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    Orphan Drugs: U.S. Access & Reimbursement

    Nearly 35 years after the Orphan Drug Act of 1983 (ODA) became law in the United States, private investment into the development of orphan disease drugs continues unabated. However, the growing array of high-priced orphan drugs is putting substantial financial pressure on payers. A key concern for stakeholders is how payers will control the growing cost of orphan drugs, and those controls will impact physician prescribing and patient access to these clinically-valuable drugs. Comprehensive analysis of payer policy regarding drugs marketed for cystic fibrosis, β-thalassemia, sickle cell disease, and orphan musculoskeletal diseases, as well as payer receptivity to key emerging agents (e.g., Vertex’s ivacaftor/tezacaftor), will provide companies with valuable insights into how payers make formulary decisions and use access restrictions to control the cost of orphan drugs on their commercial plans.


    • What cost control measures and access restrictions are payers applying to reimbursement of Spinraza and Exondys 51?
    • How sensitive are payers to orphan drug costs? Is the degree of payer sensitivity to orphan drug prices relatively static, or changing over time?
    • What pharmacoeconomic models are payers employing to assess the value of orphan drugs?
    • What access restrictions do physicians and patients encounter with orphan drugs, and how common are these access restrictions?
    • How do payer access restrictions alter orphan drug prescribing patterns?