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    Afamelanotide for treating erythropoietic protoporphyria

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    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 and evidence from patients and clinical experts show that improvements in symptoms and quality of life would be of great importance to them. But 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 is highly uncertain.

    The cost-effectiveness analyses for afamelanotide are very challenging. Based on the available evidence, the cost-effectiveness estimates are all higher than the range normally considered acceptable for highly specialised technologies. The benefits of afamelanotide may not have been captured adequately in the formal analysis. But, even when the most optimistic scenario based on additional evidence from stakeholders is considered, estimates of cost effectiveness are still higher than those considered acceptable for highly specialised technologies. So, 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.