This Regulation lays down specific rules concerning the authorisation, supervision and pharmacovigilance of advanced therapy medicinal products.
1.In addition to the definitions laid down in Article 1 of Directive 2001/83/EC and in Article 3, points (a) to (l) and (o) to (q) of Directive 2004/23/EC, the following definitions shall apply for the purposes of this Regulation:
‘Advanced therapy medicinal product’ means any of the following medicinal products for human use:
a gene therapy medicinal product as defined in Part IV of Annex I to Directive 2001/83/EC,
a somatic cell therapy medicinal product as defined in Part IV of Annex I to Directive 2001/83/EC,
a tissue engineered product as defined in point (b).
‘Tissue engineered product’ means a product that:
contains or consists of engineered cells or tissues, and
is presented as having properties for, or is used in or administered to human beings with a view to regenerating, repairing or replacing a human tissue.
A tissue engineered product may contain cells or tissues of human or animal origin, or both. The cells or tissues may be viable or non-viable. It may also contain additional substances, such as cellular products, bio-molecules, bio-materials, chemical substances, scaffolds or matrices.
Products containing or consisting exclusively of non-viable human or animal cells and/or tissues, which do not contain any viable cells or tissues and which do not act principally by pharmacological, immunological or metabolic action, shall be excluded from this definition.
Cells or tissues shall be considered ‘engineered’ if they fulfil at least one of the following conditions:
the cells or tissues have been subject to substantial manipulation, so that biological characteristics, physiological functions or structural properties relevant for the intended regeneration, repair or replacement are achieved. The manipulations listed in Annex I, in particular, shall not be considered as substantial manipulations,
the cells or tissues are not intended to be used for the same essential function or functions in the recipient as in the donor.
‘Combined advanced therapy medicinal product’ means an advanced therapy medicinal product that fulfils the following conditions:
it must incorporate, as an integral part of the product, one or more medical devices within the meaning of Article 1(2)(a) of Directive 93/42/EEC or one or more active implantable medical devices within the meaning of Article 1(2)(c) of Directive 90/385/EEC, and
its cellular or tissue part must contain viable cells or tissues, or
its cellular or tissue part containing non-viable cells or tissues must be liable to act upon the human body with action that can be considered as primary to that of the devices referred to.
2.Where a product contains viable cells or tissues, the pharmacological, immunological or metabolic action of those cells or tissues shall be considered as the principal mode of action of the product.
3.An advanced therapy medicinal product containing both autologous (emanating from the patient himself) and allogeneic (coming from another human being) cells or tissues shall be considered to be for allogeneic use.
4.A product which may fall within the definition of a tissue engineered product and within the definition of a somatic cell therapy medicinal product shall be considered as a tissue engineered product.
5.A product which may fall within the definition of:
a somatic cell therapy medicinal product or a tissue engineered product, and
a gene therapy medicinal product,
shall be considered as a gene therapy medicinal product.