EMA releases new guidance on assessing anti-cancer medicines

The European Medicines Agency (EMA) has released new guidance for pharmaceutical companies on evaluating anti-cancer medicines.

Its new advice includes details on confirmatory trials, saying they should show how medicines could both destroy or halt the growth of cancers. More detailed guidance is included on using ‘cancer progression-free survival’ in confirmatory trials and specific advice on treating non-small-cell lung cancer, prostate cancer, chronic myeloid leukaemia, and myelodysplastic syndromes, as well as haematopoietic stem cell transplantation. EMA also advises on the choice of endpoints for confirmatory trials.

The guidance also focuses on using biomarkers within non-clinical and clinical development to identify patients more likely to respond favourably to treatment. It notes that the importance of biomarkers extends beyond setting doses and treatment schedules to identifying target markets during medicine development. As a result, ‘the development of biomarker diagnostic methods should be considered early in clinical development’.

Another topic is combination therapy studies, where EMA accepts that there are limitations in making assessments based on the known individual toxicity of compounds involved in treatment. While regulators and academics have accepted this approach, EMA stressed: ‘It is acknowledged that it is frequently unknown whether combined use results in a better long-term outcome than consecutive use’ of treatments.