Cost regulators are giving the go-ahead to Amgen's Nplate for use on the National Health Service to treat certain patients with the bleeding disorder immune thrombocytopenic purpura.
ITP, which affects around 3,000-3,500 patients in the UK, is a bleeding disorder in which the immune system destroys platelets needed for normal blood clotting. Nplate (romiplostim) is a protein that mimics the action of thrombopoietin, which ultimately boosts platelet production and platelet counts, and was launched in the UK back in 1999.
In draft guidance published this morning, the National Institute for Health and Clinical Exellence said Nplate can be used on the NHS in England and Scotland to treat adults with chronic forms of the condition, but only when unresponsive to standard active treatments and rescue therapies, in patients who have severe disease, or those at high risk of bleeding requiring frequent courses of rescue therapies.
Amgen will be particularly cheered by the news given that back in 2009 NICE rejected its drug on the grounds that it is too expensive for health service use. However, the subsequent offer of a patient access scheme (PAS) - under which it offered to provide Nplate at a discounted price tag, details of which are confidential - helped boost the drug's cost-effectivness and change the Institute's mind.
Taking the PAS into account, the Committee concluded that the incremental cost effectiveness ratios for Nplate would be under £20,000 per QALY gained when treating splenectomised patients, and around £30,000 per QALY gained for those who have not had their spleens removed, thus representing value for money for the NHS.
Carole Longson, Director, Health Technology Evaluation Centre at NICE, noted that as ITP is a serious and sometimes debilitating disorder, and given that some of the current treatments have "considerable" side effects, the Institute is pleased to be able to endorse the use of Nplate "as a clinically and cost effective treatment" in eligible patients.