European regulators prepare for advanced therapies

Friday 5 December 2008
James Mills - Deputy Editor

Early and continued dialogue between the European Medicines Agency and sponsors of marketing authorisation applications is urged when they involve complex tissue-engineered products or therapies using gene and cell-based technologies.

EU legislation on advanced therapies is due to take effect at the end of the year and the EMEA's new Committee on Advanced Therapies (CAT) plans to hold its first meeting on January 15th-16th. Some of the guidelines from the agency and the European Commission that will be used to implement the legislation are still being tweaked, which both sponsors and regulators acknowledge is a necessary step in the development and oversight of bringing new products to market. Many of the guidelines are already in place, although evolving science necessitates constant review.

While sponsors and regulators negotiate the shape of acceptable and appropriate tests for advanced therapies case by case, both sides are emphasising the importance of frequent and transparent communication.

A joint meeting of The Organisation for Professionals in Regulatory Affairs (TOPRA) and the EMEA in London on December 2nd-3rd devoted a session to advanced therapies where it became clear that work is very much in progress on both sides of the regulatory divide.

Duncan Mackay of Genzyme said the industry had "lobbied hard and lobbied long for this legislation", referring to Regulation (EC) No 1394/2007, but he added that it had been a "long process to educate regulators". He pointed out that advanced therapies were generally used in small patient populations after other treatments had failed and asked whether regulators should be bolder with conditional approvals for advanced therapies. He insisted that regulation of advanced therapies should be proportionate to the risk involved.

Wilfried Dalemans of Tigenix also recommended a pragmatic risk/benefit approach to the regulation of advanced therapy products, describing the experience of a small company. He praised legislative provisions that allow small and medium-sized enterprises to have advanced therapy products certified by the EMEA before the agency conducts a formal review for marketing authorisation. Certification can reassure investors and add value to a small company even before it can bring a product to market, he said. The certification process is not open to large pharmaceutical companies.

Regulators and sponsors at the meeting both emphasised the importance of dialogue during the licensing process. The EMEA's conditions on formal scientific advice on marketing applications for therapeutic chemicals and less complex biological products, and the associated costs, are less rigorously applied at the forefront of biomedical science.

The EMEA plans to hold its first workshop for sponsors and potential sponsors of marketing authorisation applications for advanced therapy products in London on February 3rd.

• Not a subscriber? Request a FREE trial
• Subscribe to Scrip
• Email to a friend

• Facebook
• digg
• del.icio.us
• furl