human alpha-1 proteinase inhibitor (Respreeza)

following a full submission assessed under the orphan equivalent process:

human alpha1-proteinase inhibitor (Respreeza®) is not recommended for use within NHS Scotland.

Indication under review: For maintenance treatment, to slow the progression of emphysema in adults with documented severe alpha1-proteinase inhibitor (A1-PI) deficiency.

Treatment with human A1-PI for two years reduced the rate of lung density loss compared with placebo; however, there is a lack of robust evidence concerning the clinical relevance of this outcome. No improvement in pulmonary exacerbations, lung function or quality of life was demonstrated.

The submitting company did not present a sufficiently robust clinical or economic analysis and in addition their justification of the treatment’s cost in relation to its benefits was not sufficient to gain acceptance by SMC.
This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.