← The record

Irinotecan (Nanoliposomal)

Not recommended OncologyNot applicableFirst-line line 💬 consumer voice

First-line treatment of metastatic pancreatic adenocarcinoma in previously untreated patients, administered as NALIRIFOX (nanoliposomal irinotecan in combination with oxaliplatin, 5-fluorouracil, and folinic acid/leucovorin).

4
Submissions
3 resub
2016–24
On the record
ICER range
Not modelled
Cost basis

Decision on record

1 decision
  • Meeting Mar 2018 Not recommended Indicated in the treatment of metastatic adenocarcinoma of the pancreas, in combination with 5- fluorouracil and folinic acid (leucovorin) in adult patients who have been previously treated with gemcitabine-based therapy.

Access path

4 submissions · public record
  1. Nov 2016
    Not recommended

    inadequate justification of comparators, high risk of bias in trials, concerns with indirect comparison reliability…

  2. ↻ resubmitted
    Mar 2018
    Noted

    Comparator changed: 5-FU/folinic acid and mFOLFOX6 → mFOLFOX6 (oxaliplatin plus…

  3. Mar 2024
    Not recommended

    Comparator changed: mFOLFOX6 (oxaliplatin plus 5-fluorouracil/folinic acid), using…

  4. Nov 2024
    Not recommended

    Superiority of NALIRIFOX over FOLFIRINOX not adequately established by indirect evidence; concerns regarding…

RecommendedDeferredNot recommended

From the public summary

Verbatim · PSD text · may span indications
PBAC outcome
The PBAC did not recommend the listing of nanoliposomal irinotecan (nal-IRI), as part of the NALIRIFOX regimen (containing oxaliplatin, 5-fluorouracil (5-FU) and folinic acid/leucovorin), for the treatment of metastatic pancreatic adenocarcinoma (mPAC).PSD · Nov 2024
Consistent with its previous advice, the PBAC considered that the combination regimen known as FOLFIRINOX (containing irinotecan, 5-FU, LV and oxaliplatin) was the relevant main comparator in the proposed population, rather than gemcitabine with nanoparticle albumin-bound paclitaxel (Gem+NabP) as nominated by the resubmission.PSD · Nov 2024
Economic analysis
minimisation approach (para 7.15, irinotecan (nanoliposomal), PSD, March 2024 PBAC meeting) The PBAC considered that the submission’s Not adequately addressed. assumption that NALIRIFOX would replace Gem+NabP in up to 85% of patients was not The resubmission assumed uptake of NALIRIFOX reasonable, and that replacement of will result from the substitution of up to % of FOLFIRINOX should have been modelled.PSD · Nov 2024
Collectively, this has resulted in the uptake rate of NALIRIFOX and subsequent financial estimates likely being overestimated.PSD · Nov 2024
Clinical claim
NALIRIFOX is superior in terms of effectiveness compared to Gem+NabP NALIRIFOX has a different safety profile compared to Gem+NabP, however it should not be concluded that one safety profile is superior or inferior to the other FOLFIRINOX:PSD · Nov 2024
NALIRIFOX is superior in terms of effectiveness (PFS and OS) compared to FOLFIRINOX NALIRIFOX appears to have advantages in terms of incidence of haematological adverse events, including the sequelae of those effects (e.g., infection), compared to FOLFIRINOX.PSD · Nov 2024
Consumer comments
The PBAC noted and welcomed the input from individuals (1), health care professionals (11) and organisations (5) via the Consumer Comments facility on the PBS website. The comments from individuals discussed the effectiveness of irinotecan on tumour size, outlined the side effects while on treatment, and the improvements in physical activity and reduced pain after treatment.PSD · Nov 2024
The PBAC welcomed the input from consumer organisations the Australian Pancreatic Cancer Foundation (PanKind), the Pancare Foundation and Rare Cancers Australia, all supporting the listing of nal-IRI. The Committee noted the input from PanKind discussed the devastating impact of pancreatic cancer and low 5-year survival rates, and shared testimonials from patients and family members about the hopelessness and desperation for better and more affordable …PSD · Nov 2024

Cost-effectiveness

ICER not stated in the document. The PBAC did not consider the cost-utility analysis informative because FOLFIRINOX was considered the appropriate main comparator rather than Gem+NabP.

ICER / price too highEconomic model disputedPrice cut / RSA neededIndirect comparison

Decision context

PopulationPreviously untreated adults with metastatic pancreatic adenocarcinoma with ECOG PS 0-1

Why it was knocked back

  • Superiority of NALIRIFOX over FOLFIRINOX not adequately established by indirect evidence; concerns regarding reliability of indirect treatment comparisons due to transitivity violations and differences between GENERATE and NAPOLI-3 trials; uncertain clinical meaningfulness of OS improvement over Gem+NabP (1.9 months); higher rates of gastrointestinal toxicities and serious adverse events with NALIRIFOX versus Gem+NabP not adequately justified; inappropriate nominating of Gem+NabP as main comparator when FOLFIRINOX is the relevant clinical comparator; lack of robust evidence to support cost-utility analysis; uptake and financial estimates likely overestimated

Submission history

4 entries
DecidedOutcomeComparatorICEREvidence
Nov 2024 Not recommended Mixed comparator: gemcitabine plus nanoparticle albumin-bound paclitaxel (75%) and FOLFIRINOX (25%); PBAC considered FOL RCT · OS
Mar 2024 Not recommended FOLFIRINOX RCT · OS
Mar 2018 Noted mFOLFOX6 (oxaliplatin plus 5-fluorouracil/folinic acid), using 5-fluorouracil/folinic acid monotherapy as a proxy RCT · OS
Nov 2016 Not recommended 5-FU/folinic acid and mFOLFOX6 RCT · OS

Consumer voice

Nov 2024

Consumer input from individuals, healthcare professionals, and organisations supported the listing of nal-IRI (NALIRIFOX) for metastatic pancreatic cancer, highlighting the devastating burden of disease, unmet treatment needs, and the significant survival benefits demonstrated by this therapy.

The comments from individuals discussed the effectiveness of irinotecan on tumour size, outlined the side effects while on treatment, and the improvements in physical activity and reduced pain after treatment. Consumer comments · PSD
unmet needsurvival benefitside effectstreatment burdenquality of lifeaccess barriers

Similar precedents

By decision profile

Regulatory · TGA

Label narrower than PBS population — PBAC restricts to previously untreated patients with ECOG PS 0-1; TGA label includes all first-line patients without performance status specification.