1314O Cost-effectiveness of molecularly matched off-label therapies for end-stage cancer: The MetAction precision medicine study

Abstract

Precision cancer medicine (PCM), the expensive and often modestly efficacious off-label treatment with medications matched to the tumour genome of end-stage cancer, challenges the healthcare resources. We compared health effects, costs and cost-effectiveness of our MetAction PCM study with corresponding data from comparator populations given best supportive care (BSC) in two external randomised controlled trials. We designed three partitioned survival models to evaluate the healthcare costs and quality-adjusted life years (QALYs) as the main outcomes. Cost-effectiveness was calculated as the incremental cost-effectiveness ratio (ICER) of PCM relative to BSC with an annual willingness-to-pay (WTP) threshold of EUR 56,384 (NOK 605,000). One-way and probabilistic sensitivity analyses addressed uncertainty. We estimated total healthcare costs (relating to next-generation sequencing (NGS) equipment and personnel wages, molecularly matched medications to the patients with an actionable tumour target and follow-up of the responding patients) and the health outcomes for the MetAction patients versus costs (relating to estimated hospital admission) and outcomes for the BSC cases. The ICERs for incremental QALYs were twice or more as high as the WTP threshold and relatively insensitive to cost decrease of the NGS procedures, while reduction of medication prices would contribute significantly towards a cost-effective PCM strategy. Our health economic modelling compared patients with end-stage cancer analysed by NGS for the opportunity to provide molecularly matched therapies, with comparator populations instead receiving BSC, and showed that the high ICERs of PCM were driven by costs relating to the NGS diagnostics and molecularly matched medications, with a likelihood for the strategy to be cost-effective defying WTP constraints. Reducing drug expenses to half the list price would likely result in an ICER at the WTP threshold, which can be an incentive to a public-private partnership of sharing the costs of molecularly matched medicines in PCM, exemplified by the models used in the ongoing IMPRESS-Norway and Dutch DRUP trials.