EU rules for new medical products based on genes and cells
SUMMARY
Scientific advances bring new medicinal products based on gene or somatic-cell* therapy and tissue engineering* to treat human diseases. Since December 2008, legislation aims to ensure these are both safe for patients and can be available throughout the EU.
WHAT DOES THE REGULATION DO?
It lays down rules for authorising, supervising and monitoring advanced therapy medicinal products, known in the jargon as ATMPs. These can be used, for instance, in correcting knee cartilage defects in adults. The legislation protects patients from scientifically unsound treatments.
KEY POINTS
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The legislation creates a Committee for Advanced Therapies (CAT) within the European Medicines Agency. Its role is to give scientific opinions on the quality, safety and efficacy of advanced therapy medicinal products.
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The Committee for Medicinal Products for Human Use bases its authorisation decisions on the CAT’s opinions.
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Once authorisation is granted, the item is considered safe for human use throughout Europe.
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Manufacturers must comply with detailed labelling and packaging rules.
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Manufacturers must ensure that each product, and its raw materials, can be traced from initial sourcing through packaging, storage and delivery to its final destination.
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Hospitals and other medical locations must maintain traceability systems for both patients and the products used.
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Manufacturers must specify the remedial measures they will take if an authorised product is found to cause adverse reactions.
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Where there is particular cause for concern, the European Commission may request a manufacturer to establish a risk management system to identify, prevent or minimise any possible risks.
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The Commission draws up detailed guidelines on good clinical and manufacturing practice specific to advanced therapy medicinal products.
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A Commission report of March 2014 examined the status of advanced therapies in the EU and how the regulation had worked in practice. It noted that the legislation had established a recognised framework for assessing new advanced therapies.
BACKGROUND
Significant research takes place in the EU into advanced therapies. The EU’s clinical trials database, EudraCT, recorded up to 250 between 2004 and 2010. Almost 70 % of the entities involved were SMEs or not-for-profit organisations. Multinational pharmaceutical companies accounted for under 2 %.
KEY TERMS
* Somatic-cell therapy: experimental method of cloning genes and reintroducing them into cells to correct an inherited disease.
* Tissue engineering: manufacture of organs to be implanted and used in the human body.
ACT
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
REFERENCES
Act |
Entry into force |
Deadline for transposition in the Member States |
Official Journal |
Regulation (EC) No 1394/2007 |
30.12.2007 |
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Amending act(s) |
Entry into force |
Deadline for transposition in the Member States |
Official Journal |
Regulation (EU) No 1235/2010 |
1.1.2011 |
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RELATED ACTS
Report from the Commission to the European Parliament and the Council in accordance with Article 25 of Regulation (EC) No 1394/2007 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 (COM(2014) 188 final of 28.3.2014)
last update 15.10.2015