Soliris (eculizumab) was recommended by the National Institute for Health and Care Excellence (NICE) – a body which governs drugs used by the National Health Service in England and Wales – in September following a review and yesterday the body published its draft guidance for funding the mAb which treats atypical Haemolytic Uraemic Syndrome (aHUS).
There have been delays in reaching this point in part due to the high cost of the drug made by Alexion Pharmaceuticals. At £3,150 ($4,000) per 30ml vial, the cost of a full course of Soliris for an adult is estimated to be $340,000 by NICE and is up to 30 times more expensive than other mAbs. NICE adds it expects the drug to cost the UK £58m in its first year.
However, Soliris’s price tag pales in significance compared to Dutch Biotech firm UniQure‘s Glybera (alipogene tiparvovec), a gene therapy designed to restore the LPL enzyme activity required to enable the processing, or clearance, of fat-carrying chylomicron particles formed in the intestine after a fat-containing meal in patients with for lipoprotein lipase deficiency (LPLD).
Highly Specialised Technologies programme
Glybera was put on sale this week in Germany at approximately $1.4m, eclipsing Soliris as the world’s most expensive treatment. While NICE has not yet been asked to evaluate Glybera, Biopharma-Reporter.com asked spokesman Phil Ranson how the regulator is adjusting to an emerging range of biologics and new therapies that challenge healthcare budgets due to complex technology and manufacturing processes.
“NICE has set up a separate programme – the Highly Specialised Technologies programme - to evaluate high cost drugs to treat very rare conditions,” he told us. “The methodology used to evaluate these drugs differs in a number of important respects from that used to appraise mainstream drugs.”
He continued, adding a range of criteria are taken into account when considering the benefits and costs of highly specialised technologies:
“These include the vulnerability of very small patient groups with limited treatment options, the nature and extent of the evidence, and the challenge for manufacturers in making a reasonable return on their R&D investment because of the very small populations treated.”