Resource impact summary report
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 vamorolone as an option for treating Duchenne muscular dystrophy in people 4 years and over. Vamorolone is only recommended if the company provides it according to the commercial arrangement.
Eligible population for vamorolone
Table 1 shows the population who are eligible for vamorolone and the number of people who are expected to have vamorolone in each of the next 5 years.
| Eligible population and uptake | Current practice | 2024 to 2025 | 2025 to 2026 | 2026 to 2027 | 2027 to 2028 | 2028 to 2029 |
|---|---|---|---|---|---|---|
|
People eligible for vamorolone |
1,640 |
1,640 |
1,640 |
1,640 |
1,640 |
1,640 |
|
Uptake for vamorolone (%) |
0 |
75 |
85 |
95 |
95 |
95 |
|
People having vamorolone each year |
0 |
1,090 |
1,390 |
1,550 |
1,550 |
1,550 |
The following assumptions have been used to calculate the eligible population:
-
an estimated 2,500 people in the UK have Duchenne muscular dystrophy
-
approximately 2,100 (84%) of these cases are in England, based on population distribution
-
according to the company submission, an estimated 78% of people with Duchenne muscular dystrophy receive standard care glucocorticoid treatment
-
the incident population is part of the prevalent population figures
-
discontinuations in the vamorolone prevalent population have only been included for year 1. For subsequent years, these discontinuations can be locally input into the resource impact template.
The uptake for vamorolone is based on clinical expert opinion.
Treatment options for the eligible population
The comparator treatments for the eligible population are prednisolone and deflazacort.
Vamorolone and the comparator treatments are administered orally.
The comparator treatments for the eligible population and their method of administration are shown in table 2.
| Drug | Strength, container type, quantity |
|---|---|
|
Vamorolone |
100 ml, 40 mg/ml oral suspension |
|
Prednisolone |
28 x 5 mg tablets |
|
Deflazacort |
60 x 6 mg tablets |
Financial resource impact (cash items)
The company has a commercial arrangement. This makes vamorolone available to the NHS with a discount.
Users can input the confidential price of vamorolone 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.
For further analysis or to calculate the financial impact of cash items, see the resource impact template.
Capacity impact
No administration capacity impact is expected because vamorolone and the comparator treatments are all administered orally; so we do not expect there to be any additional appointments needed.
Vamorolone may result in a reduction of moderate to severe adverse events compared with prednisone. This is outlined in the resource impact template and can be assessed locally.
For further analysis or to calculate the financial capacity impact from a commissioner (national) and provider (local) perspective, see the resource impact template.
Key information
Table 3 Key information| Time from publication to routine commissioning funding |
90 days |
|---|---|
| Programme budgeting category |
PBC 07X |
| Commissioner(s) |
NHS England |
| Provider(s) |
NHS hospital trusts |
| Pathway position |
First line |
About this resource impact summary report
This resource impact summary report accompanies the NICE guidance on Vamorolone for treating Duchenne muscular dystrophy and should be read with it. See terms and conditions on the NICE website.
ISBN: 978-1-4731-6793-3
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