New guideline on cancer medicines

EMA Highlights by Eoghan McNeill – The EMA has published revised guidelines on the evaluation of anti-cancer medicines to take account of advances in research and new therapeutic options which have emerged in recent years.

The document contains guidance on all stages of clinical drug development for treatment of cancer. Alongside conventional aims such as defining the proper doses and schedule for a medicine, the guideline highlights the importance of identifying the target population during the development.

The new recommendation, which comes into effect in July, has been updated to include advice on the use of biomarkers as an integrated part of the development of medicines; combination-therapy studies; and the choice of endpoints in confirmatory trials.

It explains how the use of biomarkers throughout non-clinical and clinical development can help identify patients with an increased likelihood to respond favourably to treatment.

According to the new guideline, confirmatory trials should demonstrate that the product under investigation provides clinical benefit. “From both a clinical and a methodological perspective, the most persuasive outcome of a clinical trial is the demonstration of favourable effects on overall survival,” the EMA said.

Prolonged progression-free survival and disease-free survival are also considered to be of benefit to the patient, the regulator says. The guideline describes how to select the appropriate endpoints for confirmatory studies.