A Room with a View (and with a Gene Therapy Drug): Gene Therapy Medicinal Products and Genetic Tourism in Europe.

Abstract

In contrast to the extreme caution that has been imposed on genetic medical procedures, in European law genetic drugs, or medications, have found a legal loophole that allows flexible (perhaps too flexible) access to these drugs. In Europe, Gene Therapy Medicinal Products are a form of Advanced Therapy Medicinal Products and as such submitted to the marketing authorization procedure. However, there are legal mechanisms in place - such as compassionate use, named patient use, and hospital exception - that allow for their provision to patients without proper approval. This is not, de per se, problematic; the problem arises, though, because such mechanisms are neither properly regulated nor monitored, and their application differs substantially according to the jurisdiction. This disparity and lack of control have given rise to situations of genetic tourism, where patients in desperate need travel to so-called genetic paradises, looking for a miraculous, and extremely expensive cure. The outcome is sometimes tragic, endangering patients' safety and undermining confidence in genetic products.