Once again, the U.K.’s drug cost watchdogs are catching flak from the industry after issuing a restricted recommendation for Alexion’s pricey rare-disease med Strensiq.
In a final evaluation determination, the National Institute for Health and Care Excellence recommended (PDF) the drug for treating patients with perinatal- and infantile-onset hypophosphatasia under certain conditions and with a confidential discount.
Citing a “very high” cost and “significant uncertainties” about Strensiq’s long-term benefits, however, the institute’s experts decided not to recommend the med in children with juvenile-onset disease. The med also failed to win a nod for adults with juvenile-onset disease.
Alexion, maker of the drug, said it’s “extremely disappointed” with the review, adding that the recommendation will “deny the very small number of children, juveniles and adults, who are unnecessarily suffering serious life-limiting complications of HPP, access to the only proven treatment.”
Patients in the U.S., Germany and Japan can access the med, according to Alexion.
NICE “fundamentally failed to fairly and appropriately evaluate” Strensiq’s benefits in pediatric-onset patients, the company said. In the wake of the review, Alexion is “considering all potential procedural and other options available.”
Even still, the institute's decision is a partial reversal from 2015, when NICE balked at Strensiq’s price and decided on a preliminary basis it couldn’t sign off on the drug.
Priced at about £366,912 ($392,000) per patient per year in the U.K., Strensiq is among the world’s most expensive medicines. According to NICE, severe hypophosphatasia is seen in 1 out of 300,000 people in Europe, while about 1 out of 6,370 people experience milder forms of the disease.
Alexion agreed to make the drug available with a per-patient cost cap and a discount, both of which are confidential, according to NICE’s evaluation.
The company, hit recently by a Soliris trial failure, an exec shakeup and fraud allegations, is far from alone in criticizing NICE reviews. Among the companies to have aired their problems are Pfizer, AstraZeneca and Roche. Eisai even threatened legal action over a delay on its thyroid cancer med Lenvima.