← The record

CarmustineBiCNU

Not recommended Oncology

treatment of recurrent glioblastoma multiforme (GBM) in patients for whom surgical resection is indicated and recurrence has occurred within 6 months of temozolomide or where temozolomide is contraindicated or not tolerated due to side effects

4
Submissions
2 resub
2005–08
On the record
$45k–200k
ICER range
2 sourced ICERs · 2005–2008
ICER stated
Cost basis

Access path

4 submissions · public record
  1. TGA registered · BiCNU

    TGA label narrower than the PBS population

  2. Nov 2005
    Recommended · restricted

    vs temozolomide

  3. Mar 2006
    Not recommended

    Insufficient evidence of benefit in terms of survival gain or quality of life improvements in the broader population…

  4. Mar 2007
    Not recommended

    uncertain clinical effectiveness resulting from the lack of a common reference, unequal distribution of additional…

  5. Jul 2008
    Not recommended

    Uncertain clinical benefit, high and uncertain cost-effectiveness, trial population markedly different from requested…

RecommendedDeferredNot recommended

Cost-effectiveness

2 sourced ICERs · 2005–2008

Base case ICER stated; however, PBAC noted ICER for temozolomide-treated patients (the larger part of the population targeted) could not be estimated with confidence and would likely be significantly higher. Unadjusted ICER range was $45,000–$105,000/LYG.

The PBAC concluded that the incremental cost effectiveness ratio (ICER) for temozolomide-treated patients could not be estimated with any degree of confidence and would most likely be significantly higher than the base-case ICER which was in the range of $45,000 - $75,000 per life year gained (LYG) which was based on the most favourable hazard ratios (0.64 - 0.75) i.e., those calculated after adjustment for prognostic factors. PBAC · 2008
ICER uncertain ×2Economic model disputed ×2No head-to-head trialIndirect comparison

Decision context

Populationpatients with recurrent glioblastoma multiforme (GBM) who have failed temozolomide within the previous 6 months or are intolerant to it, or have a contraindication to temozolomide

Why it was knocked back

  • Uncertain clinical benefit, high and uncertain cost-effectiveness, trial population markedly different from requested PBS population (participants had no prior temozolomide exposure whereas restriction targets temozolomide-failed patients), considerable uncertainty about whether prior temozolomide use would diminish benefit, post-hoc subgroup analysis introduces uncertainty about extent of effectiveness advantage, increased toxicity risk (abnormal healing and pain) compared to placebo

Submission history

4 entries
DecidedOutcomeComparatorICEREvidence
Jul 2008 Not recommended placebo implants $45k–75k RCT · OS
Mar 2007 Not recommended temozolomide given orally RCT · OS, PFS
Mar 2006 Not recommended
Nov 2005 Recommended · restricted temozolomide $75k–200k RCT · OS

Similar precedents

By decision profile

Regulatory · TGA

Label narrower than PBS population — PBAC restricts to recurrent GBM with prior temozolomide failure/intolerance; TGA label includes newly-diagnosed and recurrent without prior-therapy requirement.