Amgen Ltd has been dealt a blow by the National Institute for Health and Clinical Excellence which is leaning towards barring patients with a rare blood disorder from access to the firm’s Nplate on the National Health Service.

The cost watchdog published appraisal documents this morning in which it does not recommend the use of Nplate (romiplostim) for its authorised indication - the treatment of adult patients with chronic idiopathic thrombocytopenic purpura (ITP) who are refractory to other therapies – on the basis that the therapy is not a cost effective use of NHS resources.

Chronic ITP is a potentially life-threatening automimmune bleeding disorder characterised by the mass destruction of platelets and sometimes also a reduction in platelet production, and currently affects around 3,600 adults in the country.

Amgen launched Nplate - which it claims is the first treatment specifically developed for adults with the condition - in the UK last month, offering doctors a new treatment strategy with the drug’s potential to actually boost platelet production as opposed to existing therapies – such as immunoglobulins and immunosuppressants – which focus on reducing the rate of platelet destruction instead.

The drug won a regulatory green light in Europe earlier this year following Phase III trial results which demonstrated its ability to raise and sustain platelet counts in 83% of patients whether they had a spleen removal or not. Furthermore, subsequent findings of an extension study have shown that Nplate is still effective after three years of therapy.

However, following a review of Amgen’s submission, the Appraisal Committee concluded that while there is evidence to show that Nplate is more clinically effective than placebo and is considered by specialists to have advantages over other active treatments, hard clinical evidence to demonstrate its effectiveness compared to existing therapies is lacking.

Furthermore, it claims that the manufacturer has significantly underestimated the cost per QALY gained and so it believes the treatment, which has a price tag of £1.928 per microgram, equating to a therapy cost of £8,020 a year for someone weighing 80 Kg at this dose, is simply too expensive for NHS use.