Burosumab for treating X-linked hypophosphataemia in adults

Recommendation

NICE has recommended burosumab, within its marketing authorisation, for treating X‑linked hypophosphataemia (XLH) in adults. Burosumab is only recommended if the company provides it according to the commercial arrangement.

Eligible population for burosumab

Prevalence and mortality of individuals with X-linked hypophosphataemia estimates the prevalence of XLH in adults to be 15.7 per 1 million. Abstracts from the Bone Research Society 2021 use data from the early access program to estimate that a total of 305 adults with XLH will be seen by NHS services in England.

Many patients are localised to a few expert bone centres. Users can manually enter the eligible population in the resource impact template to reflect their eligible population.

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

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

Treatment options for the eligible population

Usual treatment for XLH in adults is oral phosphate and active vitamin D. Burosumab is used in the NHS for treating XLH in people under 18; this evaluation is for treating XLH in adults.

The patient experts stated that conventional treatment such as oral phosphate and active vitamin D is ineffective at managing XLH, and many people find the treatment intolerable.

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 burosumab available to the NHS with a discount. The size of the discount is commercial in confidence.

The confidential price of burosumab 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 treatment duration for burosumab is assumed to be throughout the patient's lifetime.

Experience with the Early Access Programme indicates that burosumab is self-administered subcutaneously by most patients.

Monitoring is provided in an outpatient setting which includes additional serum phosphate tests for the initial treatment phase with burosumab; no other tests are needed beyond those provided in routine care for adults with XLH.

Using burosumab may reduce the excess incidence of fractures in people with XLH. There is uncertainty over the reduction of fracture incidence rates because of a lack of data on the risk of fracture in people with XLH and normalised serum phosphate in the longer term. The incidence rates of fractures can be inputted to reflect local practice in the resource impact template.

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

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

About this resource impact summary report

This resource impact summary report accompanies the NICE guidance on burosumab for treating X-linked hypophosphataemia in adults and should be read with it. See terms and conditions on the NICE website.

ISBN: 978-1-4731-6535-9