PBAC's latest decision on Nitisinone: Not recommended (2015). Considered for Treatment of patients with hereditary tyrosinaemia type 1 (HT-1).
PBAC outcome
Not recommended
Restricted
ICER (AUD/QALY)
$200k/QALY
Range: $105k/QALY–$200k/QALY
Submissions
2
first 2014
Submissions
2
2014 → 2015
Eligible population
Patients with a confirmed clinical diagnosis of hereditary tyrosinaemia type 1 based on detection of succinylacetone in urine and/or blood, to be treated in combination with dietary restriction of tyrosine and phenylalanine at a centre with experience in metabolic disorders.
Therapy area
Metabolic
Line of therapy
Not applicable
Evidence base
Single-arm
Primary endpoint
LY
Key trials
Larochelle 2012
Comparator
standard medical management without nitisinone (diet alone)
Economic model
CUA
ICER note
ICER stated for early nitisinone treatment based on Larochelle 2012 trial ($105,000–$200,000 per QALY). PBAC also noted that the ICER for continuation of current screening practices exceeded $200,000 per QALY. PBAC considered both ICERs unacceptably high.
Risk sharing
Risk-sharing arrangement in place — Sponsor indicated willingness to enter into discussion with PBAC on a Risk Sharing Agreement and potential mechanisms to contain the risk associated with the cost of the drug to the PBS.
Why PBAC said no
Reasons cited in the latest PSD: uncertain and unacceptably high estimate of cost effectiveness, high cost of nitisinone, unclear safety profile particularly regarding neurocognitive disorders, inadequately addressed prior PBAC concerns regarding cost per patient per year
Submission history
Nov 2014: Recommended with restriction · Authority Required
Jul 2015: Not recommended · Restricted — ICER $200k/QALY