Cost-effectiveness of molecularly matched off-label therapies for end-stage cancer – the MetAction precision medicine study

Authors

  • Anne Hansen Ree Department of Oncology, Akershus University Hospital, Lørenskog, Norway; Institute of Clinical Medicine, University of Oslo, Oslo, Norway
  • Gunhild M. Mælandsmo Department of Tumor Biology, Oslo University Hospital, Oslo, Norway; Institute for Medical Biology, University of Tromsø – The Arctic University of Norway, Tromsø, Norway
  • Kjersti Flatmark Department of Tumor Biology, Oslo University Hospital, Oslo, Norway; Institute for Medical Biology, University of Tromsø – The Arctic University of Norway, Tromsø, Norway; Department of Gastroenterological Surgery, Oslo University Hospital, Oslo, Norway
  • Hege G. Russnes Institute of Clinical Medicine, University of Oslo, Oslo, Norway; Department of Pathology, Oslo University Hospital, Oslo, Norway; Department of Cancer Genetics, Oslo University Hospital, Oslo, Norway
  • Mónica Gómez Castañeda Institute of Health and Society, University of Oslo, Oslo, Norway
  • Eline Aas Institute of Health and Society, University of Oslo, Oslo, Norway; Health Service Research Unit, Akershus University Hospital, Lørenskog, Norway; Division for Health Services, Norwegian Institute of Public Health, Oslo, Norway

DOI:

https://doi.org/10.1080/0284186X.2022.2098053

Keywords:

Cost-effectiveness, precision cancer medicine, quality-adjusted life years, willingness-to-pay

Abstract

Background

Precision cancer medicine (PCM), frequently used for the expensive and often modestly efficacious off-label treatment with medications matched to the tumour genome of end-stage cancer, challenges healthcare resources. We compared the 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.

Methods

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.

Results

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.

Conclusions

The models suggested that the high ICERs of PCM were driven by costs of 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. This can be an incentive for a public-private partnership for sharing drug costs in PCM, exemplified by ongoing European initiatives.

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Published

2022-08-03

How to Cite

Hansen Ree, A., Mælandsmo, G. M., Flatmark, K., Russnes, H. G., Gómez Castañeda, M., & Aas, E. (2022). Cost-effectiveness of molecularly matched off-label therapies for end-stage cancer – the MetAction precision medicine study. Acta Oncologica, 61(8), 955–962. https://doi.org/10.1080/0284186X.2022.2098053