This appraisal critically reviewed the manufacturer’s submission of the evidence for the clinical and cost effectiveness of romiplostim for ITP as part of NICE’s single technology appraisal process.
From the results of the manufacturer's submission and additional sensitivity analyses of the economic model conducted by the Evidence Review Group, it appeared that romiplostim was a safe treatment for idiopathic thrombocytopenic purpura (ITP), with evidence of short-term efficacy. There was no long term data on safety and no robust evidence on long-term efficacy of romiplostim. There was also no robust evidence on the long-term effectiveness or cost effectiveness of romiplostim compared with relevant comparators.
Romiplostim was recommended by NICE for the treatment of adults with chronic immune (idiopathic) thrombocytopenic purpura, whose condition was refractory to standard active treatments and rescue therapies or who had severe disease and a high risk of bleeding that needed frequent courses of rescue therapies.
Cynthia Fraser firstname.lastname@example.org
Mowatt G, Crowther M, Fraser C, Hernandez R, Jia X, Ternent L. Romiplostim for the treatment of chronic immune or idiopathic thrombocytopenic purpura: A Single Technology Appraisal. Health Technol Assess 2009;13 (Suppl. 2):63-8.