UK Charities - The Brittle Bone Society and CLIMB have been patient organisation consultees in the NICE process – for Afostase alfa for people with HPP.
Climb represents patients of ALL ages with HPP from new-borns right through to adults. Climb has represented all patients’ experiences during this process by having direct contact and communication with those living with the condition. It is our aim to continue to do this in view of the latest outcome. Climb will continue to work in partnership with all stakeholders to get the possible results for patients and their families.
Brittle Bone Society whilst working with their own OI community and other rare bone groups are aware of hopes to establish access to new treatments and therapies as they become available, and have learned that the countries of Germany and USA have approved use of this treatment.
The interim decision published 22 Sept is not a final decision and there are opportunities for patients and families to comment either via a patient organization or directly to NICE
The evaluation committee has published an interim decision and is interested in receiving comments by 13th October before they reach their final decision.
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 asfotase 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?
The full papers can be accessed here
Note that this is not NICE's final guidance on this technology. The recommendations may change after consultation. https://www.nice.org.uk/news/article/nice-to-recommend-new-drug-to-treat-rare-inherited-bone-disorder-in-children