Pharma welcomes EU regulation on advanced therapies

The pharma sector has welcomed the new EU Regulation on advanced therapies due to come into force at the end of the year, although companies have questions as to how it will operate in detail.

The Regulation has proved problematic to draft up since it touches on ethical aspects, particularly concerning the use of embryonic stem cells. It lays down a legislative framework for treatments based on genes, cells or tissues. Adopted on 13 November 2007, it is due for application on 30 December this year.

The Regulation provides for common rules for advanced therapy products and a centralised approval procedure, along with the setting up of a specialised committee within the European Medicines Agency (EMEA) that will examine marketing authorisation requests.

However, the industry has identified several points in the Regulation over which it is not clear.

The first of these is the scope of the Regulation. There are exceptions provided for various products, and particularly for those that are not mass produced. Other exceptions relate to products used only in hospital in a Member State or custom-made products for individual patients. But many products are developed in a university institute or hospital.

The evaluation process represents another stumbling block, since it will involve two EMEA committees, the CHMP and the advanced therapy committee that has yet to be formed. But the pharma industry would prefer to deal with just one committee rather than being faced with two lists of questions from two different committees.

The Regulation also provides for a 90% reduction in fees for small and medium enterprises (SMEs) requesting a scientific opinion from EMEA while other parties will be granted a 65% reduction. However, where early product development is concerned, the scientific opinion process, which is not centralised, is long and costly, particularly for small companies.

And apart from the core documents, clinical study applications are also not centralised or harmonised.

The financial advantages foreseen by the Regulation are limited, since most of the products will have orphan drug status - a status that implies financial incentives and the possibility of commercial exclusivity.

Apart from reductions applicable to scientific opinions, the text provides for the marketing authorisation fee to be cut by 50% where the requestor is a hospital or a small or medium company and proves that the drug concerned has particular public health value.

Questions have also been raised about how the Member State representatives would be selected to sit on the advanced therapy committee, whether the committee would develop "guidance" for products at the early development stage and whether there was a chance of improved harmonisation among national agencies.

Patrick Celis, Medicines Agency Scientific Administrator indicated that EMEA is currently setting up the Advanced Therapy committee and drafting the rules of procedure, particularly concerning interaction between this committee and the CHMP. The next stage will involve appointing the advanced therapy committee members and then its chairman; the committee will sit for its first meeting in January 2009.

The advanced therapy committee will comprise five members from the CHMP and one member from each Member state along with doctor and patient representatives.

Celis indicated that EMEA is also drafting guidelines, due to be finalised in June, to cover tissue-engineered products. So far, there has not been any guidance established for this category of products.