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Belzutifan for treating tumours associated with von Hippel-Lindau disease

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1 Recommendations

1.1

Belzutifan is not recommended, within its marketing authorisation, for treating von Hippel-Lindau (VHL) disease in adults:

  • who need treatment for VHL-associated renal cell carcinoma, central nervous system hemangioblastomas or pancreatic neuroendocrine tumours, and

  • when localised procedures are unsuitable or undesirable.

1.2

This recommendation is not intended to affect treatment with belzutifan that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop.

Why the committee made these recommendations

VHL disease is a genetic condition that severely affects the quality of life of people with it, and their families and carers. The condition increases risk of certain tumours developing, and surgery is the main treatment option. There are no licensed medicines for the underlying causes of VHL.

Clinical-effectiveness evidence from a small study suggests that belzutifan reduces tumour size. It also suggests that it increases the amount of time people have before their condition gets worse, but by how much is uncertain.

There are also uncertainties in the economic model, as well as assumptions that likely favour belzutifan. So, it is not clear what the most likely cost-effectiveness estimates are for belzutifan and it cannot be recommended for routine use.

Even though some of the uncertainty in the clinical-effectiveness evidence could be addressed in the Cancer Drugs Fund, the cost-effectiveness evidence suggests that belzutifan is not likely to be cost-effective. So, belzutifan is not recommended for use in the Cancer Drugs Fund.