Rare Diseases and Orphan Drugs | Access and Reimbursement | US | 2018

The Orphan Drug Act of 1983 encourages the development of drugs for rare diseases, a process with unique clinical and commercial challenges. Although manufacturers of orphan drugs generally enjoy greater pricing latitude in the small populations their products serve, the discussion about orphan drug value assessment is rapidly evolving as the number of rare disease approvals climbs. A key concern for developers is how payers will control the growing cost of orphan drugs and how this policy will affect prescribing of these clinically valuable medications. Here, we provide an analysis of payer policy in cystic fibrosis (CF), idiopathic pulmonary fibrosis (IPF), Huntington’s disease (HD), spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), beta-thalassemia, and chronic immune thrombocytopenia purpura (ITP), as well as stakeholder receptivity to key emerging and recently launched orphan agents, including Vertex Pharmaceuticals’ triple combination therapy for CF, Novartis/AveXis’s AVXS-101 for SMA, Bluebird Bio’s LentiGlobin for beta-thalassemia, Rigel’s Tavalisee for chronic ITP, and Sarepta’s golodirsen for DMD.


  • What impact do orphan drugs have on payer budgets? What measures do payers expect to implement to rein in costs associated with orphan drugs?
  • What pharmacoeconomic models do/will payers use to assess the value of orphan drugs and gene therapies?
  • What utilization management restrictions do physicians encounter when prescribing key orphan drugs, and how commonly?
  • How does payer policy influence orphan drug prescribing patterns?


Access & Reimbursement: Provides in-depth insight regarding the impact of payer policy on physician prescribing behavior so that clients can build their market access strategy and optimize their brand positioning.

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