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Economics and Politics in Rare Disease Treatments: The Rising Cost and Challenges
Orphan drug approvals are rising, offering hope for rare disease patients — but at an average cost of $370,000 per year. With policy shifts, Medicare loopholes, and falling research funding, the future of rare disease treatment hangs in the balance.

Economics and Politics in Rare Disease Treatments
The cost of living with a rare disease in the United States is significantly higher than the cost of living with other types of health conditions. Findings published in a 2022 report by Chiesi Global Rare Diseases, a Boston-based business unit of Chiesi Farmaceutici S.p.A, revealed that the economic burden of a patient living with a rare disease is approximately 10 times higher than that of a patient living with a more common affliction. Chiesi states the average overall cost per rare disease patient in the U.S. is $266,000 annually, but in comparison, the average overall cost per patient living with a so-called “mass market” disease is roughly $26,000 annually.