Sebelipase alfa for treating lysosomal acid lipase deficiency that is not Wolman disease (terminated appraisal)

The Department of Health has asked the National Institute for Health and Care Excellence (NICE) to produce guidance on using sebelipase alfa in the context of national commissioning by NHS England. The Highly Specialised Technologies Evaluation Committee has considered the evidence submitted by the company and the views of non-company consultees and commentators, clinical experts, patient experts and NHS England.

This document has been prepared for consultation with the consultees. It summarises the evidence and views that have been considered, and sets out the draft recommendations made by the Committee. NICE invites comments from the consultees and commentators for this evaluation (see section 9) and the public. This document should be read along with the evidence base (the Committee papers).

The Evaluation Committee is interested in receiving comments on the following:

·      Has all of the relevant evidence been taken into account?

·      Are the summaries of the criteria considered by the Committee, and the clinical and economic considerations reasonable interpretations of the evidence?

·      Are the provisional recommendations sound and a suitable basis for guidance on the use of sebelipase alfa in the context of national commissioning by NHS England?

·      Are there any aspects of the recommendations that need particular consideration to ensure we avoid unlawful discrimination against any group of people on the grounds of race, gender, disability, religion or belief, sexual orientation, age, gender reassignment, pregnancy and maternity?

Note that this document is not NICE's final guidance on this technology. The recommendations in section 1 may change after consultation.

After consultation:

·      The Evaluation Committee will meet again to consider the evidence, this evaluation consultation document and comments from the consultees.

·      At that meeting, the Committee will also consider comments made by people who are not consultees.

·      After considering these comments, the Committee will prepare the final evaluation determination (FED).

·      Subject to any appeal by consultees, the FED may be used as the basis for NICE’s guidance on using sebelipase alfa in the context of national commissioning by NHS England.

For further details, see the Interim Process and Methods of the Highly Specialised Technologies Programme.

The key dates for this evaluation are:

Closing date for comments: Wednesday 25 May 2016

Third evaluation Committee meeting: Wednesday 22 June 2016

Details of membership of the Evaluation Committee are given in section 8, and a list of the sources of evidence used in the preparation of this document is given in section 9.