How are you taking part in this consultation?

You will not be able to change how you comment later.

You must be signed in to answer questions

  • Question on Consultation

    Has all of the relevant evidence been taken into account?

  • Question on Consultation

    Are the summaries of clinical and cost effectiveness reasonable interpretations of the evidence?

  • Question on Consultation

    Are the recommendations sound and a suitable basis for guidance to the NHS?

  • Question on Consultation

    Are there any aspects of the recommendations that need particular consideration to ensure we avoid unlawful discrimination against any group of people on the grounds of age, disability, gender reassignment, pregnancy and maternity, race, religion or belief, sex or sexual orientation?

1 Recommendations

Open for comments Open until   Request commenting lead permission

We have updated this service so that members of the same organisation can now collaborate on a joint online response.

Read our blog to learn more.

1 Recommendations

1.1

Nirogacestat should not be used to treat progressing desmoid tumours that need systemic treatment in adults.

1.2

This recommendation is not intended to affect treatment with nirogacestat 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.

What this means in practice

These are NICE's draft recommendations. If these recommendations become final, nirogacestat 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 nirogacestat is value for money in this population.

Why the committee made these recommendations

Usual treatment for progressing desmoid tumours that need systemic treatment is non-curative and aimed at symptom management. This can include chemotherapy, which is not licensed for treating desmoid tumours.

Clinical trial evidence shows that nirogacestat increases how long people have before their condition gets worse compared with placebo.

Nirogacestat has not been directly compared with chemotherapy in a clinical trial. Indirect comparisons suggest that nirogacestat is likely to work as well as chemotherapy, but this is uncertain.

There are uncertainties with the economic model. This is because of the assumptions made about how nirogacestat affects quality of life.

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