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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
Asthma – Current Treatment – Treatment Algorithms: Claims Data Analysis – Pediatric Asthma (US)
Pediatric asthma is treated using two types of pharmacotherapies: rescue therapy for acute symptoms via bronchodilation (e.g., a SABA such as Teva’s ProAir HFA) and maintenance therapy to prevent…
Report
Acute Coronary Syndrome – Unmet Need – Unmet Need – Acute Coronary Syndrome: Secondary Prevention, on Top of Statin Treatment (US/EU)
This report covers the 12-month post-hospital management of secondary prevention in ACS patients. Reducing cardiovascular (CV) residual risk and achieving guideline-recommended LDL-C targets…
Report
Beta Thalassemia – Landscape & Forecast – Niche & Rare Disease Landscape & Forecast (US/EU5)
Beta thalassemia (BT) is a rare genetic disorder characterized by the reduced production of hemoglobin. BT minor is caused by a mutation in one hemoglobin beta (HBB) gene, and transfusion-dependent…
Report
Multiple Sclerosis – Unmet Need – Unmet Need – Multiple Sclerosis: Nonrelapsing Secondary-Progressive Multiple Sclerosis (US/EU)
No treatments are approved for nonrelapsing secondary-progressive multiple sclerosis (nrSP-MS), an MS disease course in which physical disability accumulates in the absence of superimposed relapses…
Report
Myasthenia Gravis – Current Treatment – Treatment Algorithms: Claims Data Analysis – Myasthenia Gravis (US)
Myasthenia gravis (MG) is a rare neuromuscular disorder caused by specific autoantibodies at the neuromuscular junction; in most patients, these autoantibodies target acetylcholine receptors. MG is…