Resource impact statement
NICE has recommended iptacopan, within its marketing authorisation, as an option for treating paroxysmal nocturnal haemoglobinuria (PNH) in adults with haemolytic anaemia. Iptacopan is only recommended if the company provides it according to the commercial arrangement.
We expect the resource impact of implementing the recommendations in England will be less than £5 million per year (or approximately £8,800 per 100,000 population, based on a population for England of 57.1 million people).
This is because PNH is a rare condition and the technology is a further treatment option with the overall cost of treatment for this patient group being unlikely to change significantly with the introduction of iptacopan.
Using information from the PNH National Service as a basis, Table 1 shows the population who are eligible for iptacopan and the number of people who are expected to have iptacopan in each of the next 5 years. These figures are based on the prevalent population only and account for expected growths in the total population. To avoid double counting, the eligible population also excludes people who are covered by NICE’s technology appraisal guidance on danicopan as an add-on treatment to a C5 inhibitor for treating extravascular haemolysis in adults with paroxysmal nocturnal haemoglobinuria (TA1010).
Table 1 Population expected to be eligible for and have iptacopan in England
|
Eligible population and uptake |
Current practice |
2024/25 |
2025/26 |
2026/27 |
2027/28 |
2028/29 |
|
People eligible for iptacopan – prevalent group |
116 |
117 |
118 |
119 |
120 |
121 |
|
Estimated uptake |
0% |
38% |
50% |
55% |
55% |
55% |
|
People having iptacopan treatment each year |
0 |
44 |
59 |
65 |
66 |
67 |
Iptacopan is an oral treatment and comparator treatments are intravenous or subcutaneous and require the initial loading dose to be administered in hospital, following which treatment is provided via a funded homecare service. Treatment with iptacopan will therefore save a limited number of outpatient appointments as this is delivered entirely via a funded homecare service. The need for chelation therapy within this population should also decrease, as there is a risk of this being needed with comparator treatments eculizumab and ravulizumab but not with iptacopan or pegcetacoplan. A local resource impact template has been produced to assist organisations in estimating the resource impact. Should users wish to model uptake for the incident group then they can adjust the assumptions in the market shares table within the ‘Inputs and eligible population’ worksheet. If there is any expected increase in the prevalence, users can adjust the assumptions in the population workings at the top of the ‘Inputs and eligible population’ worksheet.
The company has a commercial arrangement. This makes iptacopan available to the NHS with a discount. The size of the discount is commercial in confidence.
This technology is commissioned by NHS England. The National PNH Service is funded by NHS England as a Highly Specialised Service to treat people with PNH. The service consists of two centres, with one based at St James’ Hospital in Leeds and the other based in King’s College Hospital in London. People with PNH will be cared for and supported by one of these centres.
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