Clinical Development of Advanced Therapy Medicinal Products in Europe: Evidence That Regulators Must Be Proactive

Advanced therapy medicinal products (ATMPs), defined as gene therapy medicinal products (GTMPs), somatic cell therapy medicinal products, and tissue-engineered products (TEPs),[1,2] constitute a major class of innovative therapeutics that are being investigated as treatments for several diseases. De...

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Bibliographic Details
Published in:Molecular therapy 2012-03, Vol.20 (3), p.479-482
Main Authors: Maciulaitis, Romaldas, D'Apote, Lucia, Buchanan, Andrew, Pioppo, Laura, Schneider, Christian K
Format: Article
Language:English
Subjects:
Bone marrow
Charities
Clinical trials
Corporate sponsorship
Europe
Gene therapy
Genetic engineering
Genetic Therapy - legislation & jurisprudence
Genetic Therapy - methods
Humans
Manufacturing
Pharmaceutical industry
Product development
Small & medium sized enterprises-SME
Stem cells
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Summary:Advanced therapy medicinal products (ATMPs), defined as gene therapy medicinal products (GTMPs), somatic cell therapy medicinal products, and tissue-engineered products (TEPs),[1,2] constitute a major class of innovative therapeutics that are being investigated as treatments for several diseases. Despite the success of many such products in animal studies, few have reached more advanced regulatory milestones in Europe (Figure 1), such as ATMP Certification from the Committee for Advanced Therapies (CAT) of the European Medicines Agency (EMA),[3] regulatory scientific advice from the EMA,[4] or a marketing authorization application at the EMA.[5] Therefore, there is a need to identify the major stakeholders in the development of such products and to investigate why they have been unable to move these products farther down the development pipeline. As members of the CAT and EMA, we analyzed the data in the European Union Drug Regulating Authorities Clinical Trials (EudraCT) database (https://eudract.ema.europa. eu). Analysis of 318 ATMP trials performed between 2004 and 2010 revealed that the main sponsors are academia, charities, and small companies.
ISSN:1525-0016
1525-0024
DOI:10.1038/mt.2012.13