Roche’s potential blockbuster RoActemra has suffered a blow in the UK after appraisal consultation documents published by the National Institute for Health and Clinical Excellence have turned down its use on the National Health Service for rheumatoid arthritis.

The cost watchdog’s rejection of the drug has angered the National Rheumatoid Arthritis Society, which says the preliminary position not to allow the drug’s use for moderate-to-severe forms of the illness is a “bitter blow” for patients.

As the group’s chief executive Ailsa Bosworth explained, the decision to bar patients’ access to RoActemra (tocilizumab) means the only choices they will have is to retry therapies that have already failed or palliative care, which involves large doses of steroids that can lead to nasty side effects such as osteoporosis when taken in the longer term.

“We simply cannot accept that individuals should be denied the chance of at least regaining some quality of life and condemning them to a life of pain and disability, which could be as or even more expensive to the NHS as well as society as a whole if people, as a consequence of not being able to access clinically effective therapies, lose their jobs,” Bosworth stressed.

Rheumatoid Arthritis (RA) is a chronic, progressive and disabling autoimmune disease affecting 0.8% of the UK adult population. It is an incredibly painful condition, can cause severe disability (this varies between individuals and depends on how severe/aggressive your disease is) and ultimately affects a person’s ability to carry out everyday tasks. The disease can progress very rapidly (again the speed of progression varies widely between individuals), causing swelling and damaging cartilage and bone around the joints. Any joint may be affected but it is commonly the hands, feet and wrists.

Clinical trials of RoActemra have clearly demonstrated its potential benefits to patients with the disease. Studies including over 4,000 patients showed that the drug, alone or in combination with methotrexate - the current standard of care - or other disease-modifying anti-rheumatic drugs (DMARD) significantly reduced the signs and symptoms of RA, regardless of previous therapy or disease severity, compared to DMARDs alone, thereby having a substantial impact on quality of life.

However, while recognising that RoActemra is clinically effective compared with placebo plus DMARDs or tumour necrosis factor (TNF) antagonists, the Committee has concluded that, at an approximate cost of £9,295 a year for a patient weighing approximately 70 kg, the therapy is just too expensive for use on the NHS and would not represent a cost-effective use of resources.

'Hugely detrimental'
Commenting on the Institute’s stance on the drug, Professor Peter Taylor, the NRAS’ Medical Advisor, said aside from its affect on patients the move is also “hugely detrimental” to the UK marketplace. “It is sending a clear message to the companies who are currently investing in innovative medical research programmes into inflammatory forms of arthritis that the UK market is becoming less relevant,” he warned.

RoActemra was approved by European regulators earlier this year for adults with moderate to severe rheumatoid arthritis who have failed to respond to DMARDs or anti-TNFs. Its path in the US has, however, been a little rockier, after regulators requested extra non-clinical information back in December. Roche resubmitted its application for the drug – which is known as Actemra across the pond – in July and is still awaiting a decision.