The National Institute for Health and Excellence has advised the NHS not to use Spinraza for the rare genetic disorder spinal muscular atrophy (SMA). In its assessment it said that there was a ‘substantial benefit’ for the drug, but the commiteee felt there were ‘significant uncertainties, particularity around its long term benefits’ partnered with Spinraza’S extremely high cost, means it would not be recommended as a cost effective treatment option.
However, NICE has said that Biogen intendeds to develop a proposed managed access programme that might adresss uncertainties in the evidence and reduce financial risk to the NHS.
The Scottish Medicines Consortium has approved funding for four newly licensed medicines.
Tesaro UK’s Zeluja was accepted for use for advanced ovarian cancer. In trials, it was found to cut the risk of disease progression or death by 73%
Roche’s Alecensa, was approved for its treatment of a rare type of advanced lung cancer. It has been found by Roche to decrease the risk of progression of the disease by almost half and reduce the risk of tumours in the central nervous system by 84%
Sweden’s Orphans Ravicti was approved for its long term treatment of urea cycle disorders in adults and children.
Pharmings Ruconest was accepted for the treatment of patients with the rare condition acute hereditary angioedema, which can cause painful and dangerous swelling anywhere in the body.
However it wasn’t all approvals for NHS Scotland with BioMarins Kuvan being rejected for concerns about long term benefits and cost effectiveness. As well as Vifor Pharmas Veltassa for the treatment of hyperkalaemia being rejected for cost effectiveness issues
Eisia and MSD’s Lenvima has been approved in the USA for the first line treatment of patients with unresectable hepatocellular carcinoma. The decision was based on results of the REFLECT trial in which Lenvima showed superiority compared to Bayers Nexavar.
NICE has stuck with its position that Roche’s Perjeta is not cost effective enough to be used by the NHS, post surgery to prevent breast cancer recurrence. The institute did not recommend Perjeta, it believes that further consultation is needed ‘to give people the opportunity to commit on updated cost effectiveness estimates’ which takes into account the costs of administering the drugs and the impact of cheaper biosimilar trastuzumab on reducing the overall cost of the Perjeta regimen. Evidence showed that adding Perjeta to the already tried regimen of trastuzumab and chemotherapy after surgery only made ‘ a small difference in the proportion of people who remained free of invasive disease and this effect itself was uncertain’. NICE also stated that the incremental cost effectiveness ratios for the drug are still likely to be higher than would normally be considered a good use of NHS resources.
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