Resource impact summary report
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.
Recommendation
NICE has recommended danicopan as an add-on to ravulizumab or eculizumab as an option for treating paroxysmal nocturnal haemoglobinuria (PNH) in adults who have residual haemolytic anaemia only if:
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they have clinically significant extravascular haemolysis while on treatment with a complement component 5 inhibitor (C5 inhibitor) and
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the company provides it according to the commercial arrangement.
This recommendation is not intended to affect treatment with danicopan 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 healthcare professional consider it appropriate to stop.
Eligible population for danicopan with ravulizumab or eculizumab
Table 1 shows the population who are eligible for danicopan as an add-on to ravulizumab or eculizumab in each of the next 5 years, including population growth.
| Eligible population | Current practice (without danicopan) | 2024–25 | 2025–26 | 2026–27 | 2027–28 | 2028–29 |
|---|---|---|---|---|---|---|
|
People eligible for danicopan |
92 |
93 |
94 |
95 |
96 |
97 |
The following assumptions have been used to calculate the eligible population:
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The PNH National Service annual report estimates that 1,015 people are within the service in England. It also estimates that around 38% of these people are on complement inhibitors.
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Expert clinical opinion is that around 80% of people with PNH having C5 inhibitors will remain anaemic.
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It is also thought that about 30% of these people will have clinically significant extravascular haemolysis.
The current and future uptake for the treatment options need to be entered into the resource impact template to reflect local data and assumptions.
Treatment options for the eligible population
The comparator treatments for the eligible population are pegcetacoplan (TA778) and iptacopan (TA1000).
Most people are currently treated with pegcetacoplan. Danicopan would be an add-on treatment to ravulizumab or eculizumab. Iptacopan (NICE TA1000, published September 2024) will also be a comparator treatment. Uptake of iptacopan for this group of people is unknown at this stage but can be entered into the template to reflect local assumptions.
Danicopan is an oral treatment but as it is an add-on treatment to either ravulizumab or eculizumab which are both administered subcutaneously, a capacity benefit is therefore not expected with this treatment.
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 danicopan available to the NHS with a discount.
Users can input the confidential price of danicopan and amend other variables in the resource impact template.
The payment mechanism for the technology is determined by the responsible commissioner and depends on the technology being classified as high cost.
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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 57.16 million people).
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This is because we do not think practice will change substantially as a result of this guidance and the population size is small.
For further analysis or to calculate the financial impact of cash items, see the resource impact template.
Key information
| Time from publication to routine commissioning funding |
90 days |
|---|---|
| Programme budgeting category |
03X |
| Commissioner(s) |
NHS England |
| Provider(s) |
NHS hospital trusts |
| Pathway position |
Second line |
About this resource impact summary report
This resource impact summary report accompanies the NICE guidance on danicopan with ravulizumab or eculizumab for treating paroxysmal nocturnal haemoglobinuria and should be read with it. See terms and conditions on the NICE website.
ISBN: 978-1-4731-6125-436