Regulating gene and cell-based therapies as medicinal products

Abstract

Gene and cell-based therapies (GCTs) have the potential to revolutionize healthcare. Recently, genetically modified T cell products reached the market, which have remarkable clinical benefits for patients with certain blood cancers. However, there are concerns about how to incorporate GCTs into existing regulatory systems for medicinal products. GCTs are different from other medicinal products and there are many uncertainties about efficacy and safety, particularly on the long term. This thesis investigates the design and implementation of regulations for marketing authorization of GCTs in the European Union and other countries such as the United States and Japan. Results show that regulatory authorities are searching for justified flexibility to ensure protection of public health without discouraging product development. Authorities around the world are moving towards a regulatory approach that combines early patient access based on less clinical evidence with further data collection once products are authorized for the market. Yet, specific requirements and procedures for GCT marketing authorization differ among countries. Some countries also implemented regulatory pathways that exempt products from marketing authorization regulations in order to facilitate research and availability in clinical practice. The thesis concludes that different choices are made by countries in how to balance the protection of public health and the facilitation of product development. Sharing knowledge on product development, including knowledge on clinical outcomes, is important to strike an appropriate balance between the protection of public health and the facilitation of product development, which could ultimately benefit patients on a regional and global level.