Eur J Hum Genet 2021 Apr 14. Epub 2021 Apr 14.
Amsterdam UMC, Vrije Universiteit Amsterdam, Clinical Genetics, Section Community Genetics, Amsterdam Public Health research institute, Amsterdam, the Netherlands.
Somatic gene editing (SGE) holds great promise for making genetic therapy possible for many monogenic conditions very soon. Is our current system of European market authorization and reimbursement ready for the expected tsunami of gene therapies? At a recent workshop of the Netherlands ZonMw consortium on ethical, legal, and social implications of personalized medicine, we discussed the current possibilities for bringing new gene therapies to the clinic. In Europe, it is not yet clear whether the route via the European medicines agency as an advanced therapy medicinal product is the most appropriate for evaluation of highly personalized SGE applications, although this may optimally guarantee safety and effectiveness. Read More