Regulation of Advanced Therapies

Definitions of Advanced Therapy categories.

2.1. Gene therapy medicinal product
Gene therapy medicinal product means a biological medicinal product which has the following characteristics:

(a) it contains an active substance which contains or consists of a recombinant nucleic acid used in or administered to human beings with a view to regulating, repairing, replacing, adding or deleting a genetic sequence;
(b) its therapeutic, prophylactic or diagnostic effect relates directly to the recombinant nucleic acid sequence it contains, or to the product of genetic expression of this sequence.

Gene therapy medicinal products shall not include vaccines against infectious diseases.

2.2. Somatic cell therapy medicinal product
Somatic cell therapy medicinal product means a biological medicinal product which has the following characteristics:

(a) contains or consists of cells or tissues that have been subject to substantial manipulation so that biological characteristics, physiological functions or structural properties relevant for the intended clinical use have been altered, or of cells or tissues that are not intended to be used for the same essential function(s) in the recipient and the donor;
(b) is presented as having properties for, or is used in or administered to human beings with a view to treating, preventing or diagnosing a disease through the pharmacological, immunological or metabolic action of its cells or tissues.

For the purposes of point (a), the manipulations listed in Annex I to Regulation (EC) No 1394/2007, in particular, shall not be considered as substantial manipulations.

1.  (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 nonviable. It may also contain additional substances, such as cellular products, biomolecules, biomaterials, chemical substances, scaffolds or matrices.

Products containing or consisting exclusively of nonviable 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.

(c) Cells or tissues shall be considered ‘engineered’ if they fulfill 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.

ANNEX I:
Manipulations referred to in the first indent of Article 2(1)(c)
cutting, grinding, shaping, centrifugation, soaking in antibiotic or antimicrobial solutions, sterilization, irradiation, cell separation, concentration or purification, filtering, lyophilization, freezing, cryopreservation, vitrification.

(d) ‘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 [Medical Device Directive] or one or more active implantable medical devices within the meaning of Article 1(2)(c) of Directive 90/385/EEC [Active Implantable Medical Device Directive], and
• its cellular or tissue part must contain viable cells or tissues,or
• its cellular or tissue part containing nonviable 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.