Brussels, 5 July 2006
OPINION of the European Economic and Social Committee on the Proposal for a Regulation of the European Parliament and of the Council on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/2004 COM(2005) 567 final - 2005/02 (COD)
Full text of Opinion in MS Word file on ESC website
An article by article examination of the regulation raises a number of comments, questions and recommendations. With regard to Article 2: "Definitions":
The definitions concerning gene therapy and somatic cell therapy do not generally pose any problems given that reflection and experience have led to a consensus. These products are classed as medicines and are already regulated as such within the Community.
The definition of a tissue engineered product seems more complex however. As currently worded, the first indent of Article 2(1)(b) states that a tissue engineered product "contains or consists of engineered cells or tissues", without specifying "as an integral part". In practice, therefore, medical devices which contain tissue engineered products "with an ancillary function" are also included among innovative medicinal products. This makes the provisions of the proposed directive on medical devices meaningless.
The wording of the second indent of Article 2(1)(b) could also give rise to implementing difficulties and, in particular, overlap with the medical device directive. As tissue engineered products are covered by legislation on medicinal products, it would be desirable to mention their primary activity of disease treatment or prevention, or of altering physiological functions through pharmacological, immunological or metabolic action, rather than just referring to their properties for "regenerating, repairing or replacing a human tissue", as these properties are also shared by some types of medical device.
An effort has been made to narrow down the exact definition of a "tissue engineered product" as much as possible. Nevertheless, the difference from cell therapy (bone marrow transplants, stem cell transplants, umbilical cord blood transplants, adult or embryonic stem cells, etc.) is not entirely clear.
The Committee proposes that examples of products currently considered to have been generated by tissue engineering be used as a starting point for attempts to clarify the definition. This would assist understanding, particularly since it is no secret that the subject is the focus of debate and controversy, particularly regarding embryonic stem cells.
At this point, there are no ethical problems apart from those surrounding human embryonic stem cells (HESC).
The controversy centres on the means of producing stem cells. More specifically, the production of these cells by nuclear transfer (in other words cloning) raises major ethical questions, and no real consensus has been found within the European Union to this day. Current concerns focus on the risks of reproductive cloning, egg trafficking and the commercialisation of human body parts.
Such practices are explicitly condemned by the European Convention on Bioethics (Oviedo Convention, 1998) and by the International Bioethics Committee (UNESCO, 1997).
In the absence of a consensus between EU Member States, HESC use falls within national responsibility.
The detail given in the recitals is therefore essential, as it gives clear consideration to the reality of the debate and states that this text regulating advanced therapy medicinal products at Community level is not designed to "interfere with decisions made by Member States on whether to allow the use of any specific type of human cells, such as embryonic stem cells, or animal cells".
Neither is it designed to "affect the application of national legislation prohibiting or restricting the sale, supply or use of medicinal products containing, consisting of or derived from these cells".
On the whole, this draft regulation is relevant and useful. It provides a means of keeping up with scientific developments and deciding on definitions and the conditions for using advanced therapy medicinal products, thus serving patients' interests.
These new technologies offer patients great hopes in terms of overcoming human suffering. However, if they are to respond to legitimate expectations, especially in the field of regenerative medicine, research must be supervised using essential tests, and the protocols for these must offer an absolute guarantee of patient safety. With this aim in view, the main objectives set out in the Justification of the Commission's proposal for a Regulation (point 2.1) should be to guarantee not just a high level of health protection but also to give a guarantee of medicinal quality assurance. The issue of non-used waste - a rarely-discussed environmental aspect - must also not be overlooked.
The regulation is important, especially in the realms of gene therapy and somatic cell therapy. The caution exercised with regard to both definitions and the use of the products of tissue engineering clearly shows that the draft regulation is not intended to settle the debate once and for all or to pre-empt the deliberations of each Member State, since the ethical debate is not yet resolved and since it depends on varying interpretations of humanist values.
This draft regulation creates the preconditions for closing the regulatory gap that exists between its subject matter and the draft directive on medical devices. The general principle of risk evaluation applies to both advanced therapy medicinal products and medical devices. Complications may arise with combination products (i.e. medical devices containing tissue engineered elements). In such cases, both quality and safety must be guaranteed, and the evaluation must also cover the efficacy in use of an innovative medicinal product in a specific medical device.
In conclusion, the Committee endorses the proposed regulation while also stressing certain areas of concern for which clear solutions will be needed in order for the directive to be implemented successfully.