Nearly 35 years after the Orphan Drug Act of 1983 (ODA) became law in the United States, private investment in the development of drugs for orphan diseases continues unabated. Although the growing array of disease-modifying orphan drugs will continue to fulfill pressing unmet needs in rare-disease populations, it will also increasingly strain payer budgets. A key concern for stakeholders is understanding how clinical benefit and value-for-dollar will guide payer policy in covering orphan drugs and how that policy will affect prescribing and patient access to these clinically valuable medications. Orphan Drugs | Access and Reimbursement | US | 2017 provides comprehensive analysis of payer policy regarding drugs marketed for cystic fibrosis, beta-thalassemia, sickle cell disease, and orphan musculoskeletal diseases, as well as payer receptivity to key emerging agents (e.g., Vertex\u2019s ivacaftor\/tezacaftor), providing insights into how payers make coverage decisions and identifying levers to facilitate favorable reimbursement and uptake of novel therapeutics for rare diseases.<\/p>\n
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