Trientine Tetrahydrochloride

5.1 The PBAC did not recommend trientine tetrahydrochloride (4HCl) for the treatment of patients with Wilson disease (WD) who are intolerant to penicillamine/D- penicillamine (DPA). The PBAC noted that the only changes in the resubmission were a small price reduction for trientine 4HCl and a proposed risk sharing arrangement (RSA).PSD · Mar 2022

5.2 The PBAC noted that no consumer comments were received in support of the resubmission.PSD · Mar 2022

4.8 A summary of the key matters to be addressed is presented in Error! Reference source not found..PSD · Mar 2022

Table 2: Summary of key matters to be addressed – economic model Matter of concern Resubmission Addressed? The PBAC considered that the results of the CUA No changes were made to the model structure. No between trientine 4HCl and BSC were highly uncertain The resubmission attempted to address the PBAC’s as the studies did not provide a basis for a quantitative concern of uncertainty through a 17.6% reduction in estimate of effective size for trientine …PSD · Mar 2022

The submission claimed that trientine and DPA were non-inferior, based on observational studies comparing trientine 2HCl and DPA, and then claimed that chelation therapy is superior to BSC, based on DPA studies. Noting the acceptance by TGA of the equivalence of between trientine 4HCl to trientine 2HCl, the submission therefore described trientine 4HCl as superior in terms of effectiveness compared to BSC.PSD · Nov 2021

The ESC noted that if trientine 4HCl was non-inferior to DPA, as claimed in the submission, then the price of trientine 4HCl should be more comparable to that of DPA.PSD · Nov 2021

The PBAC noted that only one health care professional provided input via the Consumer Comments facility on the PBS website. The comment stated that trientine has been available to patients for several years via the special access scheme at a price which PBAC noted is much less than that requested in the submission. The health care professional did not support a PBS listing of trientine at a higher price than is currently available to hospitals.PSD · Nov 2021

The submission proposed a cap-based risk share arrangement (RSA) could be used to protect against use of trientine 4HCl in patients who would not otherwise be considered intolerant to DPA. The submission indicated the use and cost of trientine 4HCl could form the basis of a financial cap, with the extent of the rebate for expenditure beyond the cap to be negotiated between the sponsor and the Department of Health.PSD · Nov 2021