EU-wide regulatory framework for advanced therapies

The Commission answers to industry’s urgent need for a single, farmework for gene, cell and tissue-based therapies by proposing an integrated legal framework for all advanced therapies.

The Commission published its official proposal for a regulation for a community regulatory framework on advanced therapies on 16 November 2005. The new rules will bring all advanced – gene, cell and tissue-based – therapies within a single, integrated and tailored European regulatory framework to ensure consistency across member states. 

The main elements of the proposed framework for advanced therapies are: 

  • a centralised marketing authorisation procedure, to harmonise and facilitate access to the EU market;
  • a new expert Committee for Advanced Therapies (CAT) within the European Medicines Agency (EMEA) to assess advanced therapy products; 
  • new, tailore-made technical requirements for advanced therapy products; 
  • strengthened risk management and traceability requirements; 
  • special incentives for small and medium-sized enterprises (SMEs).   

The Commission thinks that human tissue- and cell- based products should be founded on the philosophy of voluntary and unpaid donation, anonymity of both donor and recipient, altruism of the donor and solidarity between donor and recipient. 

As to the ethical aspects, the proposal respects national competence as regards the use of for example embryonic stem cells. In accordance with the subsidiarity principle decisions on such use or non-use remain a national responsibility.

The industry welcomes the Commission's proposal as it lays down "the long-awaited harmonized rules for the approval and marketing of human tissue engineered products in the EU".  

Eucomed, the European association representing the majority of companies active in human tissue engineering in the EU: "A large share of the potential world-wide market estimated up to 400 billion euro will become accessible to the EU, if an appropriate harmonized framework is put in place. This in turn could make a substantial contribution to achieving the Lisbon goals." 

"Eucomed will now analyse the Commission’s proposal in detail. We need to be sure it fully takes account of the specific nature of human tissue engineered products which are neither pharmaceutical products nor medical devices. We also need to check whether it is in line with the approaches adopted in other parts of the world such as the US and whether it can make the EU attractive to foreign investment," said Maurice Wagner, Director General of Eucomed. 

EuropaBio, the voice of European Bioindustries: "The industry welcomes the choice of the Commission to opt for a Regulation in this field rather than a Directive to ensure that optimal and harmonised standards are used wherever a company or patient may be based in Europe."

"Europe is already late in this field yet has very promising technologies to offer. The US has had rules in place for these products for some time, but the field is changing continuously. US companies have benefited from a more predictable legislative environment and clarity. Predictability and clarity are two elements that are very important for companies to find investors prepared to invest in this research," said EuropaBio’s Secretary General Johan Vanhemelrijck.

Tissue-engineered skin substitutes are already widely used for treating, for example, severe burns and chronic wounds and knee joint injuries. Further research is being carried out to treat other joint cartilage defects and tissue engineered bone products are being developed for the treatment of bone fractures, osteoporosis and bone tumours. 

More complex products are under development to help treat various cardiovascular diseases related conditions. R&D is underway, for example, on tissue engineered blood vessels to replace damaged or blocked natural blood vessels. 

Human tissue engineering may one day regenerate entire organs such as bioartificial heart or liver.

The Commisison published its draft proposal for a community regulatory framework on advanced therapies in May 2005. A stakeholder consultation on this proposal took place right after, in the spring 2005. 

  • The regulation needs to be adopted by the Council and the European Parliament. 
  • Technical requirements should be ready shortly after adoption of the Regulation. 

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