Soliris (eculizumab) treats atypical Haemolytic Uraemic Syndrome (aHUS) - a disease which forms blood clots in small blood vessels that can lead to organ failure - and its recommendation by the National Institute for Health and Care Excellence (NICE) will see it funded by the National Health Service (NHS) in England and Wales.
The guidance issued this week is the first to fall under NICE’s highly specialised technologies programme which evaluates whether increasingly niche and expensive biologics should be paid for by the UK taxpayer.
But despite the endorsement, NICE did not receive justification of the manufacturing reasons behind the high price-tag price tag of Soliris from Alexion as demanded by the guidance committee back in March last year.
“The Committee was made aware of the annual costs of a range of other treatments that are available through nationally commissioned specialised and highly specialised services,” the guidance says. “However, it was not provided with information that explains the difference between the costs of developing and manufacturing eculizumab for aHUS and those costs for other treatments.”
It continues to say Alexion told the Committee the cost per patient of treatments for very rare diseases are high because “there are only a small number of patients from whom to recover the research, development and manufacturing costs.
“The Committee concluded that it had not been presented with sufficient justification for the high cost per patient of eculizumab in light of the manufacturing, research and development costs of a medicinal product for the treatment of a very rare condition.”
Soliris costs costs £3,150 per 30 ml vial and NICE predicts the budget impact for the first year would be £57.8m, based on a treatment costing £340,200 per adult patient.
But NICE’s highly specialised technologies programme is also assessing UniQure’s gene therapy drug Glybera (alipogene tiparvovec) for lipoprotein lipase deficiency (LPLD) which is already on sale in Germany for a staggering $1.4m per patient.
As to whether this week's recommendation has set the precedent for other biologics to be recommended without a full justification of manufacturing costs, a NICE spokesperson told this publication: "We will be reviewing the interim methods for evaluating these technologies shortly and this will be one of a number of aspects we will look at."
Soliris will be available for the estimated 200 people who suffer from aHUS in England,
One stipulation of the Committee’s approval of Soliris is the exploration of cost-saving opportunities through adjusted doses and stopping treatment, where necessary.
“Eculizumab should be funded only if important conditions are met, including the development of rules for starting and stopping treatment for clinical reasons,” said NICE Chief Exec Sir Andrew Dillon, and in order to control the price the following systems should be put in place:
- coordination of eculizumab use through an expert centre
- monitoring systems to record the number of people with a diagnosis of aHUS and the number who have eculizumab, and the dose and duration of treatment
- a national protocol for starting and stopping eculizumab for clinical reasons
- a research programme with robust methods to evaluate when stopping treatment or dose adjustment might occur