High-upfront-cost gene therapies pose considerable challenges to those conducting health technology assessment (HTA) and making resource allocation decisions. In particular, how to evaluate cost-effectiveness in the absence of definitive data about safety and effectiveness.
Since 2020, Australia has met these challenges with the ‘highly specialised therapies’ (HST) pathway. This has carved out a distinct funding pool for providing high-upfront-cost gene therapies, but not provided clear policy or guidelines on managing costs and uncertainty. To inform future guidance on health technology assessment and funding decisions, it is necessary to understand how decisions are currently being made.
This presentation will analyse federal decision making for high-cost gene therapies via public summary recommendations of the Medicare Services Advisory Committee (MSAC). With five gene therapies funded across thirteen HST applications in the last five years, and several more in the pipeline, this analysis (i) draws out key principles and patterns evident in recommendations; and (ii) evaluates the public summaries as sole tools of transparency for reimbursement decision making.
We contextualise these reimbursement decisions amongst several legal considerations, including: the constitutional challenges of federated health policy and funding, the lack of robust review mechanisms, and the absence of legal duties of fairness. These issues reflect the ongoing tension between government discretion over public health expenditure and accountability for its use.
With both the HTA framework and the HST pathway currently under review, we consider whether high-upfront-cost gene therapies necessitate greater transparency, and whether such therapies uniquely strengthen arguments for review or public obligations around fairness.