Efficacy, safety and cost of gene therapy medicinal products in the U.S. and Europe.

Abstract

Gene or advanced therapy medicinal products (ATMPs) are evolving innovative products that have recently been attracting medical and healthcare attention. ATMPs are offering advanced techniques in treating certain conditions, including rare diseases. Meanwhile, the short- and long-term safety and efficacy of these products raise concerns. In this study, a comparative analysis of gene therapy medicinal products in the U.S. and E.U. was carried out to provide an overview of their efficacy and safety. Data up until 2019 were collected from the Food and Drug Administration (FDA) and European Medicines Agency (EMA) regarding approved gene therapy products. The FDA and EMA websites and published clinical trials were searched to extract the required information. A total of eight gene therapies were identified that had been approved in the U.S. and/or E.U. These products are approved to treat various health conditions in different populations. Their efficacy and safety were evaluated on the basis of completed and ongoing pivotal clinical trials. ATMPs offer potential management for genetic disorders and rare health conditions that are untreatable. However, one of the main concerns associated with these therapies is the small sample size that is included in the pivotal clinical trials. The uncertain efficacy, incomplete trials and serious adverse events could be challenges that make some of these products a last-line therapy.