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 'Population and treatments' and 'Unit costs' worksheets in the template to reflect local data and assumptions.
Guidance recommendations
See NICE's recommendations on nusinersen and risdiplam for treating spinal muscular atrophy.
Financial and capacity resource impact
The key drivers of resource impact are:
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Both nusinersen and risdiplam have been available through managed access and treatments will now be routinely commissioned.
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The estimated average dose for risdiplam.
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The proportion of people having year 1 or year 2 plus treatment for nusinersen.
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The proportion of people having high-dose nusinersen relative to low-dose nusinersen.
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The expansion of eligibility criteria from managed access may increase the level of uptake for nusinersen and risdiplam.
The companies both have a commercial arrangement. This makes nusinersen and risdiplam available to the NHS at a discount.
Users can input the confidential price of nusinersen and risdiplam and amend other variables in the resource impact template.
The payment mechanism for the technology is determined by the responsible commissioner and depends on whether the technology is classified as high cost.
For further analysis or to calculate the financial and capacity impact from a commissioner (national) and provider (local) perspective, see the resource impact template.
Eligible population for nusinersen and risdiplam
Table 1 shows the population who are eligible for nusinersen and risdiplam and the number of people who are expected to have nusinersen and risdiplam in each of the next 3 years, excluding forecast population growth.
| Eligible population and uptake | Number of people eligible for nusinersen and risdiplam | Uptake for nusinersen (%) | Uptake for risdiplam (%) | Number of people having nusinersen each year | Number of people having risdiplam each year |
|---|---|---|---|---|---|
|
Current practice (managed access) and future practice (routine commissioning) |
1,360 |
12% |
36% |
163 |
492 |
The following assumptions have been used to calculate the eligible population:
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Up to an estimated 1,600 people have 5q SMA in the UK, and 1,360 (85%) are estimated to be based in England.
The market shares for nusinersen and risdiplam are based on blueteq data. Future market share estimates are based on the expectation that the level of use will stay the same, but this can be amended in the resource impact template to reflect local assumptions.
Treatment options for the eligible population
The comparator treatments for the eligible population are onasemnogene abeparvovec, which is administered as a one-time intravenous infusion, or best supportive care. Nusinersen is administered via intrathecal injection and risdiplam is administered orally. Both are ongoing treatments.
For more information about the treatments, such as dose and average treatment duration, see the resource impact template.
Key information
| Time from publication to routine commissioning funding |
NHS England has agreed to provide funding from routine commissioning budgets so nusinersen and risdiplam will be funded from 14 May 2026 |
|---|---|
| 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 technology appraisal guidance on nusinersen and risdiplam for treating spinal muscular atrophy and should be read with it.
ISBN: 978-1-4731-9547-9
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