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Beremagene geperpavec for treating skin wounds associated with dystrophic epidermolysis bullosa

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1 Recommendations

1.1

Beremagene geperpavec should not be used to treat skin wounds associated with dystrophic epidermolysis bullosa (DEB) in people of all ages who have mutations in the collagen type 7 alpha 1 chain (COL7A1) gene.

1.2

This recommendation is not intended to affect treatment with beremagene geperpavec 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 babies, children or young people, this decision should be made jointly by the healthcare professional, the child or young person if appropriate, and their parents or carers.

What this means in practice

These are NICE's draft recommendations. If these recommendations become final, beremagene geperpavec 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 the available evidence does not suggest that beremagene geperpavec is value for money in this population.

Why the committee made these recommendations

Usual treatment for skin wounds associated with DEB is birch bark extract and best supportive care, which includes wound management, surgery and pain management.

Clinical trial evidence shows that beremagene geperpavec with best supportive care closes wounds more quickly than placebo with best supportive care.

Beremagene geperpavec has not been directly compared in a clinical trial with birch bark extract. An indirect comparison suggests that it is likely to be more effective, but the extent of this is uncertain because the studies used different ways to measure results.

There are also uncertainties in the economic model because of the assumptions about:

  • who would have beremagene geperpavec in clinical practice

  • how the wounds develop, close and progress

  • whether people would have beremagene geperpavec at home, instead of in a specialist clinic, and how that would be delivered

  • whether beremagene geperpavec reduces non-skin-related complications of DEB

  • how much beremagene geperpavec a person needs over their lifetime.

Even when considering the condition's severity, and its effect on quality and length of life, the most likely cost-effectiveness estimates are substantially above the range that NICE normally considers an acceptable use of NHS resources.

Collecting more evidence during a managed access period may resolve some uncertainty in the evidence but the company has not submitted a managed access proposal. So, beremagene geperpavec should not be used.