Abstract

Health technology assessment (HTA) agencies use end-of-life (EoL) criteria to justify higher incremental cost-effectiveness thresholds to improve access to treatments that may not otherwise demonstrate adequate value for money. We argue that, as currently defined, EoL criteria are biased against young people and children and consider some possible alternative approaches. A key component of HTA EoL criteria is short life expectancy. For instance, the National Institute for Health and Care Excellence and the Scottish Medicines Consortium EoL criteria include a life expectancy requirement of less than 24 and 36 months, respectively. The short life expectancy criterion reflects the notion that the patient has a terminal illness, and death from that disease is imminent. However, a recent HTA assessment involving a pediatric cancer drug raises questions about the adequacy of the short life expectancy criterion. In this case, life expectancy of the indicated population was greater than 3 years, with median overall survival of approximately 4 years. However, in a pediatric population, this significantly reduced life expectancy satisfies our intuitive sense of being at the EoL, but it does not meet the HTA short life expectancy criterion. On this basis we argue that current HTA EoL criteria are biased against young people and children. We consider potential alternatives to the short life expectancy criterion, including: eliminating the criterion; utilizing a separate threshold for non-elderly patients; and redefining the criterion in terms of the proportion of a life cut short. We argue for use of the latter concept and that equity considerations help to justify the use of a higher cost-effectiveness threshold in non-elderly patient populations. As currently defined the short life expectancy criterion is biased against non-elderly patients with a limited life expectancy. Redefining EoL criteria using the concept of a life cut short may help to address this problem.

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