Advanced-therapy medicines are medicines that are made from genes and cells. They may offer groundbreaking new treatment opportunities for many diseases and injuries.
Advanced therapies are different from conventional medicines, which are made from chemicals or proteins. There are four main groups:
- gene-therapy medicines: these contain genes that lead to a therapeutic effect. They work by inserting 'recombinant' genes into cells, usually to treat a variety of diseases, including genetic disorders, cancer or long-term diseases. A recombinant gene is a stretch of DNA that is created in the laboratory, bringing together DNA from different sources;
- somatic-cell therapy medicines: these contain cells or tissues that have been manipulated to change their biological characteristics. They can be used to cure, diagnose or prevent diseases;
- tissue-engineered medicines: these contain cells or tissues that have been modified so they can be used to repair, regenerate or replace tissue;
- combined advanced-therapy medicines: these are medicines that contain one or more medical devices as an integral part of the medicine. An example of this is cells embedded in a biodegradable matrix or scaffold.
European Union legislation
On 30 December 2008, legislation on advanced-therapy medicines came into force in the European Union (EU). The legislation defines what products are 'advanced-therapy medicinal products' and describes how they are authorised, supervised and monitored to ensure that they are safe and effective.
The legislation provides incentives to encourage research and development in the area of advanced therapies, by providing fee reductions for scientific advice and applications for marketing authorisation.
Role of the European Medicines Agency
All advanced-therapy medicines are authorised centrally via the European Medicines Agency. They therefore benefit from a single evaluation and authorisation procedure.
As for all medicines, the Agency continues to monitor the safety and effectiveness of advanced therapies once they are on the market. The Agency also gives scientific support to companies to help them design systems used to monitor the safety of these medicines.
Committee for Advanced Therapies
The Agency's Committee for Advanced Therapies (CAT) plays a central role in the scientific assessment of advanced-therapy medicines. It provides the expertise that is needed to evaluate advanced-therapy medicines.
During the assessment procedure, the CAT prepares a draft opinion on the quality, safety and efficacy of an advanced-therapy medicine. It sends this to the Committee for Medicinal Products for Human Use (CHMP). Based on the CAT opinion, the CHMP adopts a recommendation for the European Commission. The European Commission may grant or refuse a marketing authorisation on the basis of the Agency's recommendation.
The CAT also:
- gives recommendations on the classification of advanced-therapy medicines;
- reviews data on the manufacture and testing of medicines developed by small companies;
- contributes towards giving scientific advice on advanced-therapy medicines;
- contributes towards an environment that encourages the development of advanced-therapy medicines;
- provides scientific expertise and advice for any initiatives related to the development of innovative medicines and therapies, at the request of the European Commission.
The CAT engages with its stakeholders through regular interaction with its interested parties.
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- Regulation (EC) No 1394/2007
- Road map to 2015
- European Commission: advanced therapies