This procedure, now compulsory for high-technology medicinal products (gene therapy and associated cell therapies, xenogenic therapies) in order to maintain the high level of scientific evaluation and thus to preserve the confidence of medical professionals and patients, will also become compulsory for orphan medicinal products; for products containing a new active substance and indicated for the treatment acquired immune deficiency syndrome, cancer, neurodegenerative disorder or diabetes and, four years after the entry into force of the Regulation, also for the treatment of auto-immune diseases, immune dysfunctions and viral diseases.

The level of scientific rigour that is normally applied and legally required under the Canadian Food and Drugs Act and its regulations in the development of potential medical treatments would have to be entirely ignored for experiments with human embryos to proceed (1210) As the largely disappointing experience with gene therapy should remind us, many highly vaunted, scientific techniques frequently failed to yield the promised results.

Mr Sven Bostyn from Maastricht University was elected reporter for the first topic which deals with the level of protection to be given to patents of sequences or partial-sequences of genes isolated from the human body.

Some first steps towards harmonisation of regulatory requirements have been taken, for instance where gene and cell therapy is concerned, since the European Agency for the Evaluation of Medicinal Products has drawn up guidelines for good practice. However, there is still no frame of reference and system of rules to govern new fields such as tissue engineering, artificial organs, and genetic testing.

In order to determine the extent and type of non-clinical studies necessary to determine the appropriate level of non-clinical safety data, the design and type of the gene therapy medicinal product shall be taken into account.

Risk factors that may be considered include: the origin of the cells (autologous, allogeneic, xenogeneic), the ability to proliferate and/or differentiate and to initiate an immune response, the level of cell manipulation, the combination of cells with bioactive molecules or structural materials, the nature of the gene therapy medicinal products, the extent of replication competence of viruses or micro-organisms used in vivo, the level of integration of nucleic acids sequences or genes into the genome, the long time functionality, the risk of oncogenicity and the mode of administration or use.