Ethical standards for informed consent in pediatric cell and gene therapy trials must remain rigorousEthical Standards Remain Firm for Children in Gene Therapy Trials

The pediatric context poses specific challenges to the clinical translation of cell and gene therapies. We conducted a narrative review of literature addressing the responsibilities of researcher-clinicians in the context of clinical trials using cell and gene therapies, with an emphasis on first-in-human trials in minors. The inclusion criteria of our search yielded 16 papers, one of them addressing the exact intersection we were looking at, with the others circling this intersection. Only one of these 16 papers make a link to the wider theoretical debate on the tension between fulfilling the aims of clinical care and the aims of clinical research when conducting clinical trials–what is often referred to as the similarity vs. difference debate–and this debate’s connection to discussions of the central concept of the therapeutic misconception. Building on others’ claim that the similarity vs. difference framing is too simplistic to capture real-world nuance, we note that the concepts of therapeutic misconception and clinical equipoise both depend on mutually exclusive positions in this debate. We do not argue for jettisoning either of these concepts. Instead, we consider the resulting paradox to be productive. It is productive to integrate discussions of the therapeutic misconception and clinical equipoise into the similarity vs. difference debate, as the limitations of these discussions become more apparent in conjunction. In other words, researcher-clinicians taking their ethical duties seriously do not need to ‘solve’ these debates, but neither should this free them from engaging with the debates. Additionally, we discuss the legal context of cell and gene therapies in North America, concluding that nothing in the surveyed ethical literature suggests that greater uncertainties inherent in the field of early-phase cell or gene therapy trials justify relaxation of standards around informed consent for cell and gene therapy clinical trials.