Accelerating Patients’ Access to Advanced Therapies in the EU

Berlin-Brandenburg Center for Regenerative Therapies (BCRT), Charité – Universitätsmedizin Berlin, Germany; Division of Medical Biotechnology, Paul-Ehrlich-Institut-Federal Institute for Vaccines and Biomedicines, Paul-Ehrlich-Straße 51-59, Langen, 63225, Germany; Department of Nephrology and Internal Intensive Care, Charité – Universitätsmedizin Berlin, Germany Correspondence:Mohamed Abou-El-Enein, Clinical Development Platform, Berlin-Brandenburg Center for Regenerative Therapies (BCRT), Charité – Universitätsmedizin Berlin, Campus Virchow, Augustenburger Platz 1, 13353 Berlin, Germany. E-mail: [email protected] Pharmaceutical developers are continuously searching for potential drug development targets. The time frame to develop and bring a new drug to the market may take 15 years, on average, starting from concept to final marketing, and the cost is likely around a billion dollars. Although technological advances, such as high-throughput identification of agents, have expedited the process of finding new drug targets, and a steady increase in new therapy concepts can be noted, many malignancies, for instance, still cannot be efficiently tackled or cured. The majority of new therapies continues to be small molecule based; advanced therapy medicinal products (ATMPs), a category that enfolds cell and gene therapies and tissue-engineered products, make up only a minor aspect of this market, although their market share is expected to be increasing. ATMPs have gained much attention because they offer potential treatments for serious conditions where small molecule therapies are inadequate. ATMPs are complex products by definition, requiring special research and development plus new regulatory and pricing strategies.