Abstract:
Rapidly evolving fields such as cell and gene therapies that involve state-of-the-art technology hold out possibilities that may be ahead of
what ethics, guidelines and the law have considered. This results in a regulatory lag. Furthermore, ethical and legal considerations are often
debated in real time as issues pertaining to these technologies that were previously not considered begin to come to the fore. Finding the
appropriate balance between facilitating potential therapeutic gains and ensuring the safety interests of recipients of the new treatments
requires close attention, especially for minors. This vulnerable population frequently has off-label treatment prescribed on the basis of
extrapolation of clinical trial data derived from adults, which is ethically and scientifically questionable. In this article we discuss how best
to maintain ethical integrity while introducing innovative cell and gene therapies to minors. We advocate that clinical trials of promising
innovative therapies should be designed so that testing in adults is followed as soon as possible by testing in minors, given the impressive
gains that have recently been reported.
