The decision by the UK's cost watchdog to reject Novartis' Gilenya, the first pill to treat multiple sclerosis, has been greeted with dismay by the company and patient groups.

In provisional draft guidance published this morning, the National Institute for Health and Clinical Excellence has decided against recommending payment by the National Health Service for Gilenya (fingolimod). The agency claims that "there are uncertainties over its clinical effectiveness and, based on the available evidence, it would not be a cost effective use of NHS resources".

NICE acknowledges that Gilenya can reduce the number of relapses in some people who have highly active relapsing-remitting multiple sclerosis (RRMS). However, it also claims it is "unclear how much the drug may help the specific groups of people for whom it is licensed – ie adults with RRMS who experience at least one relapse in a year despite being treated with beta interferons, and adults with rapidly-evolving severe RRMS who experience two or more disabling relapses regardless of their treatment. The Institute goes on to say the evidence submitted by Novartis mainly looked at a subgroup of the former.

NICE criticises comparator

As well as this, NICE’s independent committee of experts argues that it "could not determine how much fingolimod reduces the rate of relapses compared with some of the treatments already available". This is because Novartis only submitted evidence which compared Gilenya with a placebo, and Biogen Idec's Avonex (beta interferon 1a) "that is not believed to be widely prescribed in the NHS".

Rather, it claims that the submission should have included a comparison of Gilenya with other beta interferons and with Biogen/Elan Corp's Tysabri (natalizumab). Carole Longson, director of the Health Technology Evaluation Centre at NICE added that the independent committee "wasn’t given sufficient evidence to show that fingolimod could reduce relapses considerably better than the other treatments currently being used" so the drug would not represent "effective good use of NHS resources".

Unsurprisingly, Novartis says it has "a difference of opinion about how patients failing on first-line therapies are currently managed in the UK" and called on NICE to reconsider its decision before the final ruling in November. The Swiss major goes on to say that while NICE rejected the comparator used, ie Avonex, it is one of the world’s most commonly used injections, which real life data shows has broadly similar efficacy to other widely-used first-line treatments. Indeed, the Institute has suggested best supportive care (ie no active treatment at all) as the appropriate comparator for fingolimod, though according to neurologists, "this does not reflect current clinical practice in the UK".

UK 13th out of 14 countries for new MS drugs

Furthermore, during a previous draft NICE appraisal for MS, Novartis notes that "best supportive care was judged an inappropriate comparator and was overturned". The company also cites a report by Sir Mike Richards from July 2010 which highlighted that the UK is ranked 13th out of 14 countries when it comes to access to new treatments for MS; in Germany, more than 2,000 people are already benefiting from fingolimod, which received a licence at the same time as the UK.

Novartis concluded by saying that comparing Gilenya to best supportive care will unfairly restrict access to its drug "as well as any future new treatments. It will be very difficult for any new therapy to demonstrate cost effectiveness against best supportive care".

Charities have also expressed their disappointment at the NICE stance and  Simon Gillespie, chief executive of the MS Society, said "it will leave some people with no effective treatment option". He added that "access to MS treatments in the UK is very poor - in fact people with MS would be better off living almost anywhere else in Europe - and this decision will only deepen that inequality.

Mr Gillespie went on to say that "we’re concerned at how this decision has been reached and now strongly encourage NICE and Novartis to work together to look at how the treatment can be better re-considered and evaluated.”