P47 How Do HTA Agencies Manage Uncertainties in the Long-Term Clinical Benefit of Gene Therapies? An Analysis of Recent Gene Therapy HTAs in England, Germany, France, Italy, Canada, and the United States

To date, seven gene therapies (GTs) have European regulatory approval and have been reimbursed at significant upfront costs. Assessing the long-term benefits of one-time GTs is challenging for payers due to the short duration of clinical data at the time of Health Technology Assessment (HTA). This research aims to understand the methods used by HTA agencies to assess the durability of GTs and whether the concept of biological plausibility is considered by payers.

Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: B. McCormick, C. Sheppard, E. Hutt, S. Schmitz, A. Kumar Source Type: research