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Draft guidance consultation

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1 Recommendations

1.1

Donidalorsen should not be used for preventing recurrent attacks of hereditary angioedema (HAE) in people 12 years and over.

1.2

This recommendation is not intended to affect treatment with donidalorsen 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. For young people, this decision should be made jointly by the healthcare professional, the young person, and their parents or carers.

What this means in practice

These are NICE's draft recommendations. If these recommendations become final, donidalorsen would not be required to be funded and should not be used routinely in the NHS in England for the condition and population in the recommendations.

This is because there is not enough evidence to determine whether donidalorsen is value for money in this population.

Why the committee made these recommendations

Usual treatment to prevent recurrent attacks of HAE includes berotralstat, C1-esterase inhibitors, garadacimab and lanadelumab.

Evidence from a clinical trial shows that donidalorsen reduces the rate of HAE attacks compared with placebo. It has not been directly compared with any of the usual treatments. An indirect comparison suggests that it is likely to work as well as:

  • berotralstat

  • C1-esterase inhibitors

  • lanadelumab taken every 4 weeks.

But the evidence suggests that donidalorsen may be less effective than garadacimab and fortnightly lanadelumab.

There are uncertainties in the economic model because:

  • of how relative treatment effects and long-term attack rates are modelled

  • there is no stopping rule included for berotralstat

  • subsequent treatments and their impact on costs are not included.

Because of the uncertainties in the clinical- and cost-effectiveness evidence, it is not possible to determine the most likely cost-effectiveness estimates for donidalorsen. So, it should not be used.