Objectives
The objective of this study is to explore the role of economic benefit in the HTA decisions across diverse market types: cost-effectiveness driven (UK), budget-impact driven (Italy), and clinical-differentiation driven (France). More specifically, the objective is to understand whether the clinical or the economic domain yields most of the key payer considerations underlying these decisions and whether this varies across markets representing different payer archetypes.
Methods
UK, Italy, and France were chosen as representative market archetypes for cost-effectiveness, budget impact, and clinical-differentiation, respectively. ATMPs assessed by HTA bodies within these markets were identified using NICE, AIFA, and HAS databases. From these, two ATMPs were selected to illustrate contrasting examples from a P&R perspective, specifically one with a negative HTA outcome and another with a positive HTA outcome. Only the initial assessment was considered, and reassessments were excluded. Key payers’ considerations on evidence package were extracted from HTA reports and were categorised as positive, some concerns/ uncertainties, or negative and as pertaining to the clinical vs the economic domain. A comparative analysis was conducted between the two selected ATMPs across the three markets.
Results
Yescarta (first indication) and Alofisel have been evaluated and are associated with positive and negative HTA outcomes, respectively, in the selected markets. Across markets, Yescarta’s positive HTA outcomes are either linked to conditions or facilitated by MEAs, while Alofisel is only reimbursed in France and only for a sub-group.
Key payer considerations on both Yescarta and Alofisel’s value and evidence package, underlying HTA decisions, mainly pertain to the clinical domain in all markets. In France and UK, for both Yescarta and Alofisel, there are some concerns pertaining to the economic domain, but these are caused by uncertainties over cost-effectiveness that result from uncertainties in the clinical benefit in the first place.
Conclusion
Across markets, the level of clinical benefit is the key determining factor in HTA decisions for ATMPs. However, ATMPs often do not have the right level of evidence to support demonstration of clinical benefit and, consequentially of economic benefit, at the time of launch.
For this reason, alternative reimbursement schemes, such as coverage with evidence development, and payments methods are utilised by payers to allow access to these innovative treatments despite the uncertainties.
However, if the uncertainty over the clinical benefit is too high, these will not even be considered, as it was the case for Alofisel. This is especially true in markets where clinical benefit is assessed first and is a gateway to the discussion on economics.
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