The RNA-based therapeutics patisiran and inotersen for hATTR represent an important advance in the treatment of a rare genetic disorder with high unmet need. However, despite consideration of potential broader benefits and of the contextual considerations associated with treatment of an ultra-rare disorder, the pricing of these drugs in the U.S. health care system was judged to represent low long-term value for money. Further efforts are needed to help align the price of these treatments with their demonstrated benefits in order to ensure sustainable access to high-value care for all patients.