Draft NICE decision rejects Respreeza for incurable, genetic disorder

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 enzymes.

Therefore, people with the condition are at high risk of the chronic lung condition emphysema as a result of infections and environmental toxins, including tobacco smoke and pollution.

Current therapies, such as bronchodilators and inhaled corticosteroids, strive to delay the worsening of emphysema and manage its symptoms but do not target the cause of the condition.

Respreeza (human alpha-1-proteinase inhibitor) aims to supplement the missing protein in people with A1PI deficiency. It is a lifelong theray costing £57,200 per patient per year on average, and it is estimated that around 200 to 600 people could be eligible for treatment in England.

According to NICE, while the available evidence suggests that human A1PI slows progression of lung tissue damage compared with placebo, albeit with "a great deal of uncertainty," and that it may improve survival, there is no evidence that it improves lung function or quality of life.

The appraisal committee concluded that cost-effectiveness estimates for Repreeza are "much higher than the range NICE normally considers acceptable for highly specialised technologies," coming in at over £8 million per quality-adjusted life year (QALY) gained.

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