Romiplostim and eltrombopag for idiopathic thrombocytopenic purpura, June 2015

Page last updated: 30 October 2015

Abstract

Purpose

To examine the utilisation of the thrombopoietin receptor agonists (TRAs), romiplostim and eltrombopag, for idiopathic thrombocytopenic purpura (ITP).  The Pharmaceutical Benefits Advisory Committee (PBAC) requested review of these agents once data for three years of use were available.

Date of listing on PBS

  • Romiplostim – 1 April 2011.
  • Eltrombopag – 1 November 2011.

Data Source / methodology

Authority approvals data and prescription data from the Department of Human Services (DHS). Data were extracted from the date of listing (April 2011) to the most currently available data (December 2014). Prescription data was based on the date of supply.

Key Findings

  • The total of 303 patients supplied with TRA therapy in the first year of listing (April 2011 to March 2012) was XX% less than projected (XXX patients). The actual total number of patients was more than predicted in the third year of listing (506 vs. XXX, respectively).
  • The financial estimates for eltrombopag were sensitive to the proportional use of its maximal dose (75 mg). The actual mean daily dose for continuers (54 mg) was similar to the predicted average doses of 58 mg for splenectomised and 56 mg for non-splenectomised patients. The actual mean daily dose for initiators (45 mg) was lower than the predicted doses.
  • The majority of patients remained on TRA treatment for more than a year (mean duration of 1.1 years for romiplostim and 1.5 years for eltrombopag).
  •  Of the 147 patients initiating on PBS subsidised TRA therapy during the first year of listing (April 2011 to March 2012), 60 patients (41%) switched from their initial therapy. Sixty eight of the 147 patients initiating in Year 1 were grandfathered. The mean time on TRA therapy was slightly longer for patients who switched treatment (1.5 years) or who were grandfathered (1.4 years) compared to all initiators (1.3 years).
  • The PBS and RPBS (R/PBS) actual expenditure on TRA therapy has been significantly less than predicted during the first three years of listing.

Full Report