Eculizumab for atypical haemolytic uremic syndrome (aHUS), October 2020
Page last updated: 15 March 2021
Drug utilisation sub-committee (DUSC)
October 2020
Abstract
Purpose
DUSC has previously undertaken analysis of eculizumab for the treatment of atypical haemolytic uraemic syndrome (aHUS) in September 2017 and February 2019. These analyses comprised a 24-month predicted versus actual utilisation analysis (2017) and an update to the previous utilisation report.
At its June 2020 meeting, DUSC requested an update to the previous reports and adding additional data on length of time on treatment (LoT) and date of death data (DoD). Consequentially, this latest analysis provides updated utilisation data, up to and including 31 May 2020; information on patient initiations up to and including 30 June 2020; and data on LoT, and DoD data.
Date of listing on the Pharmaceutical Benefits Scheme (PBS)
1 December 2014
Data Source / methodology
The analyses used data from the Services Australia (formerly known as the Department of Human Services) supplied prescriptions database and authority approval database for dates of supply up to and including 30 November 2019. Data from Services Australia Complex Drugs aHUS monthly reports from January 2017 up to and including 30 June 2020 has also been used in this update. Analyses in the report include:
- New and prevalent patient counts by year
- Prescriptions by treatment phase
- Patient age at initiation
- Length of treatment
- Length of therapy pre and post February 2016 restriction change
- Length of treatment by top 3 initial prescribers
- Patient status
- Length of treatment using date of death data
- Prescriber type
- Initiating prescribers by prescriber type
- Patient count by provider
- Approved patient affected organs
- Restriction sequence
- Patient flow from ‘Initial’ to ‘Balance of Initial’
- Approved patient vaccination / antibiotic evidence status
Key Findings
- Since its listing in December 2014, 323 patients have been supplied eculizumab for aHUS (to the end of May 2020). Of these 87 (27%) people were on treatment at the end of the analysis period; meaning 236 people had stopped treatment.
- DoD data shows that a total of 74 people out of 298 who were supplied eculizumab from December 2014 to 20 March 2020 had died. Of these 74, 39 (13.1% of total patient) have died whilst on treatment. This indicates that some patients may have been treated beyond disease progression.
- Adults (18 years and over) make up the majority (90.4%) of patients treated with eculizumab.
- Of the top 3 prescriber types (general practitioners (GPs), nephrologists and haematologists), patients prescribed initial treatment by nephrologists and haematologists spent the longest amount of time on treatment.
- At the time of initial application approvals, 68% of patients had an ADAMTS-13 score recorded and 32% did not. Of the patients without an ADAMTS-13 score, 29.9% did not proceed to receive further treatment. The remaining 70.1% provided an ADAMTS-13 score and proceeded to receive the balance of initial supply.
- There were changes to the restriction in 2016, which may have led to a decrease in LoT. These changes included the addition of having to provide extra information in the application i.e. serial haematological results every three months while on treatment; evidence of an identifiable genetic mutation, and prior history of aHUS.
- Restriction changes in January 2017 led to an increase in the number of adult patients. These changes included adding the possibility of eligibility for treatment when non-renal thrombotic microangiopathy (TMA) related organ damage is present and clarification that the progressing TMA must be caused by aHUS.
- Initiating and prevalent patient numbers are showing signs of stabilising with 2019 being the first year that total patient numbers did not increase compared to the previous years’ total patient numbers.
- The rate of growth in PBS patients is showing signs of declining.
Full Report
PDF version of Full Report (PDF 899KB)
Word version of Full Report (Word 132KB)
