Re-Examining the Ethics of Clinical Trial Approval in a Gene-Targeted Era Through a Case-Based Approach: Exploring the Implications of the N=1 Batten Disease Trial

Using a case-based approach, this article highlights the necessity of re-examining the ethics of clinical trial approval in a gene-targeted era. It centers on the n = 1 Batten disease drug trial, in which an antisense oligonucleotide drug was introduced as a novel drug therapy for an individual child with a neurodegenerative illness, and probes the ethics of equity, safety, clinical utility, and parental autonomy of the trial as a means to raise more global questions about the ethical underpinnings of clinical trial approval. With the promise of the widespread application of personalized medicine on the horizon, it is imperative that the greater scientific community think critically about the ethical foundation upon which drugs make their way to market.