Belzutifan is recommended with managed access as an option for treating von Hippel-Lindau (VHL) disease in adults:
who need treatment for VHL‑associated renal cell carcinomas, central nervous system hemangioblastomas or pancreatic neuroendocrine tumours, and
when localised procedures are unsuitable or undesirable.
It is only recommended if the conditions in the managed access agreement for belzutifan are followed.
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 the 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.
Belzutifan has the potential to be cost effective, but more evidence is needed to reduce the uncertainties. More evidence from the trial and NHS practice could help address these. So, belzutifan is recommended for use with managed access.