July 2008 PBAC Outcomes - Subsequent Decisions not to Recommend
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Drug and Form |
TGA Indication |
Current PBS Listing |
Listing Requested by Sponsor |
PBAC Outcome and Comments |
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| Botulinum toxin type A, lyophilised powder for IM injection, 100 units vial, (Botox®) Allergan Australia Pty Ltd Major submission |
Botulinum Toxin Type A Purified Neurotoxin Complex is indicated for: treatment of blepharospasm associated with dystonia, including benign blepharospasm and VII nerve disorders (specifically hemifacial spasm) in patients twelve years and over; treatment of cervical dystonia (spasmodic torticollis); treatment of dynamic equinus foot deformity due to spasticity in juvenile cerebral palsy patients two years of age or older; treatment of severe primary hyperhidrosis of the axillae; treatment of glabellar lines associated with corrugator and/or procerus muscle activity; treatment of focal spasticity in adults; treatment of spasmodic dysphonia; Treatment of strabismus in children and adults. | Section 100 (Botulinum Toxin Program) BOTULINUM TOXIN TYPE A PURIFIED NEUROTOXIN COMPLEX Treatment of blepharospasm associated with dystonia, including benign blepharospasm and VIIth nerve disorders (hemifacial spasm) in patients 12 years and older; Treatment of dynamic equinus foot deformity due to spasticity in ambulant paediatric cerebral palsy patients 2 years of age or older; Treatment of spasmodic torticollis, either as monotherapy or as adjunctive therapy to current standard care. |
Section 100 (Botulinum Toxin Program) Treatment of moderate to severe spasticity of the lower limb in ambulatory adults following a stroke as second line therapy when standard management has failed or as an adjunct to physical therapy. To qualify for therapy patients must: be ambulatory, with or without assistive devices/support have no fixed muscle contracture in the targeted muscles for injection that would limit treatment efficacy have spasticity which reduces walking speed have a walking speed < 48 m/min over 10 metres, based on the standard 10 metre walk speed assessment. To continue treatment after the first year: Based on the standard 10 metre walk test, all patients must improve their walk distance by at least 15 m/min, relative to their baseline speed. |
The PBAC rejected the submission because of uncertain clinical benefit and a high and uncertain cost-effectiveness ratio. |
| Comparator: placebo |
Accepted |
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| Clinical Claim: Botulinum Toxin Type A is therapeutically superior and equivalent in terms of comparative safety to placebo. |
Not accepted. The PBAC noted that the claim of superior efficacy is based on a difference in the distribution in walking categories between treatment groups post treatment only. The results of an analysis of change from baseline suggest that there is no difference between the treatment groups in terms of change from baseline mobility or responder rate. |
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| Economic Claim: cost-utility |
Not accepted. The Committee considered that there was considerable residual uncertainty in this estimate due to significant overestimation of the benefit and underestimation of the total cost of treatment. |
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| Sponsor Comments: |
Nil |
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| Carmustine implants, 7.7 mg, 8, (Gliadel®) Orphan Australia Pty Ltd Major submission |
Newly-diagnosed high-grade malignant glioma patients as an adjunct to surgery and radiation. Adjunct to surgery to prolong survival in patients with recurrent glioblastoma multiforme (GBM) for whom surgical resection is indicated. | Restricted benefit Glioblastoma multiforme, suspected or confirmed, at the time of initial surgery. NOTE: Carmustine is not PBS-subsidised for use in conjunction with PBS-subsidised temozolomide. |
Restricted Benefit Recurrent glioblastoma multiforme (GBM) 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. NOTE Carmustine is not PBS-subsidised for use in conjunction with PBS-subsidised temozolomide |
The PBAC rejected the submission because of uncertain clinical benefit and high and uncertain cost-effectiveness. |
| Comparator: placebo |
Accepted |
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| Clinical Claim: Carmustine is described as having advantages in effectiveness and comparable toxicity with respect to its comparator. |
Partially accepted. The PBAC did not accept that the efficacy of carmustine in patients previously treated with temozolomide, the larger part of the population targeted by the restriction, had been demonstrated, as these patients were not included in the pivotal clinical trial. Where patients had never received temozolomide, or where exposure had been substantially truncated due to intolerance, the trial data were more applicable and clinical benefit is more robustly defined. |
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| Economic Claim: cost-effectiveness |
Not accepted. The incremental cost-effectiveness ratio in temozolomide treated patients is high and highly uncertain. The incremental cost effectiveness ratio in patients who have never received temozolomide, or where exposure has been substantially truncated is more certain but too high. |
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| Sponsor Comments: |
The sponsor will be considering its position regarding any future course of action. |
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| Etanercept, injection set containing 4 vials powder for injection 25 mg and 4 pre-filled
syringes solvent 1 mL, injection set containing 4 vials powder for injection 50 mg and 4 pre-filled syringes solvent 1 mL, injections 50 mg in 1 mL single use pre-filled syringes, 4, (Enbrel®) Wyeth Australia Pty Ltd Major submission |
Etanercept is TGA registered for: Active, adult RA in patients who have had inadequate
response to one or more disease modifying antirheumatic drugs (DMARDs); severe, active,
adult RA to slow progression of disease-associated structural damage in patients at
high risk of erosive disease; active polyarticular-course juvenile chronic arthritis;
active and progressive psoriatic arthritis (PsA) in adults, when the response to previous
disease- modifying antirheumatic therapy has been inadequate; active ankylosing spondylitis and severe chronic plaque psoriasis. |
See PBS.gov.au | To extend the current PBS listing for rheumatoid arthritis (RA) and psoriatic arthritis (PsA) to include patients with ?10 tender and swollen joints or ?2 major affected joints and to reduce the thresholds for c-reactive protein (CRP) and erythrocyte sedimentation rate (ESR) levels. |
The PBAC rejected the submission on the basis of uncertainty in the clinical data and economic model, resulting in a high and uncertain cost-effectiveness. |
| Comparator: placebo for standard care |
The appropriate comparator was used in the submission. |
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| Clinical Claim: etanercept has significant advantages in terms of effectiveness over placebo, but is associated with greater toxicity. |
The PBAC considered that there is uncertainty in the clinical effectiveness of etanercept for rheumatoid arthritis and psoriatic arthritis in the proposed additional (marginal) population as it is based on a very small number of patients. The PBAC also noted that although the short and medium-term toxicity of etanercept is well established, the long term toxicity remains uncertain. This may be important in the new population who would commence treatment with etanercept earlier in their disease. |
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| Economic Claim: Cost effectiveness |
Not accepted. The incremental cost effectiveness ratios in the additional populations were considered to be high and unacceptable. |
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| Sponsor Comments: | The sponsor will be considering its position regarding any future course of action, and refers you to its own website athttp://www.wyeth.com.au/go/top-navigation/media-room for further comment. |
| Etoricoxib, tablet, 30 mg and 60 mg, (Arcoxia®) Merck Sharp & Dohme Australia Pty Ltd Major submission |
Symptomatic treatment of the signs and symptoms of osteoarthritis (OA); treatment of acute gouty arthritis; and treatment of acute pain, including that related to primary dysmenorrhea and minor dental procedures. | Not PBS listed | ETORICOXIB Note: The use of etoricoxib for the treatment of the following conditions is not subsidised through the PBS: acute pain soft tissue injury; arthrosis without inflammatory component. Restricted Benefit Symptomatic treatment of osteoarthritis Note: No applications for increased maximum quantities and/or repeats will be authorised |
The PBAC rejected the submission on the basis that non-inferiority to celecoxib had not been demonstrated due to the inferior safety profile of etoricoxib in terms of hypertension and the potential poorer clinical outcome for the patient population for whom listing was sought. |
| Comparator: celecoxib |
Accepted |
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| Clinical Claim: Etoricoxib is: (1) no worse in terms of effectiveness with possibly better efficacy associated with the 60mg dose, and 2) in terms of safety, has i) similar changes from baseline in systolic blood pressure and diastolic blood pressure and ii) a higher but statistically insignificant rate of discontinuation due to hypertension-related adverse events, and a higher statistically significant rate of hypertension- related adverse events. |
Not accepted. The PBAC agreed that non-inferiority of etoricoxib 30 mg and 60 mg to celecoxib 200 mg was demonstrated in terms of clinical effectiveness. However the PBAC remained of the view that the non-inferiority of etoricoxib and celecoxib in terms of safety was not established. |
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| Economic Claim: cost minimisation |
Not accepted. The PBAC did not accept that non-inferiority of etoricoxib 30 mg or 60 mg to celecoxib 200 mg was demonstrated and agreed that the cost-minimisation approach taken in the re-submission was not adequately justified. |
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| Sponsor’s Comments: |
Nil |
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| Ivabradine, tablet, 5 mg and 7.5 mg (Coralan®) Servier Laboratories (Australia) Pty Ltd Major submission |
Treatment of chronic stable angina due to atherosclerotic coronary artery disease in patients with normal sinus rhythm who are unable to tolerate or have a contraindication to the use of beta blockers. | Not PBS listed | Authority Required Treatment of chronic stable angina due to atherosclerotic coronary artery disease in patients with normal sinus rhythm who are unable to tolerate or who have a contraindication to the use of beta blockers and have a resting heart rate of 75 bpm or greater. |
The PBAC rejected the submission because of uncertain clinical effectiveness in reducing mortality as claimed, and the resulting uncertain cost-effectiveness. |
| Comparator: Diltiazem and amlodipine. |
Partially accepted. The Committee considered a comparison with the long acting nitrates is also appropriate. |
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| Clinical Claim: There is inadequate evidence to make a definitive statement regarding therapeutic relativity. |
The PBAC considered the evidence for the comparative clinical effectiveness and safety for ivabradine in the treatment of angina was incomplete. Additionally there is insufficient evidence to directly support the claim that ivabradine’s putative superiority in reducing heart rate over diltiazem or amlodopine translates into reduced cardiovascular mortality. |
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| Economic Claim: Cost effectiveness |
Not accepted. The uncertainties in the clinical data mean that the cost-effectiveness analysis presented is inadequately supported. |
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| Sponsor’s comments: |
Nil |
