Resource impact summary report

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This summary report is based on the NICE assumptions used in the resource impact template. Users can amend the 'Inputs and eligible population' and 'Unit costs' worksheets in the template to reflect local data and assumptions.

NICE has recommended belzutifan 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.

Eligible population for belzutifan

The evaluation of tumour surveillance protocols and outcomes in VHL disease in a national health service (Maher et al. 2022) study estimated a maximum prevalence of 1.46 per 100,000 population.

The same study identified that of those with VHL disease, 170 had renal cell carcinomas, 183 had central nervous system hemangioblastomas and 36 had pancreatic neuroendocrine tumours. This total of 389 people equates to 46.2% of people with VHL disease.

Clinical expert opinion estimated that 35% would be unsuitable for localised procedures or that it would be undesirable.

Table 1 below shows the population who are eligible for belzutifan and the number of people who are expected to have belzutifan in each of the next 5 years. These figures include the impact of the predicted population growth.

**Table 1 Population expected to be eligible and have belzutifan in England**
Eligible population and uptake for belzutifan	Current practice	2024-25	2025-26	2026-27	2027-28	2028-29
People eligible for belzutifan	109	110	111	112	113	114
Market share for belzutifan (%)	0	33	67	95	95	95
People having belzutifan each year	0	36	74	107	108	72
People in the prevalent population who have belzutifan	0	36	74	107	108	109

Note that the number of people having belzutifan decreases in 2028-29 because the peak market share is expected to have been reached in 2026-27 and the treatment duration is modelled for 4 years.

Market share in 2024-25 is assumed lower due to the guidance publishing in October 2024.

The market share for belzutifan is based on consultant opinion. It can be amended to reflect local practice in the resource impact template.

Treatment options for the eligible population

Surgery and other localised procedures are the main treatment options for people with VHL, but sometimes they are not appropriate. The clinical and patient experts explained that there is an unmet need for effective new treatments for people with VHL when surgery is unsuitable.

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. Belzutifan is expected to deliver savings resulting from delaying the progression of VHL disease. This in turn reduces the costs associated with surgeries.

For more information about the treatments, such as dose and average treatment duration, see the resource impact template.

Financial resource impact (cash items)

The company has a commercial arrangement. This makes belzutifan available to the NHS with a discount. The size of the discount is commercial in confidence.

The confidential price of belzutifan can be put into the resource impact template and other variables may be amended.

The payment mechanism for the technology is determined by the responsible commissioner and depends on the technology being classified as high cost.

Further analysis is provided in the resource impact template, and the financial impact of cash items can be calculated.

Capacity impact

The phase 2 belzutifan trial (MK-6482-004) found the median treatment duration to be 21.7 months. Treatment was ongoing in 89% of people at point of data cut off. Treatment duration is unknown, and the resource impact template estimates 4 years but can be modelled up to 5 years.

The recommended dose of belzutifan is 120 mg (three 40 mg tablets) taken orally each day.

People with VHL disease may also require multiple surgeries and may incur costs from the short- and long-term complications of surgeries. The rates and costs associated with surgery and complications can be input into the resource impact template to reflect local assumptions.

There will be a capacity increase due to additional oral administration appointments (assumed to be one appointment in each 28-day cycle) for delivering treatment with belzutifan.

Table 2 shows the impact on capacity activity across the eligible population in each of the next 5 years.

**Table 2 Capacity impact (activity) in England**
–	Current practice	2024-25	2025-26	2026-27	2027-28	2028-29
Number of oral administration appointments	0	472	967	1,385	1,398	940
Number of follow-up appointments	0	73	149	213	215	145
Number of MRI scans	109	146	185	219	221	187

Further analysis is provided in the resource impact template, and the financial capacity impact, from a commissioner and provider perspective can be calculated.

Key information

Table 3 Key information on resource impact of belzutifan

Time from publication to routine commissioning funding	Managed access
Programme budgeting category	04B Endocrine, Nutritional and Metabolic problems, Endocrine
Commissioner(s)	NHS England
Provider(s)	Secondary care – acute
Pathway position	Treating tumours associated with von Hippel-Lindau disease

About this resource impact summary report

This resource impact summary report accompanies the NICE technology appraisal guidance on belzutifan for treating tumours associated with von Hippel-Lindau disease and should be read with it. See terms and conditions on the NICE website.

ISBN: 978-1-4731-6534-2