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

    The content on this page is not current guidance and is only for the purposes of the consultation process.

    Afamelanotide for treating erythropoietic protoporphyria

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

    1 Recommendations

    1.1 Afamelanotide is not recommended, within its marketing authorisation, for preventing phototoxicity in adults with erythropoietic protoporphyria (EPP).

    1.2 This recommendation is not intended to affect treatment with afamelanotide 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.

    Why the committee made these recommendations

    EPP is a condition in which exposure to light causes severely painful and debilitating reactions in the body. There is no treatment, and the only way to avoid reactions is to avoid light. EPP has far-reaching effects on the lives of people with the condition and their families.

    There is some evidence from clinical trials that afamelanotide provides benefits for people with EPP. Testimonies from patients and clinical experts show that improvements in symptoms and quality of life would be of great importance to them. However, it is very difficult to measure the effects of the condition and treatment, and although afamelanotide is an effective treatment the size of the benefits it provides has not been quantified.

    The cost-effectiveness analyses for afamelanotide are very challenging. Based on the best available evidence, the cost-effectiveness estimates are all very much higher than the range normally considered acceptable for highly specialised technologies. Although the full benefits of afamelanotide may not have been captured, it is not possible to conclude that afamelanotide provides appropriate value for money.

    Taking into account all of the evidence and factors affecting the decision, afamelanotide is not recommended for use in the NHS.