ABSTRACT People living with inborn errors of metabolism (inherited metabolic diseases, IMDs) rely on lifesaving orphan drugs—therapies often developed and available primarily in high‐income countries. Yet in many low‐ and middle‐income regions, especially Latin America, patients face stark inequities in access. While an estimated 300 million people worldwide live with a rare disease, most Latin American countries until recently had little to no rare disease policy infrastructure 1. This perspective highlights the disparity in IMD drug access between wealthy nations and Latin America (with Chile as a case in point), examines the ethical imperatives for equitable and timely access, and explores barriers and potential policy solutions to ensure no patient is left behind.
Cabello et al. (Sun,) studied this question.