drug development and in a knowledge-generating health care system.