ATMPs How to Successfully master Challenges and foster the Regulatory Success Rate
Advanced therapy medicinal products (ATMPs) bear game changing potential for the treatment of severe conditions for which we have no appropriate therapies today. The number of ATMPs reaching the market is expected to grow over the next decade. ATMPs comprise four distinct product categories, i.e. gene therapy medicinal products (GTMP) somatic cell therapy medicinal products (sCTMP) tissue engineered products (TEP) as well as combined ATMPs. Combined ATMPs 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. Though an EU regulatory framework is in place since 2008 and more than 900 ATMPs are in clinical trials, the number of authorized ATMPs with currently less than 10 is very low. Approximately 50 percent of the ATMP authorization applications are successful. This success rate is disappointing and significantly lower compared to other new therapeutic entities. Based on recent internal research data the overall success rate for new therapeutic entities at the time of approval is higher than 80 percent. In this article the following four key questions are discussed: