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NICE backs world’s most expensive medicine

Published on 04/09/14 at 12:01am
Solaris image

In a U-turn on its previous guidance NICE is now recommending that the £330,000 rare disease drug Soliris should be made available on the NHS under a specialist funding stream.

NICE is now saying ‘yes’ to Alexion’s Soliris (eculizumab) for patients with atypical Haemolytic Uraemic Syndrome (aHUS), an extremely rare but life-threatening disease which causes inflammation of blood vessels and the formation of blood clots throughout the body.

Around 200 people in England currently suffer from the condition and this is expected to rise by around 20 each year. Soliris, the world's most expensive medicine, is currently the only drug treatment available for the condition.

Plasma therapy and dialysis were the main treatments, but both had a limited impact on managing symptoms and reducing early deaths. These treatments also have a substantial negative effect on a patient’s quality of life.

NICE had originally said ‘no’ to the drug, asking the company for more information on its manufacturing and R&D costs. Alexion has since supplied this extra information and NICE is now content to recommend the drug.

But this recommendation comes at a high price: NICE estimates that Soliris will cost the NHS up to £58 million in the first year, rising to £82million after five years, with an average patient cost of £330,000 per year.

NICE chief executive Sir Andrew Dillon explains: “From the available evidence and from the testimony of clinicians and patients, families and carers, it is clear that [Soliris] is a significant breakthrough in the management of aHUS.

“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.”

NICE’s committee says it felt that the budget impact of recommending Soliris for aHUS in relation to the substantial benefits it offers, would be lower if the potential for dose adjustment and stopping treatment was taken into account.

Therefore, the draft guidance recommends that Soliris should only be funded if important conditions are met.

These include co-ordinating the use of Alexion’s drug through an expert centre and 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.

This programme also needs to develop protocols for starting and stopping treatment with the drug for clinical reasons, and introduce a research programme to collect data to evaluate when stopping treatment or adjusting the dose of the drug might occur.

But NICE says more cost savings will still need to be made in the future: “Given that the budget impact of [Soliris] for treating aHUS will be considerable, the draft guidance also recommends that NHS England and the company should consider what opportunities might exist to reduce the overall cost of [Soliris] to the NHS.”

AGNSS

NICE’s new role to assess ultra-rare orphan drugs for use on the NHS started in 2013, when it replaced the Advisory Group for National Specialised Services (AGNSS).

AGNSS had been in place since 2010 but the UK government decided to remove this body and hand over its reins to NICE – a decision questioned by many rare disease patient groups given NICE’s history with rejecting high cost medicines.

Speaking to Pharmafile recently on the topic of its new remit, Professor David Haslam, the chairman of NICE, admits the body is still in a ‘learning mode’ when it comes to just how it should assess these drugs.

Haslam said: “I think we are at the beginning of a really interesting new model that we’re all going to have to develop – that includes industry and regulators as well as NICE – to look at these much smaller patient populations.”

Soliris has been something of a guinea pig for its new role, and NICE has not been able to use its established cost-effectiveness formula QALY (quality-adjusted life years) in the same way as it does with other medicines, as it would never allow a drug that cost £330,000 to be recommended for NHS funding.

So NICE has had to develop a new methodology whilst assessing the medicine, which involved using an updated form of QALY to take into the high costs, but low number of patients, and with the specialised silo budget in place for such medicines.  

Alexion says that under this evolved process, its medicine produced just over 25 additional QALYs per patient compared with standard care.

NICE points out however, that this figure is markedly higher than that calculated by its own committee. But it concedes that whichever QALY figure you look at, the gains are “of a magnitude that is rarely seen for any new drug treatment”.

Alexion’s chief executive Leonard Bell says of the latest positive decision: “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.”

Budgets and costs

Despite being appraised by NICE the drug is in fact already being paid for by the NHS through an interim policy commissioned last year.

NICE confirmed today that this interim policy will remain in place pending the final outcome of NICE’s appraisal, which is scheduled for 9 October.

The money for the drug comes out of NHS England’s specialist commissioning budget – a budget that is under increasing pressure from the Cancer Drugs Fund, which is now taking £280 million a year from its total allocation of £544 million.

The amount spent on so-called high cost drugs like Soliris was £156 million for 2013/14.

Ben Adams  

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