Novartis was invited to submit evidence for this single technology appraisal for eltrombopag in September 2015.
The company did not make an evidence submission because it was unable to develop robust clinical- or cost-effectiveness analyses. The marketing authorisation for eltrombopag for treating severe aplastic anaemia was based on 1 small (n=43), non-comparative, single-centre study. The primary endpoint (response rate) was measured at 3 months, and the relationship between this and long-term outcomes is unclear. All of these factors severely limited the ability of the company to construct a robust case for clinical or cost effectiveness.
NICE has therefore terminated this single technology appraisal.