Advanced therapy medicinal products
This Regulation seeks to lay down rules concerning the authorisation, supervision and pharmacovigilance of advanced therapy medicinal products which are prepared industrially or manufactured by a method involving an industrial process and are intended to be placed on the market in Member States.
Regulation (EC) No 1394/2007 of the European Parliament and of the Council of 13 November 2007 on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/2004 [See amending act(s)].
Advanced therapies, such as gene therapy, somatic cell therapy and tissue engineering, offer new opportunities for the treatment of diseases of the human body. Because of the novelty and complexity of advanced therapy medicinal products *, they need to be regulated by appropriate legislation in order to safeguard public health.
This Regulation therefore lays down rules governing advanced therapy medicinal products which are prepared industrially or manufactured by a method involving an industrial process and are intended to be placed on the market in Member States. In addition, it amends certain provisions of Directive 2001/83/EC and Regulation (EC) No 726/2004.
Advanced therapy medicinal products must meet certain conditions before they can be placed on the market. They must obtain a marketing authorisation, to be issued by the Commission after scientific evaluation of the application by the European Medicines Agency (hereinafter referred to as the Agency).
This marketing authorisation is valid throughout the European Union, without prejudice to national ethical legislation prohibiting or restricting the use of any specific type of human or animal cells or the sale, supply or use of medicinal products containing, consisting of or derived from these cells.
This Regulation creates within the Agency a Committee for Advanced Therapies, responsible for preparing draft opinions on the quality, safety and efficacy of advanced therapy medicinal products. This Committee is to be consulted by the Agency’s Committee for Medicinal Products for Human Use regarding scientific evaluations of advanced therapy medicinal products. The Executive Director of the Agency ensures coordination between the activities of the Committee for Advanced Therapies and those of the other Committees of the Agency, in particular:
- the Committee for Medicinal Products for Human Use;
- the Pharmacovigilance Risk Assessment Committee;
- the Committee for Orphan Medicinal Products.
The donation, procurement and testing of human tissues and cells contained in certain of these medicinal products must follow the principles laid down in Directive 2004/23/EC.
Applications for the authorisation of advanced therapy medicinal products containing medical devices, bio-materials or matrices must include a description of the physical characteristics and performance of the product and a description of the product design methods.
The requirements relating to medical devices laid down in Directive 93/42/EEC apply to medical devices used in combined advanced therapy medicinal products *. Active implantable medical devices used in combined advanced therapy medicinal products must comply with the requirements imposed on them in Directive 90/385/EEC.
The Agency must evaluate a combined advanced therapy medicinal product as a whole product. The results of the assessment of the medical device part or the active implantable medical device part by a notified body shall be recognised by the Agency in its evaluation of the medicinal product.
The application for a marketing authorisation for a combined advanced therapy medicinal product shall include, where available, the results of the assessment by a notified body. If the application does not include the results of the assessment, the Agency may seek an opinion from a notified body on the conformity of the device, unless the Committee for Advanced Therapies decides otherwise.
After consulting the Agency, the Commission establishes guidelines on good manufacturing practice and guidelines on the application of good clinical practice for advanced therapy medicinal products.
Summary of product characteristics, labelling and package leaflet
The summary of the product characteristics for advanced therapy medicinal products must contain the name of the medicinal product, the composition of the product, its pharmaceutical form, clinical particulars, pharmacological properties, quality particulars, the name of the marketing authorisation holder, the marketing authorisation number and the date of the first authorisation or renewal of the authorisation (Annex II of this Regulation).
The labelling of the outer/immediate packaging of an advanced therapy medicinal product must contain information such as the name of the medicinal product, an indication of whom it is intended for (children or adults), a description of the active substance(s) expressed qualitatively and quantitatively, the pharmaceutical form, a list of excipients, the method of use, application, administration or implantation, the expiry date, the name and address of the marketing authorisation holder, the unique donation and product codes, and, in the case of advanced therapy medicinal products for autologous use *, the unique patient identifier and the statement "For autologous use only" (Annex III of this Regulation).
The package leaflet for an advanced therapy medicinal product must match the product characteristics summary. It must include the identification of the advanced therapy medicinal product (name, therapeutic group, etc.), the therapeutic indications, the information which is necessary before the medicinal product is taken or used, the necessary instructions for proper use, a description of the adverse reactions which may occur and a reference to the expiry date (Annex IV of this Regulation).
The measures envisaged to ensure the follow-up of efficacy of advanced therapy medicinal products and of adverse reactions thereto must be detailed in the application for authorisation.
If necessary, the Commission may require the setting up of a risk management system designed to identify, prevent or minimise risks related to advanced therapy medicinal products, including an evaluation of the effectiveness of that system.
In addition, the Agency may request submission of additional reports evaluating the effectiveness of the risk management system. The Agency shall also inform the Commission if it finds that the marketing authorisation holder has failed to comply with the requirements to set up such a system.
The Agency shall inform the relevant national competent authorities if serious adverse events or reactions occur in relation to a combined advanced therapy medicinal product.
The holder of the marketing authorisation must establish a system for the traceability of the individual product and its starting and raw materials.
The hospital or institution where the advanced therapy medicinal product is used must establish a system for patient and product traceability.
The applicant or holder of a marketing authorisation may request advice from the Agency on pharmacovigilance and on the risk management system.
This Regulation shall apply from 30 December 2008. The Commission shall present a report on its application by no later than 30 December 2012.
Tissue engineered products * which were legally on the market on 30 December 2008 shall comply with this Regulation by no later than 30 December 2012. Other advanced therapy medicinal products shall comply by no later than 30 December 2011.
|Key terms of the act|
|Act||Entry into force||Deadline for transposition in the Member States||Official Journal|
Regulation (EC) No 1394/2007
OJ L 324, 10.12.2007
|Amending act(s)||Entry into force||Deadline for transposition in the Member States||Official Journal|
Regulation (EU) No 1235/2010
OJ L 348, 31.12.2010