Caplacizumab

The PBAC did not recommend the listing of caplacizumab on the PBS for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP). The PBAC considered the clinical place of therapy was uncertain. The PBAC had low confidence in the trial data given trial design, size, and conduct issues.PSD · Jul 2020

The PBAC noted that the inpatient component of funding in public hospitals would require joint funding between the Commonwealth, states and territories under the usual National Health Reform Agreement (NHRA) arrangements, with only outpatient treatment eligible for subsidy under the PBS.PSD · Jul 2020

The ESC considered a cost-effectiveness/utility analysis was appropriate based on the statistically significant reduction in recurrence of TPP in the caplacizumab arm versus best supportive care. However, the ESC acknowledged the clinical importance of this result was unclear. The ESC further advised that the long-term clinical benefit was unclear and that further clinical input was required to improve the model structure.PSD · Jul 2020

The submission presented a stepped economic evaluation of caplacizumab compared with standard of care for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP). The economic model was based on survival benefit derived from combined HERCULES and TITAN trial data and a retrospective observational study of acute aTTP episodes, extrapolated to 60 years. Other modelled inputs were largely based on HERCULES trial data and assumptions.PSD · Jul 2020

immunosuppression has superior efficacy and inferior safety compared to standard of care alone Source: Table 1.1, p.2 of the submissionPSD · Jul 2020

The submission described caplacizumab 10 mg daily in combination with standard of care as superior in terms of effectiveness, and inferior in terms of safety compared with standard of care alone for the treatment of aTTP.PSD · Jul 2020