In this case discussion led by UCSF professor of neurological surgery, Philip Starr, we addressed multiple ethical dilemmas of using off-label medical devices to treat rare pediatric movement disorders. Throughout our conversation, we referred to the American Academy of Pediatrics’ policy statement, “Off-Label Use of Medical Devices in Children”.
Whole genome sequencing has enabled the discovery of lesser-known genetic mutations that cause movement disorders in children for whom traditional therapies are unsuitable. Increasingly, these patients receive treatment through physician-directed, off-label (i.e., not FDA approved) use of medical devices. Off-label use, however, raises concerns regarding payment and expectations of clinical outcomes. Dr. Starr and other members of the UCSF Surgical Movement Disorders Center presented the case of a child with a rare form of dystonia whose parents sought treatment via off-label deep brain stimulation (DBS). The clinical team expressed concerns about the family’s ability to pay for treatment in the event of health insurance coverage denial, noting the possibility of accruing millions of dollars in medical bills over time. We questioned the ethical permissibility of screening for socioeconomic status when determining treatment eligibility, asking whether rejection of low-income families constitutes an unjust barrier to access or a justifiable protection against financial turmoil. We also addressed the hyperbolic, miraculous language used to describe off-label DBS in an online crowd-sourcing campaign, which the patient’s parents created in an attempt to raise funds. The group reflected on the informed consent process, and wondered whether this inaccurate portrayal of treatment benefits reflected the parents’ genuine expectations or constituted a strategy to induce sympathy from donors. We also shared concerns about parents’ sense of desperation potentially clouding their ability to appreciate the financial burden, clinical limitations, and scarcity of research knowledge regarding off-label DBS for their children’s conditions.