Gemtuzumab OzogamicinMylotarg

The PBAC recommended the listing of gemtuzumab ozogamicin, in combination with standard intensive chemotherapy (an anthracycline and cytarabine), for the treatment of patients with previously untreated, de novo CD33-positve acute myeloid leukaemia (AML) except acute promyelocytic leukaemia, who have favourable/intermediate/unknown cytogenetic risk (where the unknown risk is due to inconclusive test results).PSD · Nov 2021

The PBAC remained of the view that the exact magnitude of the overall survival benefit was difficult to determine, and that the economic model was complex and difficult to assess. Although the resubmission had not addressed all areas of uncertainty, nonetheless, the PBAC considered that the cost-effectiveness of the listing would be acceptable, in the context of the reduced price offered in the pre-PBAC response and a high clinical need in a smaller, more …PSD · Nov 2021

The resubmission presented a modelled economic evaluation of gemtuzumab in combination with standard intensive chemotherapy compared to standard intensive chemotherapy alone for the treatment of patients with previously untreated, de novo, CD33-positive AML, except acute promyelocytic leukaemia who do not have unfavourable cytogenetic risk (i.e. favourable, intermediate or unknown risk).PSD · Nov 2021

Table 8: Key components of the economic evaluation Component Summary Gemtuzumab in combination with standard intensive chemotherapy (daunorubicin and cytarabine) versus Treatments standard intensive chemotherapy alone (daunorubicin and cytarabine), for induction and consolidation therapy 25 years in the model base case versus median follow-up of 4 years in the gemtuzumab arm and 3.4 years Time horizon in the control arm in the trial Outcomes Life years, …PSD · Nov 2021

The resubmission described gemtuzumab as superior in terms of effectiveness compared to standard of care. 22PSD · Nov 2021

The resubmission provided additional treatment effect estimates and conducted multiple interaction tests to provide additional certainty on the magnitude of treatment benefit in the subgroup with favourable/intermediate/unknown cytogenetic risk. The data were difficult to interpret and were limited given the large number of analyses conducted that were not adjusted for multiplicity.PSD · Nov 2021

The PBAC noted and welcomed the input from health care professionals (1) and organisations (3) via the Consumer Comments facility on the PBS website. Comments were received from the Leukaemia Foundation, Rare Cancers Australia, and the Australasian Leukaemia and Lymphoma Group (ALLG).PSD · Nov 2021

The PBAC sought additional clinical advice from the Haematology Society of Australia and New Zealand (HSANZ) and the ALLG. Among other matters, the PBAC noted that:  HSANZ advised that AML was a poor prognosis aggressive blood cancer, and that gemtuzumab has shown benefits in terms of event-free survival and overall survival, thus addressing an unmet clinical need.PSD · Nov 2021