Comparing cost-effectiveness outcomes of cell and gene therapies across HTA organisations

By Chloe Sheppard, Senior Analyst & Max Rex, Consultant


Cost-effectiveness analyses, as part of Health Technology Assessments (HTAs) for new therapies, appear to be growing in importance across Europe. These analyses are already required in many markets within and outside of Europe for HTAs, including Canada, England, and the Netherlands. In future, this could be the case for many other European markets. HTAs in Italy and Spain have traditionally focused on assessing the budget impact of new therapies, but new guidelines from the health authorities in both countries suggest that they are transitioning towards placing greater emphasis on cost-effectiveness to guide pricing and reimbursement (P&R) decisions.1,2

For many cell and gene therapies (CGTs), the provision of long-term cost savings to healthcare systems is often a key pillar of their value offering, despite their high upfront cost. Compared with traditional drugs, this can make it even more important for CGTs to demonstrate cost-effectiveness to convince payers of their value.

How have cell and gene therapies fared in cost-effectiveness HTAs to date?

At ISPOR Europe 2020, P4A presented a poster which analysed the cost per quality-adjusted life year (QALY) outcomes and HTA recommendations of three CGT case studies: Kymriah, Yescarta and Luxturna. Results were compared across HTA organisations in Canada, the United States, England, Scotland, the Netherlands, and Sweden. The research aimed to understand the varying assessment methodologies across the HTA organisations, and how this may impact cost-effectiveness outcomes for CGTs.

The key learnings from the research were:

  1. Several HTA organisations use process modifications for high-cost therapies, but few of these are specific to CGTs
  2. Cost per QALY outcomes for individual CGTs can vary across markets
  3. HTA outcomes for Kymriah, Yescarta and Luxturna in these markets have been largely positive

Even though HTA outcomes for Kymriah, Yescarta and Luxturna were largely positive in these markets included in our research, this could be driven in part by the nature of the conditions they are approved to treat – cancers and rare diseases. Higher cost per QALY outcomes, resulting from a higher willingness to pay from HTA organisations, are more likely to be acceptable for CGTs approved for orphan and oncology indications. As more CGTs come to market, and particularly in more prevalent indications, they will likely have to face tougher cost-effectiveness thresholds.

The current P&R model may not be sustainable as more high-cost CGTs come to market. Healthcare systems will have to adapt to the changing environment to ensure that patient access to therapies not compromised. The move from Italy and Spain towards cost-effectiveness could be an indicator that these markets are preparing to better assess the value of innovative high-cost treatments. As cost-effectiveness analyses will likely become a requirement of an increasing number of HTA assessments in European markets, all manufacturers will be forced to consider their strategies for this even more carefully in the future.