Analysts may be rethinking their sales forecasts for GlaxoSmithKline's lupus drug Benlysta after cost regulators for the National Health Service in the UK turned down funding for the drug.

In final draft guidances issued by the National Institute for Health and Clinical Excellence and the Scottish Medicines Consortium, regulators concluded that Benlysta (belimumab) could not be considered a cost-effective use of NHS resources for patients with active autoantibody positive systemic lupus erythematosus with a high degree of disease activity despite standard therapy.

Lupus - a chronic autoimmune disease that, if uncontrolled, can lead to severe, debilitating symptoms, long-term organ damage and premature death - affects more than 25,000 people in the UK, and some patients with advanced disease fail to respond to current therapy. 

Benlytsa, approved in Europe in 2011, is the first in a new class of drugs called BLyS-specific inhibitors, which work by targeting a naturally occurring protein believed to play a role in the production of antibodies which attack and destroy the body’s own healthy tissues. 


But while NICE's appraisal committee concluded that, compared with standard care, there was some evidence of the clinical effectiveness of the drug, its benefit was not enough to justify its price to the NHS, "as the cost of the drug in relation to how well it works is very high", said Sir Andrew Dillon, the Institute's chief executive.

In addition, some patients with severe disease currently receive rituximab off-label. Although it is not licensed for this use, the Committee would have liked to see data comparing the cost of Benlysta and rituximab, as it is used in clinical practice.

The Institute's sister group in Scotland also agreed. "The treatment's cost in relation to its health benefits was not sufficient and, in addition, the [GSK] did not present a sufficiently robust economic case to gain acceptance by the SMC", it said.

Anger over decision

But Simon Jose, General Manager at GSK, has slammed the decisions. "By denying access to belimumab, which is the first treatment specifically developed and licensed for lupus in over 50 years, UK patients are being left behind those in other countries including the US, Germany and Spain who already have access to this medicine” he said.

While recognising that there are finite resources within the health service, "evaluating an innovative medicine in a framework that compares it to a standard of care consisting of inexpensive generics fails to recognise the benefit of this clinically proven medicine", Jose argues, and stresses that the company believes it has not only priced the drug "responsibly" but has also offered a Patient Access Scheme to both NICE and the SMC, "which we firmly believe represents value for both a cost-constrained NHS and patients with uncontrolled lupus".

On a more general note, he berated the systematic failure to recognise and adopt innovative new medicines in the UK. "The appraisal system itself, and its failure to recognise innovation, remains a fundamental problem", he said, noting that the UK is "one of the slowest to enable patients to have access to innovative new treatments." 

NICE has not yet issued final guidance to the NHS.