NICE Recommends Hypophosphatasia Drug

The National Institute of Healthcare Excellence (NICE) issued a draft guidance on Sept. 22, 2016 recommending the use of asfotase alfa as a treatment for children with perinatal- and infantile-onset hypophosphatasia. Hypophosphatasia is a rare inherited condition, NICE said in a press statement, and only affects between one and seven babies each year in England.

The drug is marketed by Alexion Pharmaceuticals in the United Kingdom and is expected to cost £367,000 (approximately $476,386.18) per patient per year. In December 2015, Reuters reported the organization turned down the approval of asfotase alfa, because they believed it was too expensive. But the agency changed its tune on Thursday, saying in a press announcement the drug would be a “‘lifeline’ for people that could enable them to have a good quality of life but without which they would not survive.”

NICE did recommend that Alexion provide opportunities to lower the cost of the treatment in the future. The organization also recommended a cost cap, limiting the amount the NHS will have to pay her patient. Comments on the draft guidance are open until Oct. 13, 2016.

Source: NICE