ISSUE:

Gene therapies are a type of advanced therapeutical medicinal product (ATMP) with the potential to revolutionise our approach to treating disease. They offer the possibility of curing, halting and intercepting disease, rather than managing symptoms over a patient's lifetime.

Many healthcare payers around the world rely on health technology assessment (HTA) and budget impact analysis (BIA) to assess the value and affordability of new technologies, including gene therapies. Whilst research has been conducted concerning how the full value of gene therapies can be realised in HTA, much less attention has been given to the challenges posed by gene therapies to the development, use and interpretation of BIA. Current approaches to BIA may judge gene therapies as unaffordable due to BIA’s short-term time horizons and narrow scope. Given the long-term benefits and wider societal impact of gene therapies, there is some concern that BIA as used currently may be systematically disadvantaging this type of therapy.

Several questions must be explored: What is the appropriate use of BIA? Is it and should it be used in decisions of reimbursement? Should BIA methodologies be adapted to account for the disadvantages faced by gene therapies, or does it serve its purpose(s) as currently deployed? What would be the implications of any changes?

OVERVIEW:

Following a brief introduction to the issues by the moderator (Grace Hampson, Associate Director, Office of Health Economics, UK) (4 mins), the panel will debate a set of proposed policy recommendations regarding the future of BIA for gene therapies, with reference to the questions above. Panellists will each speak for 12 minutes, providing their perspectives on current challenges of using BIA for gene therapies and their suggestions on what should be done (or not) about the key challenges. 20 minutes will be reserved for audience discussion.