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Ruxolitinib for the treatment of myelofibrosis: 24 month predicted versus actual analysis

Page last updated: 15 March 2019

Drug utilisation sub-committee (DUSC)

September 2018

Abstract

Purpose

To compare the predicted and actual utilisation of ruxolitinib for myelofibrosis in the first 24 months of Pharmaceutical Benefits Scheme (PBS) listing.

Restriction (abridged)

Ruxolitinib was PBS listed for myelofibrosis on 1 February 2016.

It is restricted for use in high risk and intermediate-2 (Int-2) risk myelofibrosis, or intermediate-1 (Int-1) risk myelofibrosis. The condition must be primary myelofibrosis or post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis.

Int-1 risk myelofibrosis patients have the additional clinical criterion that they must have severe disease-related symptoms that are resistant, refractory or intolerant to available therapy.

Data Source

The analyses use data from the Department of Human Services (DHS) prescriptions database and the DHS Authority approvals database from February 2016 to the end of April 2018.

Key Findings

  • In the first and second years of PBS listing, 861 and 1,082 patients received treatment with ruxolitinib, respectively. This was [redacted] more patients than predicted in both years.
  • Despite more patients receiving treatment, there were fewer prescriptions dispensed than expected. This may be due to new patients commencing throughout a listing year and lower adherence in clinical practice than in the clinical trial setting. Insufficient data are available at this stage to establish the median time on therapy.
  • The average dose used in practice was similar to that predicted from the clinical trial setting. The average predicted daily dose was [redacted] mg per day and the actual average daily dose was 27.1 mg.
  • The proportion of patients with Int‑1 risk myelofibrosis treated with ruxolitinib was 14% and 23% in Years 1 and 2 respectively.
  • Approximately half of Int-1 patients had PBS hydroxyurea in the 2 years prior to commencing treatment with ruxolitinib.

Full Report