Medical Device Daily Contributing Writer
And MDDs

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 is a standardized approach across the EU. The environmental risk assessment should cover, it says, potential adverse effects for anyone directly exposed to the GMO — including staff in clinics, family members and the public.

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.

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 grown 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 life sciences companies have been putting their views to EU officials on the direction that 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 policymakers.

TOPS-on-Fusion gets CE mark

Impliant (Princeton, New Jersey), a developer of spine arthroplasty alternatives to fusion surgery, said that its Impliant subsidiary has received CE-mark approval for its TOPS-on-Fusion System.

The company said the system is the world's first total posterior motion preservation device that integrates lumbar fusion with total posterior arthroplasty at two adjacent levels to treat spinal stenosis, with or without facet arthrosis and spondylolisthesis.

"In clinical trials in South Africa, Belgium and Turkey, the TOPS-on-Fusion System has demonstrated effectiveness in alleviating pain stemming from degeneration of the posterior elements," said Marcus Klarl, European VP for clinical affairs. "Surgical intervention is delayed for as long as possible in favor of conservative treatment. By the time many patients reach the operating room, their spine disease has advanced to two levels."

The procedures have been performed by Dr. Louis Nel in South Africa, Dr. Azmi Hamzaoglu in Turkey and Drs. Bart Conix and Robert Hes in Belgium.

Conix and Hes said, "It is common to have patients with advanced disc and posterior disease at one level and isolated posterior problems at the adjacent level. The TOPS-on-Fusion System stabilizes both segments while allowing motion at the level adjacent to fusion."

The system is the second in a family of products from Impliant that provide surgeons with a range of alternatives to traditional spinal fusion. The device is indicated for L3-L5 and L4-S1 patients where the inferior segment is fused and the superior level is treated with total posterior arthroplasty.