‘Putting The Costs And Benefits Of New Gene Therapies Into Perspective’ by Joshua T. Cohen, James D. Chambers, Madison C. Silver, Pei-Jung Lin, and Peter J. Neumann
Dan and Hannah’s commentary:
The advent of potentially curative gene therapies for otherwise fatal diseases appears to present some unique challenges for HE modelling and reimbursement decisions due to the high cost of the therapies.
The authors propose that these can be overcome by demonstrating large QALY gains, focussing on sub-populations where cost-effectiveness is more likely to be demonstrated, and the use of lifetime duration treatment effects despite having short trials (due to genomic modification).
Whilst comparators may be overpriced in some instances, this does not appear to be a critical issue. In ICER’s analysis of Zolgensma®, a gene therapy for spinal muscular atrophy type I, the comparator (best supportive care [BSC]) is deemed to be overpriced using a willingness-to-pay threshold of US$150,000 / QALY, with a cost-effectiveness ratio of $1.8 million. However, if the cost of BSC is reduced to zero, the cost-effectiveness ratio for Zolgensma® only increases from $235,000 / QALY to $310,000 / QALY.
Our interpretation is that overpriced comparators do not appear to be as much of an issue as they seem at first glance due to the large QALY gains generated by gene therapies.
In addition to the interpretations of the authors, we have noted that the full value of these treatments may not be captured by standard modelling approaches. While direct medical costs may be very high, the societal cost benefits of patients living full, healthy and economically productive lives are not normally investigated in base case analyses.
Should NICE consider a broader societal perspective (beyond that currently described in the reference case) for gene therapies by default to fully capture the benefits of such transformative treatments?
Dan Howard and Hannah Gillies