EXCLUSIVE: NICE delays rare disease ruling again

A ruling on rare disease drug Soliris has been delayed once again, leaving patients in England baffled and concerned.

The NHS cost effectiveness watchdog NICE was due to issue its decision on Soliris last week, but announced instead a further delay; patients with the disease have already waited 18 months for a decision.

Soliris (eculizumab) is the only treatment available for atypical haemolytic uremic syndrome (aHUS), which is currently diagnosed in just 140 patients in England – making it extremely rare, an ‘ultra orphan’ disease. The drug, marketed by Alexion, is widely acknowledged as being life-changing and even life-saving – but it is also one of the most expensive drugs in the world.

Soliris costs around £250,000 per patient a year, and patients have to stay on the treatment for life. There are now signs that the government could seek price discounts before it gives the final yes to the drug.

A landmark for rare disease

The ruling isn’t just important to patients with aHUS, but also to all other rare disease sufferers, because Soliris is NICE’s first orphan disease appraisal.

There is great uncertainty about exactly what processes NICE will use to assess drugs for orphan diseases. There are fears that orphan medicines could get bogged down in drawn-out rows over affordability which have affected patient access to other NICE appraised drugs.

While campaigners say they remain optimistic that Soliris will be approved, the slowness of the new decision-making process may well be replicated in subsequent reviews. Moreover, NICE may take a hard line on the high cost of orphan medicines, which could mean negotiations on price will have to be conducted with pharma companies for all orphan drug appraisals.

“The Soliris appraisal is NICE’s first ever orphan drug appraisal – and is being watched by everyone with an interest in rare diseases”

Alastair Kent, chair of the Rare Diseases UK patient group expressed his bafflement at the delay, and said he had been given no indication as to why the decision had been postponed.

“It is deeply frustrating that you now have a situation where it is getting on for 18 months that patients have been waiting…you do have to wonder how much longer they [NICE] need to make up their minds.”

He stressed that he was ‘basically optimistic’ that NICE would approve the drug’s use, but admitted to some concerns.

“I can only hope that they aren’t trying to unduly restrict access to the drug,” he said.

Because of the life-saving nature of the drug, NHS England has interim arrangements in operation which ensure all eligible aHUS patients are already receiving the drug – a situation which makes it virtually inconceivable that NICE will reject use of the drug.

Nevertheless, NICE’s latest delay could mean it is preparing to query the Soliris price tag.

Asked if he believed that Alexion should lower the price of Solaris, Alastair Kent acknowledged the drug was expensive but said “a high price can be a fair price,” given its life-saving qualities and the difficulty of developing drugs for rare diseases.

However, faced with increasing pressure on a static NHS budget, companies like Alexion with rare disease drugs could find themselves having to negotiate with NHS England on price. Alastair Kent agreed this process, familiar from ‘mainstream’ NICE appraisals, may become a feature of rare disease reviews as well. He pointed to a new approach emerging in Scotland where such negotiations are already happening, and said he would support any talks that would help drugs find their way ‘through the maze’ of reimbursement.

Referred to NICE because of price concerns

Soliris has already been recommended by an expert committee once before, when the AGNSS panel recommended its use in June 2012. The AGNSS committee concluded that eculizumab for aHUS was: “a life-saving and life-transforming product that despite the very high cost should be available in England to patients with aHUS.”

All but one of the committee members voted in favour of recommending it, saying it would save and improve the lives of children and adults with the condition.

It added, however: “They [the panel] were unanimous in urging future negotiations to cap the cost of treatment for aHUS as patient numbers grow, both for aHUS and possible new indications.”

“Soliris had been approved by the now defunct AGNSS group – but it stressed concerns about price”

However government minister Earl Howe decided that this recommendation would not be followed, citing the cost concerns, and asked NICE to conduct its own entirely new review of the drug.

AGNSS was then abolished on 31 March 2013 as part of wider NHS reforms, which meant responsibility for ‘Highly Specialised Technologies’ appraisals now passed over to NICE.

The NICE committee will undoubtedly be aware that this Friday is Rare Disease Day, an awareness raising day for rare diseases worldwide.

If NICE has any bad news for aHUS patients, it may well wait it out another week before making its announcement, to avoid highlighting the drug access issues which is one of the hot topics of awareness day.


First test case of NICE approach to Highly Specialised Technologies – Soliris (eculizumab)



About the author:

Andrew McConaghie is an experienced journalist and pharmaphorum’s new Managing Editor, Feature Media. He has been writing about the pharmaceutical industry and NHS since 1999 and will be writing regular exclusive news and insights from the sector for pharmaphorum.

Andrew can be contacted at andrew@pharmaphorum.com