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Cinacalcet HydrochlorideAKM CINACALCET

Recommended EndocrinologyAuthority Required

For the treatment of severe primary hyperparathyroidism and persistent or recurrent hypercalcaemia following resection of parathyroid carcinoma.

6
Submissions
5 resub
2005–09
On the record
$15k–200k
ICER range
5 sourced ICERs · 2005–2009
ICER stated
Cost basis

Decisions on record

2 decisions
  • Meeting Nov 2010 Recommended Chronic kidney disease no PSD
  • Meeting Jul 2009 Not recommended Hyperparathyroidism Hypercalcaemia

Access path

6 submissions · public record
  1. TGA registered · AKM CINACALCET

    TGA label narrower than the PBS population

  2. Nov 2005
    Not recommended

    uncertain extent of clinical benefit (surrogate outcomes only, no demonstrated impact on bone fractures, cardiovascular…

  3. Nov 2005
    Not recommended

    uncertain clinical benefit (biochemical surrogate outcomes only, no demonstrated impact on bone disease or quality of…

  4. Jul 2006
    Not recommended

    uncertain clinical benefit from post hoc analysis of safety data (not primary endpoints), uncertain mortality hazard…

  5. ↻ resubmitted
    Nov 2007
    Recommended

    Comparator changed: placebo for add-on to standard care involving dietary modification…

  6. Nov 2007
    Recommended · restricted

    Comparator changed: placebo for add-on to standard care involving dietary modification…

  7. Jul 2009
    Not recommended

    lack of durability of hypocalcaemic effect, no clinical outcome data (fractures, myocardial infarction, death), no…

RecommendedDeferredNot recommended

Cost-effectiveness

5 sourced ICERs · 2005–2009

ICER range varies by population: severe PHPT $45,000–75,000 per QALY; parathyroid carcinoma $105,000–200,000 per QALY. If average increase of mortality risk is used, ICERs become >$200,000/QALY.

The PBAC noted the incremental base case cost-effectiveness ratios for the severe PHPT and the parathyroid cancer population were high, in the range $45,000 – 75,000 per QALY and $105,000 – 200,000 per QALY, respectively. PBAC · 2009
ICER uncertain ×4ICER / price too high ×2Economic model disputed ×3Price cut / RSA neededImmature survival dataSurrogate endpoint

Decision context

Populationpatients with severe primary hyperparathyroidism (defined as two consecutive readings of serum calcium > 2.85 mmol/L) and persistent or recurrent hypercalcaemia following resection of parathyroid carcinoma

Why it was knocked back

  • lack of durability of hypocalcaemic effect, no clinical outcome data (fractures, myocardial infarction, death), no difference in quality of life compared to placebo, high and uncertain cost-effectiveness ratios, uncertainty regarding extrapolation of survival benefit from maximum 4-year observation to 35-year model duration, small study populations

Submission history

6 entries
DecidedOutcomeComparatorICEREvidence
Jul 2009 Not recommended placebo and standard medical management $45k–200k RCT, Single-arm · serum calcium reduction
Nov 2007 Recommended placebo plus standard medical management RCT · other
Nov 2007 Recommended · restricted placebo plus standard medical management $15k–45k RCT
Jul 2006 Not recommended placebo for add-on to standard care involving dietary modification, vitamin D products in association with calcium-based $15k–45k RCT
Nov 2005 Not recommended placebo for add-on to standard medical management involving dietary modification, vitamin D products in association with $15k–45k RCT · OS | PFS | DFS | ORR | QoL | Surrogate
Nov 2005 Not recommended placebo for add-on to standard medical management $15k–45k RCT · iPTH response

Similar precedents

By decision profile

Regulatory · TGA

Label narrower than PBS population — PBAC restricts to severe primary hyperparathyroidism (calcium >2.85 mmol/L) and post-resection carcinoma; excludes secondary hyperparathyroidism and untreated parathyroid carcinoma.