Life saving drug for rare bone disease available

NICE has published draft guidance recommending the drug asfotase alfa for people with a rare inherited bone disease, after an agreement between NHS England, NICE and manufacturer Alexion.

The drug, also called Strensiq, will help patients with paediatric-onset hypophosphatasia, an extremely rare inherited condition which affects between one and seven babies each year. Strensiq is the first therapy that specifically targets the underlying cause of hypophosphatasia.

Ann improved deal on price and access to the drug means NICE can now widen the group of people eligible for treatment on the NHS, after a five-year managed access agreement reduced the cost of the drug to the NHS and will enable people with the highest unmet need to be identified.

Carole Longson, director of the centre for health technology evaluation at NICE, said: “Asfotase alfa is an important development in the treatment of this devastating condition that has been shown to save lives and prevent or delay its progression. However, up until now the committee felt the benefits of the drug were too uncertain in the whole population in relation to its very high price for them to be able to recommend it for any but the most seriously affected.

“The new deal, which includes a managed access agreement, between the company and NHS England means that people with the greatest clinical need for treatment can be identified and the costs and risks to the NHS have been reduced. We are very pleased therefore to be able to recommend it as an option for treating paediatric-onset hypophosphatasia.”