Draft Guidance Published By EMA on Human Clinical Trials for Cancer Drugs

The European Medicines Agency (EMA) has published new draft guidance on the evaluation of anticancer medicines in humans that covers all stages of clinical development.

A very large number of anti-cancer compounds are being developed however only a minority have obtained marketing authorization “due to poor activity or evidence of a detrimental safety profile. Until non-clinical models with good predictive properties have been defined, this situation is likely to remain essentially unchanged and the absence of such models is considered to constitute the greatest hurdle for efficient drug development within the foreseeable future.

The guidance specifically encourages the development of molecule-specific preclinical models to assess and predict anticipated activity as well as safety. The aim of the guidance is to classify compounds according to reasonable designs of exploratory studies, “i.e. cytotoxic compounds where toxicity and ORR (overall response rate) are considered suitable markers of activity in dose finding studies vs. non-cytotoxic compounds where ORR and/or toxicity may not serve this purpose.

Guidance is also provided on studies for combinations of drugs with minimal activity as monotherapies.  Overall the purpose of the guideline is to provide guidance on all stages of clinical drug development for the treatment of malignancies, including drug resistance modifiers or normal tissue protective compounds.



Posted by Dr. Tim Sandle