NICE says yes to Soliris – but warns on price
Soliris – the most expensive drug in the world – has been recommended by NICE to treat the rare disease aHUS, finally ending a review lasting three years.
After a long deliberation, a special committee at NICE has decided that Soliris (eculizumab) should be used for patients with atypical haemolytic uraemic syndrome (aHUS), a life-threatening disease which causes blood clots throughout the body.
Marketed by US firm Alexion, Soliris is known as the most expensive drug in the world thanks to its US price of $536,629 per patient per year. Remarkably, NICE estimates that the drug costs up to £340,200 ( more than $571,000) per adult patient per year – which (at current exchange rates) would make the UK price even higher than in the US.
The disease affects just 200-or-so patients in England, and there are concerns about it consuming a disproportionately large part of the budget - NICE estimates it would cost the NHS a total of £58 million in the first year, rising to over £80 million in the fifth year.
NICE has nevertheless now issued a preliminary recommendation for the drug, which has been greeted with relief by patient groups and Alexion.
However NICE has warned that it sees no reason why Soliris should have such a high price – even among rare disease drugs. This comes after NICE earlier this year requested information from Alexion on its R&D and other costs to justify its pricing.
Despite these inquiries, NICE has no power to demand pharma company's cut prices. It can, however, recommend a number of measures to limit costs.
Its new recommendation says dose adjustment and starting and stopping criteria can be used to ensure the publicly-funded national health service gets value for money.
The measures include:
• co-ordinating Soliris use through an expert centre
• putting in place systems for monitoring how many people are diagnosed with aHUS, how many receive the drug, at what dose and for how long
• Protocols to be developed for starting and stopping treatment for clinical reasons
• Introduce a research programme to collect data to evaluate when stopping treatment or adjusting the dose of the drug might occur
While these recommendations match closely those of patient group aHUS UK and Rare Disease UK, Alexion has issued its own warning about NICE's conditions. Specifically, it says that directing doctors on what stopping criteria to use is outside of NICE's remit, and that it contradicts Soliris' licensed indication.
Commenting on the draft guidance, NICE chief executive Sir Andrew Dillon said: "Eculizumab radically improves the quality of life of the small number of people with aHUS. Until it became available, people with aHUS were at risk of kidney failure needing dialysis, other organ failure, and early death.
"Plasma therapy and dialysis were the main treatments, both of which had a limited impact on managing symptoms and reducing early deaths, but a substantial negative effect on a patient's quality of life.
"From the available evidence and from the testimony of clinicians and patients, families and carers, it is clear that eculizumab is a significant breakthrough in the management of aHUS. The drug offers people with the disease the possibility of avoiding end-stage renal failure, dialysis and kidney transplantation, as well as other organ damage.
"The drug is, however, very expensive. In making its decision the independent Evaluation Committee needed to take into account the total fixed budget for highly specialised services as a whole and how it is allocated.
These cost concerns led the committee to put forward the recommendations on monitoring how many patients receive the drug, at what dose and for how long, as well as the starting and stopping criteria and the research programme.
Alexion warns on NICE remit
Responding to the announcement, Alexion's chief executive Leonard Bell said:
"We are pleased that NICE has once again confirmed that patients with aHUS are at constant risk of sudden, progressive and life-threatening damage to vital organs including the kidney and other organs, and that eculizumab is a significant breakthrough for patients with this devastating disorder."
Turning to the question of discontinuing treatment, Bell commented that the EMA-approved label specifically directs that treatment is recommended to continue for the patient's lifetime, unless the discontinuation of eculizumab is clinically indicated.
He said: "We believe that it is important that NICE work within its remit and that decisions regarding continuation of eculizumab should be made by the treating physician based on best clinical judgment."
The company says it will contribute specific comments to NICE ahead of the final decision being made after a public meeting on 9 October.
Leonard Bell said the company will address NICE's conditions and look forward to working with the NHS to "ensure equity such that patients with aHUS in England, like patients with PNH in England [the drug's other licensed indication] have equal and sustained access to this life-transforming therapy."
Because of the life-threatening nature of aHUS, eligible patients with aHUS in England have been receiving the drug for some time.
New and existing aHUS patients currently receive Soliris through an interim policy commissioned by NHS England last year. This policy will remain in place pending the final outcome of NICE's appraisal.
Implications for high cost drugs
The decision on Soliris has particular significance, because it is the first treatment to be reviewed by NICE's new Highly Specialist Technology (HST) Evaluation Committee. NICE observers in pharma will be glad to see the approval, but its positive decision won't mean that cost issues will go away.
NHS England, is the health service body which controls the budget for specialist drug spending, and is currently struggling to control an overspend predicted to hit £330 million this financial year. It will be exerting pressure on pharma companies in rare diseases to control costs, just as it recently promised to do in relation to high cost cancer treatments.
These concerns will also affect the next drugs the HST committee looks at – the next being two enzyme replacement therapies: asfotase alfa – another Alexion drug and BioMarin's Vimizim (elosulfase alfa).
