AbstractThis study explores tensions between rarity, research, and clinical decision-making in surgical diseases through a case study of pediatric pulmonary vein stenosis (PVS), an extremely rare and severe pediatric cardiopulmonary disease. We first examine the history of PVS treatment and illustrate how the field has been characterized by rapid clinical changes that outpace evidence. Through this context, we characterize patients and their ability to contribute to research as a limited resource that future patients depend on. We argue that this establishes future patients as legitimate stakeholders in decisions regarding current-day clinical management. Exploring this tension, we ask, to what extent should just resource utilization and research priorities shape clinical decisions, and can the dual role of patients-both as care recipients and as a limited resource-be ethically balanced? The article interrogates these questions through two proposals: standardizing care across centers, and regionalizing PVS treatment. We argue that regionalization is ethically justifiable, whereas standardization is not. Ultimately, we conclude that while a patient's primary role is as a recipient of care, research and resource considerations can and should still inform clinical management of rare diseases. Importantly, this is ethically possible only under conditions that preserve patient welfare and the accepted standard of care.
Asher et al. (Mon,) studied this question.