ISSUE: Gene therapies treat, cure, or prevent disease by modifying the expression of an individual’s genes. Genetic diseases are rare, but are often associated with significant morbidity and mortality. Currently, Luxturna is the only FDA-approved gene therapy; however, approval for AVXS-101 is expected in early 2019 with a purported price tag of 4-5 million dollars per one-time treatment. While the current pricing and reimbursement structure may be able to handle such prices for a few ultra-rare conditions, what about when these therapies treat a condition that is more wide-spread? Hemophilia and its pipeline of eight gene therapies provide a case study for this scenario. Hemophilia is a lifelong condition with an average annual cost of approximately $185,000 per patient. Preliminary data on gene therapies have demonstrated clinical improvements and potential cost-offsets for up to three years. While questions of durability remain, payers must assess the value of these therapies. With the first hemophilia gene therapies expected to come to market in 2020, there is a pressing need to discuss this issue from multiple perspectives.

OVERVIEW: This panel will discuss gene therapy from multiple perspectives, using hemophilia as a case study. Lou Garrison will comment on the value of one-time curative therapies and pose key questions for discussion: What does gene therapy mean for patients, providers, and payers? Can the current system handle more widespread use of gene therapies? Is hemophilia an opportunity for payers to implement non-traditional payment models and how would patients and providers be affected? Mark Skinner will describe the patient experience and meaningful, patient-centered outcomes (15 minutes). Rebecca Kruse-Jarres will represent the provider perspective and discuss the balance between clinical best practices and cost-effective care (15 minutes). Jared Tate will discuss payer strategies to manage hemophilia spend and concerns surrounding current reimbursement and pricing models (15 minutes). Organized by the ISPOR Health Technology Assessment – Pharmacy & Therapeutics (HTA-P&T) Roundtable – North America