NICE recommends romiplostim for the treatment of rare blood disorder
NICE has recommended the use of romiplostim for the treatment of patients with severe, chronic immune (idiopathic) thrombocytopenic purpura (ITP).
ITP is a rare bleeding disorder in which the immune system destroys platelets, which are needed for normal blood clotting. As a result, people with the disease have abnormally low levels of platelets in the blood.
The condition is currently thought to affect 3,000 - 3,500 people in the UK; some patients may only have the condition for a short period of time, but when a patient has ITP for over 12 months this is defined as having chronic ITP.
In this final guidance, romiplostim is recommended for the treatment of adults with chronic ITP whose condition does not respond to standard active treatments and rescue therapies or who have severe disease, and a high risk of bleeding that requires frequent courses of rescue therapies, and if the manufacturer, Amgen, makes romiplostim available with the rebate on the list price agreed under the patient access scheme.
Only a haematologist should initiate and supervise treatment with romiplostim, according to the guidance.
Dr Carole Longson, Director, Health Technology Evaluation Centre at NICE said: “We are pleased to be able to recommend the use of romiplostim as a clinically and cost effective treatment for some people with severe, chronic ITP, a serious and sometimes debilitating disorder. The manufacturer submitted a patient access scheme, and the cost of the treatment to the NHS is reduced.”
27 April 2011