Orphan Drugs | Access and Reimbursement | US | 2017

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’s 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.

Questions Answered in This Report:

  • With high list prices and expanding treatment choice, orphan drugs (ODs) will be increasingly subject to reimbursement restrictions and utilization controls. Which ODs receive favorable formulary placement, and what approaches do payers use to manage utilization and costs? How restrictive do prescribers perceive these controls to be? How do utilization controls affect prescribing and OD selection? How will prescribing and OD selection change in the future?
  • Payers will increasingly focus on value assessment and pharmacoeconomic analysis in formulary decision-making for rare-disease drugs. What do payers perceive as the threshold for employing utilization management (UM) controls on rare-disease drugs? What types of cost-effectiveness analyses are managed cared organizations (MCOs) using, and which pharmacoeconomic data are most compelling? What types of contracting agreements do payers use when covering ODs?
  • Emerging ODs that bring meaningful clinical benefit and/or positive differentiation from current SOCs on clinical or cost factors will most likely obtain favorable reimbursement terms and uptake. How will emerging ODs be reimbursed and prescribed? What improvements do neurologists, pulmonologists, hematologists, and MCOs desire from emerging ODs?


  • Markets covered: United States.
  • Methodology: Surveys of 32 hematologists/pediatric hematologists, 31 neurologists/pediatric neurologists, 30 pulmonologists/pediatric pulmonologists, and 30 MCO officials (i.e., 15 pharmacy directors and 15 medical directors) in February and March of 2017.
  • Indication coverage: Cystic fibrosis, Duchenne muscular dystrophy, spinal muscular atrophy, sickle cell disease, beta-thalassemia.
  • Key drugs covered: Kalydeco, Orkambi, Cayston, Tobi Podhaler, Spinraza, Exondys 51, Exjade, Desferal, Ferriprox
  • Key companies mentioned: Biogen, Sarepta Therapeutics, Vertex Pharmaceuticals, Gilead, Novartis, Apotex, Genentech, Boehringer Ingelheim
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