Development of recombinant DNA technology has induced in the public fears and speculation regarding potential risks. In fact, implementation of gene therapy involves technological approaches which might not be devoid of potential side effects (as with many conventional therapeutic means addressing severe conditions).
Regulation of Gene Therapy is intended to assess for potential risks in order to restrict those; but also to delineate a margin where safety is being secured. This should contribute to better understanding and improved public acceptance. Safety and regulatory aspects of gene therapy can be envisaged along three lines: 1st/ experimental and pre-clinical research; 2nd/ Manufacturing of gene-therapy products; 3rd/ Clinical trials and development.
Developments of Gene Therapy approaches requires to consider already existing regulation. This conditions further need for additional regulation. In any case, this evolving field will require adaptation to evolving knowledge. What would more specifically belong to gene therapy is the level of complexity resulting from intrication of the various critical levels of concerns; since gene therapy conjugates basic science, such as gene regulation and gene-delivery systems, with manufacture of biotech products and finally, potential evolution inside the body of a patient following therapeutic intervention.
In order to harmonize positions within EEC, three Directives were adopted by the Council releasing regulation applying to the use of Genetically Modified Organisms. Difficulties have been encountered. In some countries implementation under the form of National Laws is still pending while revision of the European Directives is currently in process. Consensus seems difficult to reach: in fact, the directives are flexible enough so that each national Authority may interpret them in a way that reflects the existing national procedure. This generates heterogeneity.
Current heterogeneity justifies to establish a survey of National Regulations as well as to circulate to potential users expert information related to European Regulation. This should ultimately contribute to harmonization via dissemination of knowledge and expertise. A series of tasks will be undertaken toward this end including: 1-/ Record of Regulatory Information from individual Members States; 2-/ Translation (in a language understandable Europe wide) towards Circulation; 3-/ Circulation of Information including both national regulation and overall record by means of printed reports, Telematics (Internet) and potential support of two Europe-based journals specialized in the field; 4-/ Record of Regulatory Information from European Union relevant to the field of Gene Transfer and Therapy in Human; 5-/ Establishment of a booklet of recommendations intended for users towards usage of European Regulation; updated whenever appropriate; 6-/ Bioethics: 6-1-/ Survey of Opinions released by the Group of Advisers on the Ethical Implications of Biotechnology of the European Commission and 6-2-/ Report on Declarations of the International Bioethics Committee (Unesco); 7-/ Interaction with: the European Commission/DGXII/ DGIII/ EMEA, OECD Biotechnology Unit, ISHAGE and finally Industrial Platforms.
This programme involves the contribution of expertised users representing several countries (11) among which less-favoured ones inside the European Union. Subsidiarity in Scientific Expertise is taken care of. In addition, some partners have already conducted Clinical Trials in their countries; thus experiencing Regulation of Gene Therapy as 'Users'. Dissemination of knowledge and reports on ongoing progresses will be ascertained. We will address extended audience taking advantage of identified networks and suitable contacts with: Professional associations (collaboration with EWGT, in particular), Regulatory bodies, Bioethics groups, Public Authorities and Services, as well as Industrial Platforms. Our purpose is to provide Users with relevant information and contribute to harmonization.