Eltrombopag for the treatment of chronic idiopathic (immune) thrombocytopenic purpura (ITP): a single technology appraisal
This project was carried out as part of the National Institute for Health and Care Excellence (NICE) Single Technology Appraisal (STA) process. The manufacturer of eltrombopag (GlaxoSmithKilne plc) submitted an evidence review and an economic model assessing the cost-effectiveness of eltrombopag against a range of treatment comparators for two distinct treatment scenarios (Watch and Rescue care and long-term treatment of ITP) in two patient groups (splenectomised patients and non-splenectomised patients). The Aberdeen HTA group were commissioned to act as the Evidence Review Group (ERG) for the project, and to provide an independent report reviewing the manufacturer’s evidence submission.
Outcome and Translation
Based on the manufacturer’s submitted evidence and the subsequent ERG review and analysis of this evidence, eltrombopag was not initially recommended within its marketing authorisation for the treatment of chronic ITP: in splenectomised adults whose condition is refractory to other treatments (for example, corticosteroids, immunoglobulins) oras second-line treatment in non-splenectomised adults wheresurgery is contraindicated.
Following a subsequent review of the guidance and submission of a Patient Access Scheme, NICE updated their guidance to recommend eltrombopag as a treatment option for these patients, only if:
- Their condition is refractory to standard active treatments and rescue therapies or
- They have severe disease and a high risk of bleeding that needs frequent courses of rescue therapies.
HERU researchers involved in this research project: Dwayne Boyers (HERU) and Luke Vale (HERU/HSRU)
External collaborators: M Crowther (Department of Haematology, Aberdeen Royal Infirmary); D Jenkinson, C Fraser, G Mowatt and X Jia (HSRU, University of Aberdeen)
Boyers, D., Jia, X., Crowther, M., Jenkinson, D., Fraser, C. and Mowatt, G. (2011) 'Eltrombopag for the treatment of chronic idiopathic (immune) thrombocytopenic purpura (ITP)', Health Technology Assessment, 15(Suppl 1), 23-32.
Boyers, D., Jia, X., Jenkinson, D. and Mowatt, G. (2012) 'Eltrombopag for the treatment of chronic immune or idiopathic thrombocytopenic purpura: a NICE Single Technology Appraisal', Pharmacoeconomics, 2012, 30(6), 483-495.
Boyers, D., Jia, X., Jenkinson, D. and Mowatt, G. (2013) 'Eltrombopag for the treatment of chronic immune or idiopathic thrombocytopenic purpura—a NICE single technology appraisal', PharmacoEconomics Italian Research Articles, 15(3), 143-152.
Boyers, D., Jia, X., Crowther, M., Jenkinson, D., Fraser, C. and Mowatt, G. (2010) 'Eltrombopag for the treatment of chronic immune (idiopathic) purpura, a single technology appraisal', NICE Appraisal Committee Meeting, Manchester, January 2010.
Boyers, D., Hernández, R., Vale, L., Maclennan, G., Crowther, M. and Mowatt, G. (2012) 'NICE Single Technology Appraisal (STA) process: a case study of chronic idiopathic (immune) thrombocytopenic purpura', [poster] Health Technology Assessment International (HTAi), Bilbao, Spain, 25-27th June.