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

    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 race, gender, disability, religion or belief, sexual orientation, age, gender reassignment, pregnancy and maternity?

  • Question on Consultation

    Has all of the relevant evidence been taken into account?

  • Question on Consultation

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

  • Question on Consultation

    Are the recommendations sound and a suitable basis for guidance to the NHS?
The content on this page is not current guidance and is only for the purposes of the consultation process.

1 Recommendations

Closed for comments This consultation ended on at   Request commenting lead permission

1 Recommendations

1.1

Idebenone is not recommended, within its marketing authorisation, for treating visual impairment in Leber's hereditary optic neuropathy (LHON) in people 12 years and over.

1.2

This recommendation is not intended to affect treatment with idebenone 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 clinician consider it appropriate to stop. For young people, this decision should be made jointly by them, their clinician, and their parents or carers.

Why the committee made these recommendations

Standard care for LHON includes nutritional supplements, genetic counselling and lifestyle management advice. There are no licensed medicines for the underlying causes of LHON, so there is an unmet need for new treatments.

Evidence from a clinical trial directly comparing idebenone with standard care suggests that idebenone is no better at improving vision. But this is uncertain because the trial only included a small number of people and was short. Longer-term trials show that idebenone improves vision but, in these trials, it was not compared with standard care. An indirect treatment comparison also suggests that idebenone is no better than standard care at improving vision in the long term. Overall, it is uncertain how effective idebenone is compared with standard care, and further research is needed to estimate its potential benefits.

These clinical uncertainties are also present in the economic model. So, it is not clear what the most likely cost-effectiveness estimates are for idebenone. Further economic modelling is needed, and idebenone is not recommended.