NICE has rejected CSL Behring’s alpha1-proteinase inhibitor Respreeza for treating genetic emphysema in draft guidance, saying that it would cost a massive £8 million to give a patient a year of quality life with the treatment.
NHS England’s cost-effectiveness watchdog, the National Institute for Health and Care Excellence (NICE), issued a first draft document saying that it did not currently recommend the drug.
A1PI deficiency is an incurable, rare and life-limiting genetic disorder in which a lack of the protein A1PI makes the body, especially the lungs, vulnerable to attack from its own infection-fighting enzyme, neutrophil elastase.
The enzyme is released by neutrophils and macrophages to fight infections but if it is uncontrolled, it can damage tissue – particularly the lungs – if there insufficient inhibitor, alpha-1 antitrypsin.
This means that individuals with A1PI deficiency are at high risk of the chronic lung condition emphysema as they are more likely to succumb to infections and environmental toxins such as tobacco smoke and pollution. The condition can also affect the liver.
In the guidance NICE stated: “The available evidence, although associated with a great deal of uncertainty, suggests that human A1PI slows progression of lung tissue damage compared with placebo, and may improve survival. However, there was no evidence that it improves lung function or quality of life.
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