There is an ever-growing number of gene therapies progressing through clinical trials towards licensure. Providing long-term effects are a typical feature of gene therapies, however, the data to support long-term durability claims are often limited at licensure, given that long-term monitoring studies are often part of post-approval commitments by companies. Assessment of the potential for long term durability of the product and cost-effectiveness, based on the existing data, must therefore be completed by health technology assessors |
This review explores the potential benefits of enhancing the ability to predict the durability of gene therapy effects using different analogue categories of data. These analogue categories were evaluated to determine their ability to support claims of sustained gene therapy effects by utilizing arguments based on biological plausibility. The authors suggest a framework for identifying potential analogues, which may aid in establishing plausible or theoretical long-term outcomes that can be incorporated into value assessments of gene therapies.