The European Medicines Agency has revised its guideline for the evaluation of anticancer medicinal products. The guideline offers guidance on all stages of clinical drug development for anticancer medicines and covers the use of biomarkers, combination therapy studies and the choice of endpoints in confirmatory trials.
According to EMA, a large number of anticancer compounds have been developed but very few have successfully obtained a marketing authorisation because of poor activity or inappropriate safety profiles. “Until non-clinical models with good predictive properties have been defined, this situation is likely to remain essentially unchanged,” explained the guideline, “and the absence of such models is considered the greatest hurdle for efficient drug development within the forseeable future.
Among other topics, the guideline highlights how biomarkers can help identify the target population that is most likely to benefit from the proposed compound. In addition, the guideline focuses on the importance of confirmatory trials that demonstrate a product’s clinical benefits.
Guidance is also provided for combination studies, such as combinations of drugs with minimal activity as a monotherapy, but synergistic effects when combined, as well as combinations of conventional cytotoxics.
The guideline will come into effect on 1 July 2013.
EMA’s guideline on anticancer medicinal products was first adopted in 1996, and was revised in 2001, 2003 and 2005.