DRW Monthly
June 2007
Issue No. 7

EU Approves Regulation on Advanced Therapy Medicinal Products (ATMPs)

The European Council of Ministers reached a first-reading agreement in approving the Regulation on advanced therapy medicinal products (ATMPs) (gene therapy, somatic cell therapy and tissue engineered products) on 31 May 2007, following the European Parliament’s adoption of the amended European Commission (EC) proposal on 25 April 2007. The EC initially released its Proposal for a Regulation of the European Parliament and of the Council on Advanced Therapy Medicinal Products and Amending Directive 2001/83/EC and Regulation (EC) No 726/2004 in November 2005.

The legislation aims to establish a single, integrated European regulatory framework for ATMPs. The three kinds of advanced therapies covered in this Regulation are based on genes, cells and tissues and represent “innovative, regenerative therapies which combine aspects of medicine, cell biology, science and engineering for the purpose of regenerating, repairing or replacing damaged tissue/cells”(1). Whereas gene and cell therapy products have been classified as medicinal products and are regulated as such under the Directive 2001/83/EC on medicinal products, tissue engineered products have not yet been legally classified as either medicinal products or medical devices. Consequently, the regulation of tissue engineered products has varied according to each Member State’s classification and authorization of a particular product. The creation of a comprehensive framework for ATMPs will facilitate the research, development and authorization of these products as well as improve patient access to them.

The key elements of the Regulation include:

• the establishment of a centralized marketing authorization procedure for ATMPs;
• the formation of a multidisciplinary expert Committee for Advanced Therapies (CAT) within the European Medicines Agency to assess advanced therapy products, develop criteria and guidelines to evaluate these products, and monitor scientific developments in the field and;
• the provision of technical and risk management requirements adapted to the particular characteristics of these products to ensure quality and safety.(2)
  

  The Regulation applies to all ATMPs falling within the general scope of the Community legislation on medicinal products that are “intended to be placed on the market in a Member State and either prepared industrially or manufactured by a method involving an industrial process.”(3) The marketing authorization application for ATMPs will be required to be submitted through a centralized approach. The Committee for Medicinal Products for Human Use will be responsible for evaluating these products and will be assisted by the newly formed CAT within EMEA. In addition to complying with the pharmacovigilance requirements applicable to all centrally authorized medicinal products, applicants submitting ATMP applications will have to provide detailed information regarding procedures that will be followed to ensure product efficacy. Moreover, the EC may require the MAH to establish a risk management system or that specific post-marketing studies be performed. Traceability data will also have to be retained by the marketing authorization holder for a minimum of thirty years in order to guarantee that the individual product and its starting and raw materials can be traced through sourcing, manufacturing, packaging, transport and delivery.
  

  The next step in the approval process is for the Regulation to be translated into all EU official languages, checked by the linguistic revisers of the Council and European Parliament, formally adopted and finally signed. The Regulation is expected to be published within a few months and will apply in all 25 EU Member States one year after entry into force, or in mid-2008.
  

  In spite of its advances in integrating the EU CTD principles, the Czech government, along with its Central and Eastern European neighbors, still has work ahead to fully incorporate the administrative provisions of the EU CTD into national law. Differences with respect to interpreting the application process and the Competent Authority's view of his or her role and responsibilities in carrying out clinical research guidelines still vary widely from country to country. The ultimate goal should be a common understanding and approach to EU CTD concepts among all the EU member states.
  
  Related Resources
  

• 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
  

• Human Tissue Engineering and Beyond: Proposal for a Community Regulatory Framework on Advanced Therapies (Consultation Paper)
  

• New European Regulation on Tissue Engineering to Benefit Patients
  

• Proposal for a Regulation of the European Parliament and of the Council on Advanced Therapy Medicinal Products and Amending Directive 2001/83/EC and Regulation (EC) No 726/2004
  

• The Future EU Regulation on Advanced Therapies
  
  

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  (1) New European Regulation on Tissue Engineering to Benefit Patients
  (2) The Future EU Regulation on Advanced Therapies
  (3) New EU Rules Proposed for Gene, Cell and Tissue Engineered Therapies
  
  If you desire further information about this topic or any other regulatory issue, please feel free to contact me:

  Diane Whitworth
  DRW Research & information services, LLC
  (tel.) 301.916.9669
  drwresearch@comcast.net
  

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