Objectives: To evaluate alternative methods to calculate and/or attribute economic surplus in the cost-effectiveness analysis of
single or short-term therapies.
Methods: We performed a systematic literature review of articles describing alternative methods for cost-effectiveness
analysis of potentially curative therapies whose assessment using traditional methods may suggest unaffordable
valuations owing to the magnitude of estimated long-term quality-adjusted life-year (QALY) gains or cost offsets. Through
internal deliberation and discussion with staff at the Health Technology Assessment bodies in England and Canada, we
developed the following 3 alternative methods for further evaluation: (1) capping annual costs in the comparator arm at
$150 000 per year; (2) “sharing” the economic surplus with the health sector by apportioning only 50% of cost offsets or
50% of cost offsets and QALY gains to the value of the therapy; and (3) crediting the therapy with only 12 years of the
average annual cost offsets or cost offsets and QALY gains over the lifetime horizon. The impact of each alternative
method was evaluated by applying it in an economic model of 3 hypothetical condition-treatment scenarios meant to
reflect a diversity of chronicity and background healthcare costs.
Results: The alternative with greatest impact on threshold price for the fatal pediatric condition spinal muscular atrophy type
1 was the 12-year cutoff scenario. For a hypothetical one-time treatment for hemophilia A, capping cost offsets at $150 000
per year had the greatest impact. For chimeric antigen receptor T-cell treatment of non-Hodgkin’s lymphoma, capping cost
offsets or using 12-year threshold had little impact, whereas 50% sharing of surplus including QALY gains and cost offsets
greatly reduced threshold pricing.
Conclusions: Health Technology Assessment bodies and policy makers will wrestle with how to evaluate single or short-term
potentially curative therapies and establish pricing and payment mechanisms to ensure sustainability. Scenario analyses
using alternative methods for calculating and apportioning economic surplus can provide starkly different assessment
results. These methods may stimulate important societal dialogue on fair pricing for these novel treatments.