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NICE blocks second Alexion rare disease drug in a week

Says high cost and “considerable uncertainties” over long-term benefits prohibit routine NHS use

AlexionPatients with the rare inherited genetic disorder lysosomal acid lipase deficiency (LAL-D) will not be able to get routine treatment with Alexion's Kanuma, says NICE.

A final ruling from the UK's cost-effectiveness watchdog has concluded that the high cost of Kanuma (sebelipase alfa) - at almost £500,000 per patient per year - cannot be justified by its clinical benefits.

The verdict comes just days after the agency excluded two patient groups in its final determination on rare bone disorder drug Strensiq (asfotase alfa), giving the go-ahead only for perinatal- and infantile-onset hypophosphatasia and excluding children and adults with juvenile-onset disease.

LAL-D is caused when an enzyme that is responsible for breaking down fats within cells is deficient or absent, causing toxic build-up. The disease primarily affects the liver, gastrointestinal and cardiovascular systems, leading to multi-organ damage and premature death.

Alexion said it is disappointed with the Kanuma decision, noting that infants with LAL-D typically do not live to see their first birthday without treatment, while around 50% of children and adults with the disease progress to liver complications such as fibrosis or cirrhosis, with some needing a liver transplant within three years of the start of symptoms.

It said that trials of Kanuma showed that five out of nine infants survived beyond three years of age, achieving normal developmental milestones, and accused NICE of "disregarding expert medical opinion" included in an access plan developed by clinicians, patient groups and NHS England to make sure the drug is used in the most needy patients.

The company said the decision is "an injustice to patients in England and based on arbitrary cost-effectiveness measures" and if not reversed could have "devastating consequences for patients with LAL-D who may face significant suffering, multi-organ damage and potential premature death”.

After the Strensiq appraisal concluded Alexion criticised NICE for not engaging with drugmakers enough during the process to arrive at a deal on pricing and ignoring stakeholder input, but the agency's health technology evaluation centre director, Professor Carole Longson, insists that is not the case.

"Throughout this extremely complicated evaluation we have given the company many opportunities to improve the terms under which sebelipase alfa could be offered to patients," she said. "However, even their best offer to date falls far short given the considerable uncertainties about its longer term benefits and its very high cost."

The Kanuma decision has been met with consternation by the Society for Mucopolysaccharide Diseases, which represents LAL-D patients and their carers.

"We are particularly devastated by the announcement from NICE to deny babies with LAL-D access to life saving therapy," said the SMD's chief executive Christine Lavery, who added: "These babies need immediate access to treatment following diagnosis. Without treatment children will unquestionably die a cruel and inhumane death within days or weeks".

"Should a right to life be denied when there is a treatment that not only demonstrates clinical benefit but allows children to thrive, grow, meet their developmental milestones and have a normal childhood?" she asked.

The verdict shows that the UK lags behind other countries in funding medicines for patients suffering from rare and ultra-rare diseases with limited treatment options, continued Lavery.

"The NICE Highly Specialised Technology (HST) process is proving to be both inconsistent and ineffective in assessing the significant medical and human benefits of a treatment for patients with a severe and devastating rare disease for which there are no other treatment options."

Yesterday NICE also published draft guidance not recommending NHS coverage of AbbVie's Venclyxto (venetoclax), which was recently approved for treating chronic lymphocytic leukemia.

Article by
Phil Taylor

16th February 2017

From: Regulatory



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