A New Regulation Advanced Therapy Medicinal Products. By Alexandra Hartmann, MSc

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1 A New Regulation Advanced Therapy Medicinal Products By Alexandra Hartmann, MSc 8 December 2008

2 Regulation EC No 1394/2007 for Advanced Therapy Medicinal Products (ATMP Regulation) 1 was published in the Official Journal on 10 November 2007 and will apply from December The regulation was anxiously anticipated by industry, since the objective was to achieve harmonized legislation, especially for tissue engineering products (TEPs), in the EU. The first TEPs were placed on the market in the early 21 st century, but regulations were not defined in many Member States and requirements varied from one Member State to another, making many of these innovative products available only on local markets. The limited availability of these products to patients is also due to the fact that most TEP manufacturers are small or mediumsized enterprises (SMEs), which only operate in their own regions. The situation was dissatisfying, and community legislation was requested by industry and Competent Authorities. Many parties stressed that the new legislation should avoid overlap with existing legislation and minimize the risk of gray areas for borderline products. Since ATMPs are indicated for the purpose of regeneration, repair or replacement of damaged tissue and cells, the scope of this new regulation includes gene therapy products and somatic cell therapy products, as well as tissue-engineered products. It was agreed by all parties participating in the consultation that for ATMPs, special technical requirements need to be taken into account and that the safety and efficacy of these products need to be evaluated by experienced experts in the field. The regulation as lex specialis amends Directive 2001/83/EC 2 and Regulation EC No 726/2004; 3 therefore, ATMPs are classified as medicinal products and need to follow the principles of pharmaceutical legislation and standards, which include a mandatory Centralized Procedure for ATMP registration. Not all stakeholders supported the mandatory Centralized Procedure; SMEs especially raised concerns because of the administrative and financial burden. Since legislation applicable to medicinal products will also apply to ATMPs, they, like medicinal products, must: submit product dossiers in electronic CTD format 4 include a Pediatric Investigational Plan (PIP) 5 in applications for the Centralized Procedure but 6 designations for orphan status can be submitted as applicable and orphan drug incentives also apply for ATMPs One of the strongest arguments for the Centralized Procedure was implementation of the Committee for Advanced Therapies (CAT), as well as making products available to patients in all Member States. But ethical concerns were taken into account, and individual Member States can pass national laws that prohibit or restrict products containing, consisting of or derived from particular kinds of cells. Committee for Advanced Therapies (CAT) CAT will be comprised of specific experts who cover all areas of scientific expertise applicable to ATMPs, e.g., medical devices. All Member States will be represented on CAT and members may be accompanied by an expert for meetings. In addition, the European Commission will nominate representatives from patient associations and representatives for clinicians. CAT s main task will be to evaluate an ATMP s quality, safety and efficacy and prepare a draft opinion for final approval by the Committee for Medicinal Products for Human Use (CHMP). CAT will advise applicants on product development if the related product falls within the scope of the regulation. Its members also will provide recommendations on the regulatory route for borderline products. Further advice and assistance may be requested for development of guidance documents, for scientific advice procedures and for any other task related to the expertise available within CAT. Definitions Due to the great diversity of cell- and tissuebased products, it is often difficult to determine whether a product falls within the scope of the ATMP Regulation. Especially when deciding whether a product meets the definition of a tissue-engineered product, a very detailed outline of product characteristics is essential. Hence, the European Medicines Agency (EMEA) provides a recommendation in a 60 day process. The definitions of gene therapy medicinal product and somatic cell therapy product are given in Directive 2001/83/EC and have not been altered. During consultation, the framework for which products should be included in the scope of tissue-engineered products and combined advanced therapy products was discussed extensively, since the gray areas and any remaining unregulated products should be eliminated by the ATMP Regulation. Choosing the correct classification is still Regulatory Focus 9

3 challenging, but the main principles of the definition for products containing or consisting of cells or tissues are as follows: These products will only be considered as ATMPs if their cell and tissue components are substantially manipulated and follow the definition of engineered. Process steps that are not considered as substantial manipulation are listed in Annex I 7 of the regulation. If a product that contains or consists of tissues or cells of human origin has only undergone manipulation such as lyophilization or freezing, the manufacturer must comply with requirements described in Directive 2004/23/EC 8 and its implementing directives. If a product contains or consists of nonengineered, nonviable tissues or cells of animal origin, it is regulated as a medical device. A product that contains a medical device as an integral part and that contains viable cells falls under the definition of ATMPs. The same is true if the tissue and cell component is nonviable but has the primary function on the body. If the device part of such a combined product has the primary function on the body and the nonviable tissue or cells are of animal origin, assessment will be performed by a Notified Body according to Medical Devices Directive (MDD 9 ). If the tissue or cell part is of human origin, the concerned Competent Authority of the tissue establishment should be involved in evaluating quality, safety and functionality. Technical Requirements for ATMPs During the consultation, the need to take into account specific technical characteristics of tissue engineering products was highlighted. For example, traceability from donor to recipient and vice versa to protect against potential risk to the patient is an important safety factor that needs to be ensured. Especially for traceability, there is a shared responsibility between the Marketing Authorization Holder and the user (e.g., hospital, private practitioner). The users should have a system in place that links each product with the concerned patient. To date, EMEA has published some guidance as to what data will be expected to substantiate ATMP quality, safety and efficacy, with respect to: 10 human cell-based medicinal products safety and efficacy follow-up risk management 11 Further guidelines on more specific topics, e.g., xenogeneic cell therapy medicinal products and gene therapy medicinal products are available on the EMEA website for medicines and emerging science. In parallel, the European Commission started public consultations on the following changes or alterations: Part IV, Annex I to Directive 2001/83/ EC, to implement specific requirement for the content of the CTD product dossier 12 certification of quality and nonclinical data for small and medium-sized enterprises, 13 to give provisions on how and what data should evaluated within this new procedure good clinical practice specific to advanced therapy medicinal products 14 All these new guidelines and/or (drafts of) directives emphasize a risk-based approach, whereby the risk for patients should be defined on a product-by-product basis, with the manufacturer responsible for implementing the appropriate actions and collecting supporting data to eliminate unacceptable risk. Incentives The commission proposed several incentives to promote further development of ATMPs. Fee reduction for scientific advice is introduced for all ATMP applicants to encourage contacting the 10 December 2008

4 agency during development. 90% reduction in fees for scientific advice for SMEs and 65% reduction for other applicants the possibility of submitting quality and other nonclinical data in order to obtain an evaluation and certification of such data for SMEs all usual incentives for SMEs, such as support by the SME office and translation service will also apply for registered SMEs producing ATMPs during the transition period, 100% reduction of the marketing authorization fee for products that are legally on the market as of 30 December 2008 during the transition period, 50% reduction of the marketing authorization fee and first year postauthorization activities for hospitals and SMEs that can prove a specific health interest for the product Transition period Tissue engineering technology is currently used for treating burns and ulcers and cartilage defects with repair and regeneration systems. These tissue engineering products are legally on the market in some Member States. More complex products, such as therapies to repair heart tissue damaged by myocardial infarction (MI), are in the development stage. Products that are currently legally on the market will benefit from the transition protection. This adopted measure ensures a supply for patients and helps concerned companies and hospitals file product dossiers for licensing applications. Gene and somatic cell therapy products, which have been regulated in the EU since before Regulation EC No 1394/2007 went into force, shall comply no later than 30 December The licensing procedure for tissue engineering products shall be finalized at the latest on 30 December Since many guidelines for ATMPs and especially tissue engineered products are still under development, EMEA 15 is encouraging companies that have products legally on the market to discuss available data, and is offering to provide scientific advice on a case-by-case basis to support companies in preparing a product dossier for a successful evaluation. However the acceptance of clinical data from registry studies and/or trading experience as proof of safety and efficacy is still unclear. A request for controlled clinical trials would result in the failure of several SMEs, since they are not able to sponsor such trials. Furthermore, for some indications, it is not possible to show efficacy within the transition period. Donation and Procurement The critical starting materials for ATMPs are tissues or cells. Access to human tissues and cells is regulated by the national adoption of Directive 2004/23/EC and related implementing Directive 2006/17/EC. 16 The primary objective was to have a common standard for quality and safety introduced in all Member States. Some Member States decided to implement more-stringent measures. This led to different approval procedures for running a procurement center and placed a heavy administrative burden especially on SMEs and hospitals to get access to cells and tissues. Exemptions ATMPs that are prepared on a nonroutine basis are exempt from the regulation. Such products can be used only within one Member State in a hospital under a medical practitioner s exclusive responsibility. The Member States shall ensure that the quality and safety of these products comply with standards equivalent to those implemented at the community level. These products will be available only by individual medical prescription. There should be no competitive advantage for ATMPs that apply for the exemption, due to the request for equivalent quality standards and their limited production on nonroutine basis. F Regulatory Focus 11

5 References 1. Regulation (EC) No 1394/2007 of the European Parliament and of the Council of 13 November 2007 on advanced therapy medicinal products and amending Directives 2001/83/EC and Regulation (EC) No 726/ Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the Community code relating to medicinal products for human use. 3. Regulation (EC) No 726/2004 of the European Parliament and of the Council of 31 March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency. 4. EMEA/563366/2007 EMEA implementation of electronic-only submission and ectd submission in the centralised procedure: statement of intent. 5. Regulation (EC) No 1901/2006 of the European Parliament and of the Council of 12 December 2006 on medicinal products for paediatric use and amending Regulation (EEC) No 1768/92, Directive 2001/20/ EC, Directive 2001/83/EC and Regulation (EC) No 726/ Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products. 7. Regulation (EC) No 1394/2007, ANNEX I Manipulations referred to in the first indent of Article 2(1)c. 8. Directive 2004/23/EC of the European Parliament and of the Council of 31 March 2004 on setting standards of quality and safety for the donation, procurement, testing, processing, preservation, storage and distribution of human tissues and cells. 9. Council Directive 93/42/EEC of 14 June 1993 concerning medical devices. 10. EMEA/CHMP/410869/2006 Guideline on Human cellbased medicinal products. 11. EMEA/149995/2008 Guideline on safety and efficacy follow-up risk management of advanced therapy medicinal products. 12. Public Consultation Paper: Proposal to amend Annex I to Directives 2001/83/EC as regards advanced therapy medicinal products. 13. Public Consultation Paper: Certification of quality & non-clinical data for small and medium-sized enterprises. 14. Public Consultation Paper: Guideline on good clinical practice specific to advanced therapy medicinal products. 15. EMEA/326145/2008 EMEA announcement: to manufacturers, companies and hospitals having advanced therapy medicinal products legally on the Community market in accordance with national or Community legislation. 16. Commission Directive 2006/17/EC of 8 February 2006 implementing Directive 2004/23/EC of the European Parliament and of the Council as regards certain technical requirements for the donation, procurement and testing of human tissues and cells. Author Alexandra Hartmann, MSc, is director of regulatory and clinical affairs for Arthro Kinetics Biotechnology GmbH in Krems an der Donau, Austria. She can be reached via at 12 December 2008

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