BioWorld International Correspondent

BRUSSELS, Belgium - The European Medicines Agency is planning tougher rules for authorization of gene therapy medicines. A just-published draft guideline sets out scientific principles and methodology to be used for the environmental risk assessment of GMO-containing gene therapy medicinal products.

The move is in part a response to the current chaotic regulatory situation in Europe. Under European Union rules, national authorities are responsible for regulating investigational medicinal products consisting of GMOs, but "National law and the application of directives for clinical trials differ in the EU member states," the Agency admits.

What the agency wants to impose, therefore, is a standardized approach across the EU. The environmental risk assessment should cover, it said, potential adverse effects for anyone directly exposed to the GMO - including staff in clinics, family members, and the general public. It should also evaluate possible adverse effects for animals, plants and micro-organisms.

For genetically modified cells, the assessment should identify risks with recombinant viruses administered with or contained within the cells, or potentially resulting from mobilization or recombination after administration to the patient. It also should, if necessary, define measures to reduce the risks to acceptable levels.

The agency also is working with the pharmaceutical industry to refine European thinking on the potential for using biomarkers. According to professor Bruno Flamion, who chairs the agency's scientific advice working party, industry requests for agency advice on biomarkers have risen from 58 procedures in 2000 to 261 procedures in 2006.

A recent agency workshop delivered a positive appraisal on the progress observed in drug development when using predictive biomarkers to optimize the understanding of the potential impact of the drug in a given indication and the design of the trials, so that targeted therapies could be delivered - particularly in oncology.

But the agency said that more fundamental scientific and clinical knowledge is required for the qualification of biomarkers as surrogate endpoints. It is urging scientists from academia and from industry to share with regulators advances in this direction, to ensure a timely uptake of innovation in this area.

Meanwhile, European biotech companies have been putting their views to EU officials on the direction EU health policy should take. They have stressed the need for the biotech industry to have an opportunity to contribute to all EU-led efforts to improve information to patients on the grounds that real knowledge about the most advanced therapies is often available only within biotech companies, due to the novelty and complexity of approaches to disease management.

They also want more discussion on the scope for disease prevention. Diagnostics will become increasingly important as it becomes possible to move from a phenotype to a genotype approach to disease management, companies are suggesting to policy-makers.