Innovative Deal Between NHS England, NICE and Manufacturer Alexion Allows Patient Access to Rare Disease Drug
Hypophosphatasia (HPP) is a rare inherited bone condition which is often fatal in baby and debilitating in older children and adults. The disease can lead to bone deformities and affect 1 per 6,370 of the population. The drug asfotase alfa was previously only recommended for use in babies by NICE in draft guidance, as evidence showed it could be life-saving.
A new ‘managed access agreement’ between NHS England and manufacturer Alexion, alongside new draft NICE guidance, will broaden access of asfotase alfa to infants, children and adult patients with paediatric-onset HPP, who experience the most disabling symptoms and are expected to benefit most from therapy. The deal is considered novel because it is a value-based risk sharing agreement to provide wider cost-effective access for patients, informed by their first-hand experience of the ongoing impact that treatment is having on their health and quality of life.