From August 2017, children and adults with a rare inherited metabolic bone disease called Hypophosphatasia (HPP) have been able to access a potentially life-enhancing drug called asfotase alfa (Strensiq).
The drug asfotase alfa was previously only recommended for use in babies by NICE in draft guidance, as evidence showed it could be life-saving. The Managed Access Agreement, alongside the new guidance in 2017, meant that access to the drug was broadened to infants, children, and adult patients with paediatric-onset HPP, who experience the most disabling symptoms and who are expected to benefit most from therapy. The agreement between, NICE, NHS England and Alexion runs for 5 years and enables time for further information and data to be collected.
Metabolic Support UK has been the patient organisation consultee in this process – representing people of all ages with HPP and working closely with Soft Bones UK. As part of this, we have held a consultation with those who have access to the drug via the Managed Access Agreement, and from this we have identified a clear need for some patient information to be developed to clarify what the terms of the agreement are and what they mean in reality for patients and their families.
With input from NICE, NHS England, Alexion and Soft Bones UK, we are pleased to be able to present our patient information on the asfotase alfa Managed Access Agreement for treating Hypophosphatasia. We would like to thank all those who have taken the time to contribute feedback to us regarding the drug and the Managed Access Agreement both to Metabolic Support UK and Soft Bones UK. We will continue to collaborate to ensure that we continue to represent the voices of those we support.
You can download the document here: Managed Access Agreement for HPP v1.1