NEWS: Climb and The Brittle Bone Society submit appeal on final NICE decision for HPP

Climb has appealed the NICE Review Committee decision not to approve the treatment of severely affected juvenile and adult patients with the treatment asfotase alpha.


Whilst Climb is extremely pleased that the treatment will be available for all perinatal and infantile cases of the disorder under the Managed Access Agreement criteria, they are disappointment with the final decision.

The appeal is based on some procedural issues. Climb has fully engaged in this process at every stage driven by the commitment to ensure that the patient experience of this ultra-rare disease has been represented, which is vital to NICE for decision making.

However Climb did not have a place on the panel to represent juvenile patients at the final meeting. This may have affected the ability of the panel to make a decision. There was no evidence that NICE have taken into account some of the crucial information previously submitted and would have welcomed the opportunity to present these key areas again at the final meeting.

UK patient support charity, The Brittle Bone Society are pleased that treatment will be available for all perinatal and infantile cases of the disorder HPP under the Managed Access Agreement criteria but are minded to support comment alongside Climb and share their disappointment with the final outcome not to approve the treatment of severely affected juvenile and adult patients with the treatment asfotase alpha.

The Brittle Bone Society were consultees in this process. As advocates of the rare disease community (albeit focussing their support primarily for people with Osteogenesis Imperfecta) it is imperative that alongside our colleagues at Climb we continue to seek assurances from NICE to take full account of clinical and professional expertise and testimony in these matters.

From the report findings it looks like healthcare professionals stated clear benefit for children with juvenile onset HPP and that treatment would prevent avoidable suffering of these children. The Charity has engaged where possible in this process and hopes for a better outcome for the HPP community.

Juvenile patients who would benefit would be suffering considerably with the disease and it would be impacting on their quality of life, specifically their mobility and pain levels as set out in the stringent Managed Access Agreement criteria. Taking into the account the range of clinical experts and also the patient consultation carried out, Climb believe that the Managed Access Agreement is robust and its still not clear why NICE has not taken into account the clinical expertise offered. Data around juvenile patient numbers seems to not have been taken into account. The Climb appeal has been supported in writing to by all the clinicians involved.

The clinical expertise for adults was absent from the final meeting. This disease in adults causes immense suffering due to constant debilitating pain and reduced mobility. Over a length of time without treatment this causes other significant health problems.

The medical information in this area is limited but believed by our experts that the treatment could significantly improve the lives of these adults. For anyone who does not respond to treatment, the treatment will not be continued. Climb believe that the expert views on this were not taken into account or given the opportunity to present this information for the NICE panel’s consideration.

An oral hearing to consider this appeal was due to take place on Thursday 13th April 2017.

Prior to the appeal hearing NICE agreed to consider additional information provided by Alexion (the manufacturers of asfotase alpha).  In order to allow the NICE Highly Specialised Technologies committee to undertake this process the scheduled appeal hearing  was cancelled, and the additional evidence will be reviewed to ascertain if this alters the recommendation for this product.

For more information, please contact Climb on 0845 241 2173.