Rare Diseases and Orphan Drugs | Access & Reimbursement | US | 2021

As the number of gene therapy approvals rises, novel approaches to value assessment and reimbursement schemes for these innovative, high-cost treatments are rapidly gaining importance. Developers aiming to assess the market potential of investigational gene therapies face a host of challenging questions. How do payers make reimbursement decisions for gene therapies? How will they absorb the high cost of gene therapies? How will payer coverage policies affect physician prescribing decisions? This study analyzes the pricing and reimbursement (P&R) policies that regulate market access for gene therapies and explores the impact of P&R decisions on uptake of and patient access to gene therapies that treat the debilitating neuromuscular rare diseases spinal muscular atrophy and Duchenne muscular dystrophy.


  • To what degree do gene therapies impact payer budgets? What measures are payers likely to implement to rein in increasing costs?
  • How will the reimbursement environment for gene therapies evolve as more gene therapies reach the market?
  • How can manufacturers optimize P&R terms to facilitate market access?
  • What pharmacoeconomic models will payers use to assess the value of gene therapies?
  • How will potentially curative therapies be valued against chronic therapies that last lifelong?


U.S. Access & Reimbursement provides integrated brand- and disease-level insight on reimbursement dynamics and the impact of U.S. payer policy on physician prescribing behavior in the market access environment, including up-to-date analysis of drug coverage and restriction policies and payer and prescriber perspectives on key marketed drugs and receptivity to emerging therapies.

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