NICE approval for restricted-use hypophosphatasia therapy

pharmafile | September 23, 2016 | News story | Medical Communications, Sales and Marketing Alexion Pharmaceuticals, Strensiq, hypophosphatasia 

The National Institute for Health and Care Excellence (NICE) has issued draft guidance recommending the use of Alexion Pharmaceutical’s Strensiq (asfotase alfa) for children with perinatal- and infantile-onset hypophosphatasia.

The recommendation comes after the institute rejected the rare metabolic bone disease treatment on the basis that its benefits were “too uncertain to justify its high cost.” NICE is now providing Stensiq to the NHS with a proposed cost cap and recommends it as part of a five-year managed access agreement which will facilitate more information to be gathered on treatment duration and dosage “before the guidance is reviewed and a further decision made on whether funding should be continued.” NICE has indicated the annual price of therapy stands at £367,000.

Carole Longson, director of NICE’s health technology evaluation centre, said “the committee concluded that [Strensiq] improved the probability of survival in perinatal- and infantile-onset hypophosphatasia compared with best supportive care. The committee also accepted that [Strensiq] was likely to be clinically effective across a range of outcomes, such as reducing the need for respiratory support and the severity of rickets for people with perinatal- and infantile-onset hypophosphatasia.” 

But the manufacturer was “disappointed” that the guidance does not cover patients with juvenile-onset hypophosphatasia. The company stated: “we are also disappointed that NICE disregarded the proposed managed access agreement, which was developed in collaboration with expert physicians, the patient advocacy community, as well as NHS England, to ensure access for patients most likely to have a clinical benefit from treatment with Strensiq.”

“By disregarding the consensus position developed directly with treating physicians and patient organisations, Alexion believes the NICE assessment process has fundamentally failed to appropriately evaluate the benefits of Strensiq for patients with paediatric-onset hypophosphatasia, and is delaying timely access to treatment for the small number of patients with [the disease] in England.”

Matt Fellows

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