From Cost Burden to Systemic Resilience: Structural Transformation of Healthcare Economies in the Era of Rare Disease

Rare diseases have changed from a pure medical problem into a system-wide economic challenge. The wide use of genetic sequencing, progress in drug development, and longer survival of patients have turned rare disease spending from high one-time costs into lifelong continuous consumption, placing great pressure on the healthcare system. This paper analyzes the economic burden of rare diseases from three dimensions: individuals, medical insurance, and society. It points out that direct medical costs are only one part, while hidden costs such as productivity loss and caregiving costs are even heavier. Current drug cost-effectiveness evaluation standards and annual budget payment models cannot accommodate orphan drugs, which are costly and require long-term treatment. This paper proposes a shift from passively bearing costs to enhancing health system resilience. By setting up a national rare disease registration and monitoring system, building multi-level risk-sharing funds, and using payment methods based on treatment results and payment in installments, the healthcare system can better deal with long-term pressure. These measures not only help rare disease patients but also prepare the whole society better for future public health challenges.