Lenalidomide for myelodysplastic syndrome: predicted versus actual analysis
Page last updated: 2 March 2018
Drug utilisation sub-committee (DUSC)
September 2017
Abstract
Purpose
To compare the predicted and actual utilisation of lenalidomide for myelodysplastic syndrome (MDS) since it was PBS listed for this indication in October 2013. To list any key uncertainties identified by the PBAC during evaluation of the submission and explore what information about these can be determined from studies of current utilisation or information from key stakeholders.
Current PBS restriction for MDS
Listed under the s100 Highly Specialised Drug (HSD) Authority Required PBS program for treatment of transfusion-dependent, low risk or intermediate stage (low risk/INT-1), deletion 5q MDS. Details of the full restriction are attached in Appendix A.
Prescribers need to provide information specified in the restriction in writing to the Department of Human Services (DHS) to commence treatment (establish eligibility) and to continue treatment (establish response to treatment has occurred within 16 weeks of treatment). Once the first continuation is approved prescribers obtain subsequent prior approval for continuation by telephone.
Data Sources
Data to assess utilisation was obtained from the Department of Human Services (DHS) PBS prescription claims database and the DHS authority approvals database.
Key Findings
- The actual prescription utilisation was much less than predicted due both to a lower than predicted number of patients treated and lower than predicted prescriptions per patient per year.
- The reason for the number of patients treated being less than predicted is not clear.
- The proportion of eligible patients could have been an overestimate. This report notes that the prevalence was incorrectly calculated in the estimates considered by the PBAC. When corrected during this analysis, the eligible patient numbers were slightly less. The estimate of the proportion of MDS patients that are low risk/INT-1 was based on a small study and clinical information which may not accurately reflect the overall Australian experience.
- Lower than expected uptake rate may be a result of patient and prescriber experience of intolerance and adverse events or achievement of less than expected benefit in a larger number of patients with transfusion dependent MDS in practice.
- The submission overestimated the number of prescriptions per patient per year. This was due to a “part cycle correction” not being applied to each year which allows for patients to commence treatment during the year.
- This analysis found that the number of prescriptions per patient overall was slightly more than expected, but these were spread over a longer period than expected and included breaks in treatment.
- The proportion of use of the 5 mg capsules (32.7%) was higher than predicted. This result is consistent with PBAC’s concern that adverse events and dose reduction would be greater than predicted.
- The predicted cost savings from reduction of deferasirox use were not realised.
Full Report
