PatisiranONPATTRO

7.1 The PBAC did not recommend patisiran for treatment of patients with hATTR with polyneuropathy. The PBAC considered there was a high unmet need in the requested patient population, and that efficacy and safety of patisiran compared with BSC had been demonstrated by the clinical evidence, although there was a limited duration of randomised evidence. The PBAC considered that the submission had not demonstrated patisiran is cost-effective. 41PSD · Jul 2023

7.2 The primary reason for this outcome was due to the economic evaluation presented.PSD · Jul 2023

6.48 The submission presented a modelled economic evaluation comparing patisiran with BSC, for the treatment of patients with hATTR amyloidosis with polyneuropathy. ThePSD · Jul 2023

Table 9: Summary of model structure, key inputs and rationale Component Description Treatments Patisiran versus best supportive care (BSC) 40 years versus up to 18 months for the placebo arm in the APOLLO trial and up to 54 months Time horizon (4.5 years) for the patisiran arm in the APOLLO trial and the Global OLE study.PSD · Jul 2023

6.44 The submission described patisiran as superior in terms of delaying disease progression, reducing neuropathy symptoms, and improving health-related quality of life compared to placebo. The claim was supported by the primary outcome of change from baseline in mNIS+7, and secondary outcomes including Norfolk QoL-DN scores (a measure of health-related quality of life), motor strength (measured on NIS- Weakness scale), daily and social activities …PSD · Jul 2023

6.45 The submission described patisiran as comparable in terms of safety compared to placebo. This claim was not adequately supported. While overall safety was comparable between treatment arms in the APOLLO trial, patisiran treated patients experienced more infusion-related reactions. The ESC also noted that mRNA interference therapies are relatively new and that there is limited long term experience with these types of therapies.PSD · Jul 2023

6.2 The PBAC noted and welcomed the input from individuals (57) and organisations (2) via the Consumer Comments facility on the PBS website. The comments described a range of benefits of treatment with patisiran including improvement in the individual’s condition and the slowing of deterioration. The input also described an improvement in quality of life on treatment, but noted the time needed for regular infusions.PSD · Jul 2023

6.3 The PBAC noted the advice received from the Australian Amyloidosis Network (AAN) discussing the effects of the condition and need for effective therapies. It was noted that patients with hATTR are typically younger than their wtATTR counterparts, and they require assistance from multiple specialists to continue to function as best they can.PSD · Jul 2023