Botulinum Toxin Type A
Moderate to severe lower limb focal spasticity following a stroke or other acute central nervous system injury (including traumatic brain injury and spinal cord injury).
Decisions on record
- Meeting Nov 2008 Recommended Spasticity of the upper limbs of children with cerebral palsy
- Meeting Jul 2008 Recommended Spasticity of the upper limbs
- Meeting Jul 2008 Not recommended 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);
Access path
- Jul 2008Not recommended
uncertain clinical benefit, high and uncertain cost-effectiveness, post-hoc analysis of efficacy, no difference between…
- ↻ resubmittedNov 2008Recommended · restricted
ICER dropped $75,000 → $15,000 (-80%)
- Nov 2008Recommended · restricted
ICER dropped $75,000 → $15,000 (-80%)
- Nov 2009Not recommended
High and uncertain cost-effectiveness, uncertainty in utility values extrapolated to 12 months, model sensitivity to…
- ↻ resubmittedMar 2010Recommended · restricted
- Nov 2011Not recommended
uncertain clinical benefit (small and borderline clinically significant benefit compared to BSC, large placebo effect)…
- ↻ resubmittedJul 2012Recommended · restricted
- Jul 2013Recommended · restricted
Listing: Authority Required → Restricted
- Nov 2013Recommended · restricted
Listing: Restricted → Authority Required
- Mar 2017Recommended · restricted
Comparator changed: best supportive care → sacral neuromodulation (sacral nerve…
- Mar 2018Not recommended
Comparator changed: sacral neuromodulation (sacral nerve stimulation) → best supportive…
- ↻ resubmittedJul 2018Recommended · restricted
Evidence: Single-arm → Other
- Nov 2018Deferred
Evidence: Other → RCT
- Mar 2019Recommended · restricted
Comparator changed: Placebo/Standard management → standard of care / placebo
- PBS listing · Restricted
From the public summary
5.1 The PBAC recommended the Section 100 (Botulinum Toxin Program), Authority Required (STREAMLINED) listing of botulinum toxin type A (BOTOX®) for focal 9PSD · Mar 2019
5.2 In November 2018, the PBAC considered that there was a clinical need for effective treatments for lower limb focal spasticity following stroke. The PBAC noted the request to add additional acute aetiologies, including traumatic brain and spinal cord injuries, to the requested population. The PBAC noted that this request was supported by the Rehabilitation Medicine Society of Australia and New Zealand (RMSANZ).PSD · Mar 2019
4.5 The minor resubmission provided a new base case and sensitivity analyses based on advice provided by the PBAC when considered at the November 2018 meeting.PSD · Mar 2019
Changes to the economic model requested by the PBAC (BOTOX® PSD, November 2018) and presented in the resubmission included: A sensitivity analysis using an alternative response criteria (as defined by a change in Modified Ashworth Scale (MAS) score of > one point) (paragraph 7.12); A stepped economic analysis with a re-specified base case with the following parameters the PBAC considered a more appropriate base case would (paragraph 7.13): o …PSD · Mar 2019
4.4 Whilst the resubmission did not present new clinical information, the sponsor indicated it did not accept the PBAC position that BOTOX® was inferior in terms of safety compared to standard of care/placebo (paragraph 7.10, BOTOX® PSD, November 2018). The sponsor reiterated its argument that a clinical claim of non- inferior safety versus placebo was supported by the evidence presented in the previous submission.PSD · Mar 2019
6.2 The PBAC noted and welcomed the input from individuals (3), health care professionals (9) and organisations (1) via the Consumer Comments facility on the PBS website. The comments described a range of benefits of treatment with BOTOX®, as part of broader management plan, including improved mobility, reduction of pain, improved quality of life and a reduction in the burden of care.PSD · Nov 2018
Cost-effectiveness
result in an incremental cost-effectiveness ratio (ICER) of approximately $15,000 to $45,000 per quality adjusted life year (QALY). PSD · 2019
Decision context
PopulationAdults aged 18 years or older with moderate to severe lower limb focal spasticity following a stroke or other acute neurological event (including traumatic brain injury and spinal cord injury), defined as Modified Ashworth Scale rating of 3 or more.
Risk sharingSpecial Pricing Arrangements apply
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2019 | Recommended · restricted | standard of care / placebo | $15k–45k | RCT · MAS |
| Nov 2018 | Deferred | Placebo/Standard management | — | RCT · OS | PFS | DFS | ORR | QoL | Surrogate |
| Jul 2018 | Recommended · restricted | — | — | Other |
| Mar 2018 | Not recommended | best supportive care | — | Single-arm |
| Mar 2017 | Recommended · restricted | sacral neuromodulation (sacral nerve stimulation) | — | Other |
| Nov 2013 | Recommended · restricted | best supportive care | — | RCT |
| Jul 2013 | Recommended · restricted | best supportive care | $15k–45k | RCT · Headache days per month |
| Jul 2012 | Recommended · restricted | best supportive care | $15k–45k | RCT |
| Nov 2011 | Not recommended | best supportive care | $15k–45k | RCT · QoL, headache days per month |
| Mar 2010 | Recommended · restricted | placebo | $15k–45k | RCT |
| Nov 2009 | Not recommended | placebo | $15k–45k | RCT · HDSS |
| Nov 2008 | Recommended · restricted | standard management (physical and/or occupational therapy) | $15k | RCT · GAS, QUEST |
| Nov 2008 | Recommended · restricted | standard management (physical and/or occupational therapy) | $15k | RCT · GAS |
| Jul 2008 | Not recommended | placebo | $75k–105k | RCT |
Consumer voice
No consumer comments were received for this item.
The PBAC noted that no consumer comments were received for this item. Consumer comments · PSD