22 September 2016 - NICE recommends ‘lifeline’ drug for babies and young children, in draft guidance.

NICE has published draft guidance recommending the drug asfotase alfa for children with perinatal- and infantile-onset hypophosphatasia – a very rare inherited condition affecting between one and seven babies each year in England.

Described as a ‘step-change’ in the management of the condition, offering a ‘lifeline’ for people that could enable them to have a good quality of life but without which they would not survive, the £367,000 per patient per year drug is the first therapy that specifically targets the underlying cause of hypophosphatasia.

The draft guidance recommends asfotase alfa for children with the condition as part of a managed access agreement and when the company provides the drug with the proposed cost cap which will limit the cost the NHS will have to pay per patient.

The five-year period of the agreement will allow more information to be gathered on how long treatment should continue, and when treatment could be stopped or the dose reduced before the guidance is reviewed and a further decision made on whether funding should be continued.

The draft guidance also states that the company should provide further opportunities to reduce the short-term cost of asfotase alfa to the NHS.

Read NICE press release