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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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Ever since Novartis’s Kymriah became the first chimeric antigen receptor (CAR) T-cell therapy approved by the FDA for a cancer indication (acute lymphoblastic leukemia) in 2017, this class of…
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