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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.

Related Market Assessment Reports

Report
Venous Thromboembolism – Current Treatment – Treatment Algorithms: Claims Data Analysis – Venous Thromboembolism (US)
Venous thromboembolism (VTE) is the obstruction of a vein caused by a thrombus, described as deep vein thrombosis (DVT) or pulmonary embolism (PE), depending on the vein affected. Anticoagulants…
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Cystic Fibrosis – Unmet Need – Unmet Need – Cystic Fibrosis (US EU)
Cystic fibrosis (CF) is a genetic disease associated with more than 2,000 mutations identified in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. These mutations cause aberrant…
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Asthma – Current Treatment – Treatment Algorithms: Claims Data Analysis – Adult Asthma (US)
Two types of pharmacotherapies are used in parallel to treat adult asthma: rescue drugs and maintenance therapies. Rescue drugs are inhaled therapies that treat the acute symptoms of asthma via…
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Attention-Deficit-Hyperactivity Disorder – Current Treatment – Treatment Algorithms: Claims Data Analysis – Adult ADHD (US)
Attention-deficit / hyperactivity disorder (ADHD), a common childhood neurodevelopmental condition that can persist into adulthood, affects about 6% of U.S. adults, or approximately 15.5 million…
Report
Hyperkalemia – Current Treatment – Treatment Algorithms: Claims Data Analysis – Hyperkalemia (US)
Hyperkalemia is defined as a serum potassium level above 5.0 mEq/L. This report covers the management of chronic hyperkalemia through diuretic therapy, correcting metabolic acidosis when present,…