Renewed interest in the development of gene therapies has fueled commercial interest and investment into novel gene replacement technologies, which has resulted in a strong, diverse gene therapy pipeline and several regulatory approvals. As the number of gene therapy approvals rises, novel approaches to value assessment and reimbursement schemes 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 debilitating rare diseases such as SMA, beta thalassemia, and others.


  • 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 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.

Table of contents

  • Rare Diseases And Orphan Drugs - Access & Reimbursement - Detailed, Expanded Analysis - Rare Diseases And Orphan Drugs (US)

Author(s): Raina Priyadarshini, Ph.D; Chris Lewis

Raina is a senior analyst on the Infectious, Rare, and Niche Diseases team at Decision Resources Group.

Before joining DRG, Raina worked in drug development for novel disease targets. She gained experience assessing market research and competitive intelligence in rare disorders in multiple therapy areas.

She holds a Ph.D. in molecular biology from the National Institute of Immunology in India.

Chris Lewis serves as primary research manager, U.S. Access and Reimbursement, with responsibility for coordination, content review and content generation of the market access and reimbursement insights at DRG. Content is based on online surveys of managed care organizations and physicians and expert analysis of reimbursement and prescribing patterns of key therapies treating various disease states.

Lewis was an analyst/senior analyst for the group’s HealthLeaders-InterStudy subsidiary for eight years, specializing in the managed care and pharmacy benefit management industries. Throughout her tenure, she has produced the Health Plan Analysis reports for California, New York, New Jersey, Connecticut, and Pennsylvania and authored DRG’s series of pharmacy benefit manager profiles. She has also conducted numerous webinars for the group. She is a seasoned journalist with a B.A. in communications from California State University, Sacramento.

Related Reports

Rare Diseases And Orphan Drugs - Landscape & Forecast - Disease Landscape & Forecast: Gene Therapies For Rare Diseases

Approximately 80% of rare diseases have a genetic etiology. Gene therapies hold the potential to transform the treatment of rare diseases and may provide a functional cure for some. However, these...

View Details