In a workshop of the Cancer Medicines Forum at the European Medicines Agency (EMA), an international panel of experts has identified ways in which studies to optimise cancer treatments can improve the evidence situation after the approval of new drugs. The results were published in the Journal of Cancer Policy and contain a catalogue of measures to strengthen the anchoring of such studies.

When it comes to the approval of new cancer drugs, central questions often remain unanswered. These include the optimal dosage in different patient groups, the duration of treatment, the comparison with other therapy options and the best sequence of treatments. These gaps in knowledge mean that patients do not always receive the best possible therapy. The problem has been exacerbated in recent years as there has been a greater focus on accelerated approval than on optimizing clinical use.

The publication summarises the findings of the workshop, in which the Institute for Quality and Efficiency in Health Care (IQWiG) was also involved. It describes how therapy optimization studies can provide evidence for informed treatment decisions both in the development phase and after the market launch. Such studies could clarify open questions about dosage, duration and combinations and increase the benefit for patients.

The proposed catalogue of measures aims to systematically integrate therapy optimisation studies into drug development and knowledge-generating care. This is to ensure that new cancer drugs are not only approved quickly, but also used optimally.

The work underlines the need for a stronger focus on everyday clinical practice already in early development phases and after approval. It offers regulatory authorities, manufacturers and healthcare systems concrete approaches to improve the quality of care for oncological diseases.

The publication was published in the Journal of Cancer Policy (DOI: 10.1016/j.jcpo.2025.100544).

Original Paper:

Accelerating cancer treatment optimisation: A multistakeholder roadmap from the Cancer Medicines Forum – ScienceDirect

Editor: X-Press Journalistenbüro GbR

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