EU regulation on advanced therapies

Turning research promises into reality for patients

drawing of gene therapyThe European Commission released its latest proposal on a European Regulation on Advanced Therapies in November 2005. ‘The mere fact that the Commission and the European regulatory authorities are working on a regulation goes to show that these innovative products are a real hope for patients, and in particular those affected by rare diseases’ says Fabrizia Bignami, Therapeutic Development Officer at Eurordis.

There are three kinds of advanced therapies: gene therapy, somatic cell therapy, and tissue engineering. Gene and cell therapies are currently being tested at clinical level for the treatment of specific genetic diseases, rare cancers, and other neurodegenerative disorders. Tissue engineering is a new biotechnology that combines various aspects of medicine, biology and engineering, to produce, repair or replace human tissues. It is easy to grasp the potential of these new therapies in helping to improve the quality of life of rare disease patients. However, advanced therapies are based on complex and new manufacturing processes; the expertise for the evaluation of such products is scarce for the moment; and aspects such as long-term follow-up of patients and risk management strategies for the post authorisation phase are crucial. The creation of an EU regulation is a way of addressing these complex factors by offering a framework to support advancement and to ensure patient safety.

But there is a second reason for the European Commission to propose a regulation. Gene and cell therapy products have been classified as medicinal products and are Nicolas Rossignolregulated as such in the EU. On the other hand, tissue-engineered products aren’t classified as medicinal products or medical devices; they therefore lie outside any regulatory framework. As a consequence, the way tissue-engineered products are classified and authorised varies according to Member States, which makes their circulation in the EU difficult, and ultimately could prevent patients from accessing them. ‘It also leads to market segmentation and undermines industry development,’ says Nicolas Rossignol, from DG Enterprise and Industry. The Commission therefore decided to address the problem by grouping the three kinds of advanced therapies under a single regulatory framework.

The purpose of the Regulation is to facilitate research, development and authorisation of advanced therapy products to improve patient access to them. An extensive consultation with patients, industry, hospitals, doctors, regulators, and the research community was led by the Commission. Eurordis contributed to the process by sending its observations. The key measures in the Commission’s proposal are:

  • the creation of a central marketing authorisation procedure for advanced therapy products;
  • the creation of the Committee for Advanced Therapies (CAT) within the European Medicines Agency (EMA) to provide advice in the process ; CAT will be in charge of developing criteria and guidelines for the evaluation of these products;
  • the provision of technical and risk management requirements to ensure quality and safety.

 

drawing of researchersNicolas Rossignol insists on the ethical issues linked to gene and cell therapy. It is very clear that the competence on the use or non-use of any type of cells, including the use of embryonic stem cells, for ethical reasons remains a national responsibility.’ The proposal will now be taken to the European Parliament and to the Council for adoption. ‘It will take about three years for the proposal to become law,’ says Fabrizia Bignami. ‘Advanced therapy products will be tested clinically more and more. We are definitely moving closer to seeing a new kind of medicinal product available to rare disease patients. Another very positive point is the fact that the importance of the role of patient representatives is clearly stated in the draft regulation.’

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This article was previously published in the March 2006 issue of our newsletter.

Author: Jerome Parisse-Brassens
Photos : Nicolas Rossignol © European Communities, 1997-2005
Graphics: Gene therapy © Genetics Home Reference, a service of the U.S. National Library of Medicine.
Information Published: January 22, 2006
Cartoon of researchers © European Community, 2006

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Page last updated: 16/08/2011