A Catalyst for Reform: Charting a Future for Orphan Drug Exclusivity

Forty years after the passage of the Orphan Drug Act, the Catalyst Pharmaceuticals v. Becerra decision has ignited debate over the scope of orphan drug exclusivity. After a court ordered the U.S. Food & Drug Administration (FDA) to revoke its approval of a drug for a rare neuromuscular disorder, which it found violated another drugmaker’s market exclusivity, FDA countered by reaffirming its commitment to its own interpretation of the Orphan Drug Act. This Comment explores the cascading effect that the clash between FDA and the Catalyst court has had, and could continue to have, by charting how these events have created doctrinal uncertainty—impacting the pharmaceutical industry, FDA, and federal courts. Uncertainty in the wake of Catalyst produces trickle-down effects that may deter orphan drug development, exacerbate high drug prices, and impede patient access. This Comment calls for resolving this unsettled landscape through congressional and agency actions to clarify the scope of orphan drug exclusivity and promote the goals of the Orphan Drug Act.