This detailed, expanded analysis explores key access and reimbursement topics in the rare disease space in the United States, such as the approaches managed care organizations take to control both utilization and costs of orphan drugs. The study further explores U.S. payers’ perspectives on market access considerations for several rare diseases including cystic fibrosis, Duchenne muscular dystrophy, hemophilia A, and idiopathic pulmonary fibrosis.

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