Technology forecast: advanced therapies in late clinical research, EMA approval or clinical application via hospital exemption

摘要

ABSTRACT Background : The umbrella term ATMPs (Advanced Therapy Medicinal Products) comprises cell therapies, gene therapeutics and tissue engineered products. After implementation of the Regulation 1394/2007, only a couple of products have obtained a centralized European marketing authorisation. Objectives : The aim of the presented study is to give an overview on ATMPs available within the European Union either via centralized marketing authorisation or via national Hospital exemption. Additionally, a forecast on innovative ATMPs in the process of EMA approval as well as in phase III and IV clinical trial is provided. Methods : Systematic literature search including ‘grey literature’ and database reviews as well as manual search following pre-defined search terms. Results : 8 ATMPs are currently available via centralized marketing authorisation. 6 new product launches are expected before 2020. At least 32 additional ATMPs are available in individual European Union member states via Hospital exemption. Another 31 potential ATMP candidates could be identified in industry-driven phase III research projects. Conclusion : Advanced therapeutic medicinal therapies are still in their early days, but constantly evolving. By 2020, innovative therapies targeting retinal dystrophy, ?-thalassemia, scleroderma, sickle-cell anaemia, adrenoleukodystrophy and leukaemia shall be available on the market.