EverolimusAfinitor

7.1 The PBAC deferred its decision about whether to extend the listing of everolimus to include the treatment of refractory seizures associated with tuberous sclerosis complex (TSC), to allow for negotiations to establish the appropriate restriction, and a price for treatment that could be considered cost-effective.PSD · Dec 2017

7.2 The PBAC agreed with the ESC that it might be appropriate to consider a whole-of- disease approach to PBS subsidy of everolimus for TSC, given the ability to block the disease pathway, rather than individual consideration of disease presentations as more evidence becomes available, noting that the evidence presented in the submission was solely focused on the management of refractory epilepsy.PSD · Dec 2017

6.22 The submission presented a modelled economic evaluation comparing EVR with best supportive care for refractory seizures associated with TSC. The costs and outcomes calculated for EVR in the model reflected the medication and administration costs and outcomes in HT EVR arm of EXIST-3.PSD · Dec 2017

Table 8: Summary of model structure and rationale Component Summary Time horizon 5 years in the model base case versus 18 weeks in trial Outcomes QALYs Methods used to generate results Cohort expected value analysis A simple Markov model with four health states based on reduction in seizure frequency from baseline: “Seizure free (100%)”, “Responder (≥50% to <100%)”, “Partial responder (≥25% to <50%)” and “Discontinued (<25%)”; death was not modelled.PSD · Dec 2017

6.10 The re-submission described everolimus as non-inferior to sunitinib in terms of clinical effectiveness for the treatment of patients with advanced pNET, with a different, but manageable, safety profile. 6.11 The PBAC accepted the re-submission’s clinical claim.PSD · Mar 2014

6.7 The re-submission did not alter its November 2011 claim that everolimus has superior efficacy and inferior safety in mRCC compared with placebo.PSD · Mar 2014

6.2 The PBAC noted and welcomed the input from individuals (2), and organisations (1) via the Consumer Comments facility on the PBS website. The comments described a range of benefits of treatment with everolimus including reduced seizure frequency and severity, improved quality of life for patients and their families, and reduced carer burden.PSD · Dec 2017

6.34 The submission proposed a special pricing arrangement for the proposed listing that included an effective price cheaper than the current PBS listing for TSC (i.e., SEGAs, visceral tumours) on a cost/mg basis. It was anticipated that there would also need to be a Deed of Agreement for the proposed indication of refractory seizures associated with TSC and the Sponsor is willing to discuss the content with the PBAC.PSD · Dec 2017